Search By Condition or Specialty.
| Full Name |
Adzynma (ADAMTS13, recombinant-krhn) |
| Drug |
Adzynma |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) |
| Disease |
Congenital thrombotic thrombocytopenic purpura (c-TTP) |
| Therapeutic Area |
Hematology |
| Enrollment Form Link |
• Orsini Prescription Form
• Adzynma Patient Support Enrollment Form (optional) |
| Phone Number |
800-720-0789 |
| Fax Number |
877-251-0709 |
| Product Website |
adzynma.com |
| Full Name |
Aldurazyme® (laronidase) |
| Drug |
Aldurazyme |
| Manufacturer |
Genzyme Corporation |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms |
| Disease |
Mucopolysaccharidosis I |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
aldurazyme.com/healthcare |
| Patient Resources |
National MPS Society |
| Full Name |
Amvuttra® (vutrisiran) |
| Drug |
Amvuttra |
| Manufacturer |
Alnylam Pharmaceuticals, Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of:
- The polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
- The cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits
|
| Disease |
- Hereditary ATTR amyloidosis (hATTR) – Polyneuropathy (PN)
- Cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM)
|
| Therapeutic Area |
Neurology, Cardiology |
| Enrollment Form Link |
Start Form |
| Phone Number |
800-372-9581 |
| Product Website |
amvuttrahcp.com |
| Patient Resources |
Amyloidosis Research Consortium |
| Full Name |
ANDEMBRY® (garadacimab-gxii) |
| Drug |
garadacimab-gxii |
| Manufacturer |
CSL Behring |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
andembryhcp.com |
| Patient Resources |
HAEA |
| Full Name |
Arcalyst® (rilonacept) |
| Drug |
Arcalyst |
| Manufacturer |
Kiniksa Pharmaceuticals (UK), Ltd. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS), and Muckle-Wells Syndrome (MWS) in adults and children 12 years and older; maintenance of remission of Deficiency of Interleukin-1 Receptor Antagonist (DIRA) in adults and pediatric patients weighing 10 kg or more; and treatment of recurrent pericarditis (RP) and reduction in risk of recurrence in adults and children 12 years and older |
| Disease |
Cryopyrin-Associated Periodic Syndromes (CAPS); Deficiency of Interleukin-1 Receptor Antagonist (DIRA); Recurrent Pericarditis (RP) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
|
| Phone Number |
800-473-3261 |
| Fax Number |
877-576-6745 |
| Product Website |
arcalyst.com/hcp |
| Full Name |
ATTRUBY™ (acoramidis) |
| Drug |
Acoramidis |
| Manufacturer |
BridgeBio Pharma, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
ATTRUBY is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. Please see the full Prescribing Information. |
| Disease |
Cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
ATTRUBY Enrollment Form |
| Phone Number |
888-331-6574 |
| Fax Number |
877-349-1473 |
| Product Website |
www.attruby.com |
| Patient Resources |
Amyloidosis Research Consortium |
| Full Name |
Austedo® (deutetrabenazine) |
| Drug |
Austedo |
| Manufacturer |
Teva Neuroscience, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Indicated in adults for the treatment of Chorea associated with Huntington’s disease and tardive dyskinesia |
| Disease |
Tardive Dyskinesia (TD); Huntington’s Disease Chorea |
| Therapeutic Area |
Psychiatry, Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-691-0718 |
| Fax Number |
877-819-2424 |
| Product Website |
austedohcp.com |
| Full Name |
Austedo® XR (deutetrabenazine) |
| Drug |
Austedo XR Extended Release |
| Manufacturer |
Teva Neuroscience, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Indicated in adults for the treatment of Chorea associated with Huntington’s disease and tardive dyskinesia |
| Disease |
Tardive Dyskinesia (TD); Huntington’s Disease Chorea |
| Therapeutic Area |
Psychiatry, Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-691-0718 |
| Fax Number |
877-819-2424 |
| Product Website |
www.austedohcp.com |
| Full Name |
AVLAYAH™ (tividenofusp alfa-eknm) |
| Drug |
tividenofusp |
| Manufacturer |
Denali Therapeutics Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of neurologic manifestations of Hunter syndrome (Mucopolysaccharidosis type II, MPS II) when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment |
| Disease |
Mucopolysaccharidosis II (MPS II) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
|
| Phone Number |
800-240-9572 |
| Fax Number |
847-427-7976 |
| Product Website |
avlayah.com |
| Patient Resources |
National MPS Society |
| Full Name |
Berinert® [C1 esterase inhibitor (human)] |
| Drug |
Berinert |
| Manufacturer |
CSL Behring |
| Route of Administration |
Intravenous |
| Site of Care |
Home |
| Approved Indication |
Treatment of acute abdominal, facial, or laryngeal hereditary angioedema (HAE) attacks in adult and pediatric patients |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
berinert.com/professional |
| Patient Resources |
HAEA |
| Full Name |
Brineura® (cerliponase alfa) |
| Drug |
Brineura |
| Manufacturer |
BioMarin Pharmaceutical Inc. |
| Route of Administration |
Intraventricular |
| Site of Care |
Healthcare Facility |
| Approved Indication |
Indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency |
| Disease |
Ceroid Lipofuscinosis (CLN2) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
brineura.com/hcp |
| Patient Resources |
Batten Disease Support & Research Association |
| Full Name |
CASGEVY™ (exagamglogene autotemcel) |
| Drug |
Casgevy |
| Manufacturer |
Vertex Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
Treatment of patients aged 2 years and older with:
- Sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs)
- Transfusion-dependent β-thalassemia (TDT)
|
| Disease |
Sickle cell disease (SCD) and Transfusion-dependent β-thalassemia (TDT) |
| Therapeutic Area |
Hematology; Cell & Gene Therapy |
| Phone Number |
1-833-837-8395 |
| Full Name |
Cerdelga® (eliglustat) |
| Drug |
Cerdelga |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test |
| Disease |
Gaucher Type I Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
hcp.cerdelga.com |
| Patient Resources |
National Niemann Pick Disease Foundation |
| Full Name |
Cerezyme® (imiglucerase) |
| Drug |
Cerezyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions: anemia, thrombocytopenia, bone disease, hepatomegaly or splenomegaly |
| Disease |
Gaucher Type I Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
cerezyme.com/hcp |
| Patient Resources |
National Niemann Pick Disease Foundation |
| Full Name |
Cinryze® [C1 esterase inhibitor (human)] |
| Drug |
Cinryze |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Routine prophylaxis against angioedema attacks in adults, adolescents, and pediatric patients (6 years of age and older) with Hereditary Angioedema (HAE) |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
cinryze.com/hcp |
| Patient Resources |
HAEA |
| Full Name |
Crysvita® (burosumab-twza) |
| Drug |
Crysvita |
| Manufacturer |
Kyowa Kirin North America |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older and FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adult and pediatric patients 2 years of age and older |
| Disease |
Tumor-induced Osteomalacia (TIO); X-linked Hypophosphatemia (XLH) |
| Therapeutic Area |
Endocrinology & Bone Specialty |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-7614 |
| Fax Number |
877-793-4897 |
| Product Website |
crysvitahcp.com |
| Full Name |
DAWNZERA™ (donidalorsen) |
| Drug |
donidalorsen |
| Manufacturer |
Ionis Pharmaceuticals |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
844-444-4305 |
| Fax Number |
877-558-0007 |
| Product Website |
dawnzerahcp.com |
| Patient Resources |
HAEA |
| Full Name |
Dojolvi® (triheptanoin) |
| Drug |
Dojolvi |
| Manufacturer |
Ultragenyx |
| Route of Administration |
Oral or Gastrointestinal Tube |
| Site of Care |
Home |
| Approved Indication |
The treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD) |
| Disease |
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-546-7102 |
| Fax Number |
877-389-5186 |
| Product Website |
dojolvihcp.com |
| Full Name |
EKTERLY® (sebetralstat) |
| Drug |
sebetralstat |
| Manufacturer |
KalVista Pharmaceuticals |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
|
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
ekterly.com/ |
| Patient Resources |
HAEA |
| Full Name |
Elaprase® (idursulfase) |
| Drug |
Elaprase |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II) |
| Disease |
Mucopolysaccharidosis II |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
elaprase.com/hcp |
| Patient Resources |
National MPS Society |
| Full Name |
Elelyso® (taliglucerase alfa) |
| Drug |
Elelyso |
| Manufacturer |
Pfizer |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Long-term enzyme replacement therapy (ERT) for adults with a confirmed diagnosis of Type 1 Gaucher disease |
| Disease |
Gaucher Type I Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
elelyso.pfizerpro.com |
| Patient Resources |
National Niemann Pick Disease Foundation |
| Full Name |
Elevidys™ (delandistrogene moxeparvovec-rokl) |
| Drug |
Elevidys |
| Manufacturer |
Sarepta Therapeutics, Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
Treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age:
- For patients who are ambulatory and have a confirmed mutation in the DMD gene
- For patients who are non-ambulatory and have a confirmed mutation in the DMD gene
|
| Disease |
Duchenne muscular dystrophy (DMD) |
| Therapeutic Area |
Neurology; Genetics; Cell & Gene Therapy |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-870-8931 |
| Fax Number |
877-694-2546 |
| Product Website |
elevidyshcp.com |
| Patient Resources |
Alex Rezcalla Foundation, Jett Foundation, Muscular Dystrophy Association, and Parent Project Muscular Dystrophy |
| Full Name |
Elfabrio® (pegunigalsidase alfa-iwxj) |
| Drug |
Elfabrio |
| Manufacturer |
Chiesi |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of adults with confirmed Fabry disease |
| Disease |
Fabry disease |
| Therapeutic Area |
Neurology, Nephrology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
833-656-1056 |
| Fax Number |
636-355-3610 |
| Product Website |
hcp.elfabrio.com |
| Full Name |
ENCELTO™ (revakinagene taroretcel-lwey) |
| Drug |
Encelto |
| Manufacturer |
Neurotech Pharmaceuticals, Inc. |
| Route of Administration |
Intravitreal |
| Site of Care |
Healthcare facility |
| Approved Indication |
Indicated for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel) |
| Disease |
Idiopathic macular telangiectasia type 2 (MacTel) |
| Therapeutic Area |
Ophthalmology; Cell & Gene Therapy |
| Enrollment Form Link |
Start Form |
| Phone Number |
888-477-1165 |
| Product Website |
ENCELTO.com/ecp |
| Full Name |
Epioxa™ |
| Drug |
Epioxa |
| Manufacturer |
Glaukos Corporation |
| Route of Administration |
Ophthalmic |
| Site of Care |
Healthcare facility |
| Approved Indication |
Indicated for the treatment of keratoconus in adults and pediatric patients aged 13 years and older, in conjunction with the O2n System and the Boost Goggles |
| Disease |
Keratoconus |
| Therapeutic Area |
Ophthalmology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
847-849-1374 |
| Fax Number |
877-860-2146 |
| Product Website |
epioxa.com |
| Full Name |
Esbriet® (pirfenidone) |
| Drug |
Esbriet |
| Manufacturer |
Genentech |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of idiopathic pulmonary fibrosis (IPF) |
| Disease |
Idiopathic Pulmonary Fibrosis (IPF) |
| Therapeutic Area |
Pulmonology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-355-9366 |
| Fax Number |
877-358-9246 |
| Product Website |
esbriethcp.com |
| Patient Resources |
|
| Full Name |
Evkeeza® (evinacumab-dgnb) |
| Drug |
Evkeeza |
| Manufacturer |
Regeneron Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Adjunct to other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients, aged 5 years and older, with homozygous familial hypercholesterolemia (HoFH) |
| Disease |
Homozygous Familial Hypercholesterolemia (HoFH) |
| Therapeutic Area |
Cardiology, Metabolic Disorder |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-645-4142 |
| Fax Number |
877-473-0199 |
| Product Website |
evkeezahcp.com |
| Full Name |
Fabrazyme® (agalsidase beta) |
| Drug |
Fabrazyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease |
| Disease |
Fabry disease |
| Therapeutic Area |
Neurology, Nephrology, Genetics |
| Enrollment Form Link |
• CareConnect Personalized Support Services Enrollment
• Orsini Prescription Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
hcp.fabrazyme.com |
| Full Name |
Firazyr® (icatibant) |
| Drug |
Firazyr |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
firazyr.com/hcp |
| Patient Resources |
HAEA |
| Full Name |
Haegarda® [C1 esterase inhibitor subcutaneous (human)] |
| Drug |
Haegarda |
| Manufacturer |
CSL Behring |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Routine prophylaxis to prevent Hereditary Angioedema (HAE) attacks in patients 6 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
haegarda.com/hcp |
| Patient Resources |
HAEA |
| Full Name |
Hemgenix® (etranacogene dezaparvovec-drlb) |
| Drug |
Hemgenix |
| Manufacturer |
CSL Behring |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes |
| Disease |
Hemophilia B |
| Therapeutic Area |
Hematology; Cell & Gene Therapy |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-975-8693 |
| Fax Number |
877-740-7535 |
| Product Website |
hemgenix.com/hcp |
| Full Name |
Hepcludex® (bulevirtide‑gmod) |
| Drug |
bulevirtide |
| Manufacturer |
Gilead Sciences, Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
The treatment of chronic hepatitis delta (HDV) infection in adults without cirrhosis or with compensated cirrhosis |
| Disease |
Chronic hepatitis delta (HDV) infection |
| Therapeutic Area |
Hepatology |
| Phone Number |
847-457-4259 |
| Fax Number |
877-860-2504 |
| Enrollment Form Link |
Enrollment Form |
| Product Website |
hepcludexhcp.com |
| Full Name |
Icatibant Acetate |
| Drug |
icatibant acetate |
| Manufacturer |
Generic |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Patient Resources |
HAEA |
| Full Name |
iDose® TR (travoprost intracameral implant) |
| Drug |
travoprost |
| Manufacturer |
Glaukos Corporation |
| Route of Administration |
Intracameral |
| Site of Care |
Healthcare facility |
| Approved Indication |
Indicated for the reduction of intraocular pressure in patients with open-angle glaucoma or ocular hypertension |
| Disease |
Open-angle glaucoma; Ocular hypertension |
| Therapeutic Area |
Ophthalmology |
| Enrollment Form Link |
Start Form |
| Phone Number |
800-550-7202 |
| Fax Number |
877-277-3139 |
| Product Website |
idosetrhcp.com |
| Full Name |
Ingrezza® (valbenazine) |
| Drug |
Ingrezza |
| Manufacturer |
Neurocrine Biosciences |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of adults with tardive dyskinesia or chorea associated with Huntington’s disease |
| Disease |
Tardive Dyskinesia (TD); Huntington’s Disease Chorea |
| Therapeutic Area |
Psychiatry, Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-279-1676 |
| Fax Number |
877-868-1681 |
| Product Website |
ingrezzahcp.com |
| Full Name |
Isembyld™ (apitegromab-mstn) |
| Drug |
apitegromab |
| Manufacturer |
Scholar Rock |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of spinal muscular atrophy (SMA) in adults and pediatric patients 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment |
| Disease |
Spinal muscular atrophy (SMA) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-221-4943 |
| Fax Number |
847-892-1205 |
| Website |
isembyldhcp.com |
| Patient Resources |
Cure SMA and Muscular Dystrophy Association |
| Full Name |
ITVISMA® (onasemnogene abeparvovec-brve) |
| Drug |
onasemnogene abeparvovec-brve |
| Manufacturer |
Novartis Gene Therapy |
| Route of Administration |
Intrathecal |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in SMN1 gene |
| Disease |
Spinal Muscular Atrophy (SMA) |
| Therapeutic Area |
Neurology; Cell & Gene Therapy |
| Enrollment Form |
Enrollment Form |
| Phone Number |
800-697-5048 |
| Fax Number |
877-471-5704 |
| Website |
itvisma.com |
| Patient Resources |
Cure SMA and Muscular Dystrophy Association |
| Full Name |
JASCAYD® (nerandomilast) |
| Drug |
nerandomilast |
| Manufacturer |
Boehringer Ingelheim |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
- The treatment of idiopathic pulmonary fibrosis in adult patients
- The treatment of progressive pulmonary fibrosis in adult patients
|
| Disease |
Idiopathic pulmonary fibrosis (IPF); Progressive pulmonary fibrosis (PPF) |
| Therapeutic Area |
Pulmonology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
847-239-5646 |
| Fax Number |
847-423-6217 |
| Product Website |
jascayd.com |
| Patient Resources |
|
| Full Name |
Kalbitor® (ecallantide) |
| Drug |
Kalbitor |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Treatment of acute attacks of hereditary angioedema (HAE) in patients 12 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
kalbitor.com/hcp |
| Patient Resources |
HAEA |
| Full Name |
KEBILIDI™ (eladocagene exuparvovec-tneq) |
| Drug |
Kebilidi |
| Manufacturer |
PTC Therapeutics, Inc. |
| Route of Administration |
Intraputaminal |
| Site of Care |
Healthcare facility |
| Approved Indication |
Indicated for the treatment of adult and pediatric patients with aromatic 13 L-amino acid decarboxylase (AADC) deficiency |
| Disease |
Aromatic L-amino acid decarboxylase (AADC) deficiency |
| Therapeutic Area |
Neurology, Genetics, Cell & Gene Therapy |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-221-3495 |
| Fax Number |
877-810-7806 |
| Product Website |
kebilidi.com |
| Full Name |
LAMZEDE® (velmanase alfa-tycv) |
| Drug |
LAMZEDE |
| Manufacturer |
Chiesi Farmaceutici |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients |
| Disease |
Alpha-mannosidosis |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
(800) 240-9572 |
| Fax Number |
(847) 427-7976 |
| Product Website |
lamzede.com/hcp |
| Full Name |
LISRAYA™ (brepocitinib) |
| Drug |
brepocitinib |
| Manufacturer |
Priovant Therapeutics |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of dermatomyositis in adult patients |
| Disease |
Dermatomyositis |
| Therapeutic Area |
Rheumatology, Neurology, Dermatology |
| Phone Number |
847-453-3265 |
| Fax Number |
877-471-5764 |
| Enrollment Form Link |
Enrollment Form |
| Product Website |
lisrayahcp.com |
| Full Name |
Livdelzi® (seladelpar) |
| Drug |
Livdelzi |
| Manufacturer |
Gilead Sciences, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA |
| Disease |
Primary Biliary Cholangitis (PBC) |
| Therapeutic Area |
Hepatology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-263-8004 |
| Fax Number |
877-846-0402 |
| Product Website |
livdelzihcp.com |
| Patient Resources |
• PBCers Organization
• Orsini/PBCers 2025 Webinar – “Specialty Pharmacy: Partners in Your Care” | Watch On-Demand → |
| Full Name |
Loargys® (pegzilarginase-nbln) |
| Drug |
pegzilarginase |
| Manufacturer |
Immedica Pharma |
| Route of Administration |
Intravenous or Subcutaneous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
LOARGYS is an arginine specific enzyme indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency (ARG1-D), in conjunction with dietary protein restriction. |
| Disease |
Arginase 1 Deficiency (ARG1-D); Urea Cycle Disorders (UCDs) |
| Therapeutic Area |
Metabolic Disorder |
| Enrollment Form |
Enrollment Form |
| Phone Number |
844-982-5691, select option 2 |
| Fax Number |
844-982-5693 |
| Product Website |
loargys.com |
| Patient Resources |
flok |
| Full Name |
Lumizyme® (alglucosidase alfa) |
| Drug |
Lumizyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Patients with Pompe disease (GAA deficiency) |
| Disease |
Pompe Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form Link |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
lumizyme.com |
| Patient Resources |
Pompe Alliance, United Pompe Foundation |
| Full Name |
LYFGENIA™ (lovotibeglogene autotemcel) |
| Drug |
lovotibeglogene autotemcel |
| Manufacturer |
Genetix Biotherapeutics Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events |
| Disease |
Sickle Cell Disease (SCD) |
| Therapeutic Area |
Hematology; Cell & Gene Therapy |
| Enrollment Information |
Visit lyfgeniahcp.com |
| Phone Number |
847-849-1588 |
| Fax Number |
877-860-1726 |
| Website |
lyfgeniahcp.com |
| Full Name |
MACI® (autologous cultured chondrocytes on a porcine collagen membrane) |
| Drug |
MACI |
| Manufacturer |
Vericel Corporation |
| Route of Administration |
Surgery |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The repair of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults |
| Disease |
Cartilage Defect Repair |
| Therapeutic Area |
Orthopedics; Cell & Gene Therapy |
| Enrollment Form Link |
MACI Enrollment Form |
| Phone Number |
800-388-1903 |
| Fax Number |
N/A |
| Product Website |
maci.com |
| Full Name |
Mifepristone |
| Drug |
Mifepristone |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Control hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing’s syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery |
| Disease |
Cushing’s Syndrome |
| Therapeutic Area |
Endocrinology, Metabolic Disorder |
| Enrollment Form Link |
Mifepristone Enrollment Form |
| Phone Number |
(888) 204-7730 |
| Fax Number |
(877) 218-6288 |
| Full Name |
miglustat |
| Drug |
Miglustat |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option
Use in combination with MIPLYFFA™ (arimoclomol) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older |
| Disease |
Gaucher Type I Disease, Niemann-Pick disease type C (NPC) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Miglustat Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Patient Resources |
National Niemann Pick Disease Foundation |
| Full Name |
MIPLYFFA™ (arimoclomol) |
| Drug |
Miplyffa |
| Manufacturer |
Zevra Therapeutics, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older |
| Disease |
Niemann-Pick disease type C (NPC) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-668-4198 |
| Fax Number |
888-668-2143 |
| Product Website |
miplyffa.com |
| Full Name |
MYQORZO™ (aficamten) |
| Drug |
aficamten |
| Manufacturer |
Cytokinetics, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
MYQORZO is a cardiac myosin inhibitor indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. |
| Disease |
Obstructive hypertrophic cardiomyopathy (oHCM) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
847-849-1445 |
| Fax Number |
847-892-1127 |
| Product Website |
www.myqorzohcp.com |
| REMS Website |
www.myqorzorems.com |
| Full Name |
Naglazyme® (galsulfase) |
| Drug |
Naglazyme |
| Manufacturer |
BioMarin Pharmaceutical Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy Syndrome) |
| Disease |
Mucopolysaccharidosis VI |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Naglazyme Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
naglazyme.com |
| Patient Resources |
National MPS Society |
| Full Name |
Nexviazyme® (avalglucosidase alfa-ngpt) |
| Drug |
Nexviazyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of patients 1 year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) |
| Disease |
Pompe Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Nexviazyme Enrollment Form Link |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
nexviazyme.com |
| Patient Resources |
Pompe Alliance, United Pompe Foundation |
| Full Name |
nintedanib |
| Drug |
nintedanib |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Indicated in adults for:
• Treatment of idiopathic pulmonary fibrosis (IPF)
• Treatment of chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype |
| Disease |
Idiopathic Pulmonary Fibrosis (IPF); Chronic Fibrosing Interstitial Lung Diseases (ILDs) |
| Therapeutic Area |
Pulmonology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-373-1452 |
| Fax Number |
888-975-1456 |
| Patient Resources |
|
| Full Name |
Ofev® (nintedanib) |
| Drug |
Ofev |
| Manufacturer |
Boehringer Ingelheim |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Treatment of idiopathic pulmonary fibrosis (IPF) in adults, treatment of chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype in adults, or slowing the rate of decline in pulmonary function in adults with systemic sclerosis-associated interstitial lung disease (SSc-ILD) |
| Disease |
Idiopathic Pulmonary Fibrosis (IPF); Chronic Fibrosing Interstitial Lung Diseases (ILDs); Systemic Sclerosis Interstitial Lung Disease (SSc-ILD) |
| Therapeutic Area |
Pulmonology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-373-1452 |
| Fax Number |
888-975-1456 |
| Product Website |
ofev.com |
| Patient Resources |
|
| Full Name |
OLPRUVA® (sodium phenylbutyrate) |
| Drug |
Olpruva |
| Manufacturer |
Zevra Therapeutics, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Chronic management of adult and pediatric patients weighing 20 kg or greater and with a body surface area (BSA) of 1.2 m2 or greater, with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS) |
| Disease |
Urea Cycle Disorders (UCDs) |
| Therapeutic Area |
Metabolic Disorder |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-837-8905 |
| Fax Number |
877-369-3806 |
| Product Website |
olpruvahcp.com |
| Patient Resources |
flok |
| Full Name |
Onpattro® (patisiran) |
| Drug |
Onpattro |
| Manufacturer |
Alnylam Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults |
| Disease |
Hereditary ATTR amyloidosis (hATTR) – Polyneuropathy (PN) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Onpattro Start Form |
| Phone Number |
800-690-8236 |
| Fax Number |
877-445-8481 |
| Product Website |
onpattro.com |
| Patient Resources |
Amyloidosis Research Consortium |
| Full Name |
Oxlumo® (lumasiran) |
| Drug |
Oxlumo |
| Manufacturer |
Alnylam Pharmaceuticals |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients |
| Disease |
Primary Hyperoxaluria Type 1 (PH1) |
| Therapeutic Area |
Nephrology |
| Enrollment Form Link |
Oxlumo Start Form |
| Phone Number |
800-460-5217 |
| Fax Number |
877-276-8563 |
| Product Website |
oxlumo.com |
| Full Name |
PALSONIFY™ (paltusotine) |
| Drug |
paltusotine |
| Manufacturer |
Crinetics Pharmaceuticals, Inc. |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option |
| Disease |
Acromegaly |
| Therapeutic Area |
Endocrinology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
847-239-5680 |
| Fax Number |
847-892-1203 |
| Product Website |
palsonify.com |
| Full Name |
PASATRU™ (garetosmab-grts) |
| Drug |
PASATRU™ |
| Manufacturer |
Regeneron Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
To reduce formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP) |
| Disease |
Fibrodysplasia Ossificans Progressiva (FOP) |
| Therapeutic Area |
Rheumatology, Genetics |
| Enrollment Form Link |
www.pasatruhcp.com |
| Phone Number |
847-464-8049 |
| Fax Number |
847-892-1181 |
| Product Website |
www.pasatruhcp.com |
| Full Name |
Photrexa® (riboflavin 5’-phosphate ophthalmic solution) |
| Drug |
Photrexa |
| Manufacturer |
Glaukos |
| Route of Administration |
Intraocular |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of progressive keratoconus (1.1) and corneal ectasia following refractive surgery |
| Disease |
Keratoconus; Corneal Ectasia |
| Therapeutic Area |
Ophthalmology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-550-7207 |
| Fax Number |
877-277-3139 |
| Full Name |
pirfenidone |
| Drug |
Pirfenidone |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of idiopathic pulmonary fibrosis (IPF) |
| Disease |
Idiopathic Pulmonary Fibrosis (IPF) |
| Therapeutic Area |
Pulmonology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-355-9366 |
| Fax Number |
877-358-9246 |
| Patient Resources |
|
| Full Name |
Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) |
| Drug |
Pombiliti & Opfolda |
| Manufacturer |
Amicus Therapeutics |
| Route of Administration |
Intravenous and oral |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT) |
| Disease |
Pompe Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Pombiliti & Opfolda Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
pombilitiopfolda.com |
| Patient Resources |
Pompe Alliance, United Pompe Foundation |
| Full Name |
Ruconest® [C1 esterase inhibitor (recombinant)] |
| Drug |
Ruconest |
| Manufacturer |
Pharming Group |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE) |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Ruconest Patient Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
ruconest.com |
| Patient Resources |
HAEA |
| Full Name |
SAJAZIR™ (icatibant) |
| Drug |
Sajazir |
| Manufacturer |
Cycle Pharmaceuticals |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
(800) 356-4252 |
| Fax Number |
(877) 277–7318 |
| Product Website |
sajazir.com/hcp/ |
| Patient Resources |
HAEA |
| Full Name |
Sapropterin |
| Drug |
sapropterin dihydrochloride |
| Manufacturer |
Generic |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU), in conjunction with a Phe-restricted diet |
| Disease |
Phenylketonuria (PKU) |
| Therapeutic Area |
Endocrinology, Metabolic Disorder |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
(773) 598-6135 |
| Fax Number |
(877) 299-8203 |
| Patient Resources |
Alliance of PKU Families, flok, and National PKU Alliance |
| Full Name |
SEPHIENCE™ (sepiapterin) |
| Drug |
sepiapterin |
| Manufacturer |
PTC Therapeutics |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU) |
| Disease |
Phenylketonuria (PKU) |
| Therapeutic Area |
Endocrinology, Metabolic Disorder |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
(847) 521-4499 |
| Fax Number |
(888) 726-9306 |
| Product Website |
sephience.com |
| Patient Resources |
Alliance of PKU Families, flok, and National PKU Alliance |
| Full Name |
SKYSONA® (elivaldogene autotemcel) |
| Drug |
elivaldogene autotemcel |
| Manufacturer |
Genetix Biotherapeutics Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
Indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD) without an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant |
| Disease |
Cerebral Adrenoleukodystrophy (CALD) |
| Therapeutic Area |
Neurology; Cell & Gene Therapy |
| Enrollment Information |
Visit skysona.com |
| Phone Number |
847-849-1588 |
| Fax Number |
877-860-1726 |
| Website |
skysona.com |
| Full Name |
Takhzyro® (lanadelumab-flyo) |
| Drug |
Takhzyro |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
Prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 2 years and older |
| Disease |
Hereditary Angioedema (HAE) |
| Therapeutic Area |
Allergy & Immunology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-356-4252 |
| Fax Number |
847-631-6918 |
| Product Website |
takhzyro.com |
| Patient Resources |
HAEA |
| Full Name |
Tzield® (teplizumab-mzwv) |
| Drug |
Tzield |
| Manufacturer |
Sanofi |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Indicated to:
- Delay the onset of Stage 3 type 1 diabetes (T1D) in adult and pediatric patients 1 year of age and older with Stage 2 T1D
- Delay the decline in endogenous insulin production in pediatric patients aged 8 to 17 years recently diagnosed with Stage 3 T1D. This indication is approved under accelerated approval based on evidence of reduced C-peptide decline. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
|
| Disease |
Type 1 Diabetes (T1D) |
| Therapeutic Area |
Endocrinology & Bone Specialty |
| Enrollment Form Link |
Tzield Enrollment Form |
| Phone Number |
800-670-5321 |
| Fax Number |
877-655-4364 |
| Product Website |
tzield.com |
| Full Name |
Veopoz™ (pozelimab-bbfg) |
| Drug |
Veopoz |
| Manufacturer |
Regeneron Pharmaceuticals |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease |
| Disease |
CD55-deficient protein-losing enteropathy (CHAPLE) Disease |
| Therapeutic Area |
Allergy & Immunology, Genetics |
| Enrollment Form Link |
Veopoz Enrollment Form |
| Phone Number |
800-438-2375 |
| Fax Number |
877-440-0891 |
| Product Website |
veopoz.com |
| Full Name |
Viltepso® (viltolarsen) |
| Drug |
Viltepso |
| Manufacturer |
NS Pharma |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
The treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping |
| Disease |
Duchenne Muscular Dystrophy (DMD) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Viltepso Enrollment Form |
| Phone Number |
800-759-0445 |
| Fax Number |
877-286-3620 |
| Product Website |
viltepso.com |
| Patient Resources |
Alex Rezcalla Foundation, Jett Foundation, Muscular Dystrophy Association, and Parent Project Muscular Dystrophy |
| Full Name |
Vimizim® (elosulfase alfa) |
| Drug |
Vimizim |
| Manufacturer |
BioMarin Pharmaceutical Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) |
| Disease |
Mucopolysaccharidosis IVA |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Vimizim Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
vimizim.com |
| Patient Resources |
National MPS Society |
| Full Name |
Vowst™ (fecal microbiota spores live-brpk) |
| Drug |
Vowst |
| Manufacturer |
Aimmune Therapeutics |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
Prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI) |
| Disease |
Recurrent C. difficile Infection (rCDI) |
| Therapeutic Area |
Infectious Disease; Gastroenterology |
| Enrollment Form Link |
|
| Phone Number |
800-485-4885 |
| Fax Number |
877-640-5179 |
| Product Website |
vowsthcp.com |
| Full Name |
Vpriv® (velaglucerase alfa) |
| Drug |
Vpriv |
| Manufacturer |
Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Long-term enzyme replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease |
| Disease |
Gaucher Type I Disease |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Vpriv Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
hcp.vpriv.com |
| Patient Resources |
National Niemann Pick Disease Foundation |
| Full Name |
Vyepti® (eptinezumab-jjmr) |
| Drug |
Vyepti |
| Manufacturer |
Lundbeck |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The preventive treatment of migraine in adults |
| Disease |
Migraine |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Vyepti Enrollment Form |
| Phone Number |
800-259-7145 |
| Fax Number |
877-892-3019 |
| Product Website |
vyeptihcp.com |
| Full Name |
Vyndamax® (tafamidis) |
| Drug |
Vyndamax |
| Manufacturer |
Pfizer |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization |
| Disease |
Hereditary ATTR amyloidosis (hATTR) – Cardiomyopathy (CM); Wild type transthyretin amyloid cardiomyopathy (wtATTR-CM) |
| Therapeutic Area |
Cardiology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
800-930-2043 |
| Fax Number |
877-684-3116 |
| Product Website |
vyndamax.pfizerpro.com |
| Patient Resources |
Amyloidosis Research Consortium |
| Full Name |
Wainua™ (eplontersen) |
| Drug |
Wainua |
| Manufacturer |
AstraZeneca |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
The treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults |
| Disease |
Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Wainua Enrollment Form |
| Phone Number |
800-986-4975 |
| Fax Number |
877-369-5207 |
| Product Website |
wainuahcp.com |
| Patient Resources |
Amyloidosis Research Consortium |
| Full Name |
Xenpozyme® (olipudase alfa-rpcp) |
| Drug |
Xenpozyme |
| Manufacturer |
Sanofi Genzyme |
| Route of Administration |
Intravenous |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients |
| Disease |
Acid Sphingomyelinase Deficiency (ASMD) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Xenpozyme Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Product Website |
xenpozyme.com |
| Full Name |
Yargesa™ (miglustat) |
| Drug |
Yargesa |
| Manufacturer |
Edenbridge Pharmaceuticals |
| Route of Administration |
Oral |
| Site of Care |
Home or Healthcare Facility |
| Approved Indication |
Monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option
Use in combination with MIPLYFFA™ (arimoclomol) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older |
| Disease |
Gaucher Type I Disease, Niemann-Pick disease type C (NPC) |
| Therapeutic Area |
Neurology, Genetics |
| Enrollment Form Link |
Yargesa Enrollment Form |
| Phone Number |
800-240-9572 |
| Fax Number |
877-220-7581 |
| Patient Resources |
National Niemann Pick Disease Foundation |
| Full Name |
YORVIPATH® (palopegteriparatide) |
| Drug |
Yorvipath |
| Manufacturer |
Ascendis Pharma |
| Route of Administration |
Subcutaneous |
| Site of Care |
Home |
| Approved Indication |
The treatment of hypoparathyroidism in adults |
| Disease |
Hypoparathyroidism |
| Therapeutic Area |
Endocrinology |
| Enrollment Form Link |
Enrollment Form |
| Phone Number |
888-204-7802 |
| Fax Number |
877-471-8175 |
| Product Website |
yorvipathhcp.com |
| Patient Resources |
HypoPARAthyroidism Association |
| Full Name |
Zolgensma® (onasemnogene abeparvovec-xioi) |
| Drug |
Zolgensma |
| Manufacturer |
Novartis Gene Therapy |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene |
| Disease |
Spinal Muscular Atrophy (SMA) |
| Therapeutic Area |
Neurology; Cell & Gene Therapy |
| Enrollment Form |
Enrollment Form |
| Phone Number |
800-697-5048 |
| Fax Number |
877-471-5704 |
| Website |
zolgensma.com |
| Patient Resources |
Cure SMA and Muscular Dystrophy Association |
| Full Name |
Ztalmy® (ganaxolone) |
| Drug |
ganaxolone |
| Manufacturer |
Immedica Pharma |
| Route of Administration |
Oral |
| Site of Care |
Home |
| Approved Indication |
The treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older |
| Disease |
Cyclin-dependent kinase-like 5 (CDKL5) Deficiency Disorder |
| Therapeutic Area |
Neurology |
| Enrollment Form Link |
Ztalmy Enrollment Form |
| Phone Number |
844-ZTALMY1 |
| Fax Number |
844-ZTALMYF |
| Website |
ztalmy.com |
| Full Name |
ZYNTEGLO™ (betibeglogene autotemcel) |
| Drug |
betibeglogene autotemcel |
| Manufacturer |
Genetix Biotherapeutics Inc. |
| Route of Administration |
Intravenous |
| Site of Care |
Healthcare Facility |
| Approved Indication |
The treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions |
| Disease |
Transfusion-dependent β-thalassemia (TDT) |
| Therapeutic Area |
Hematology; Cell & Gene Therapy |
| Enrollment Information |
Visit zynteglohcp.com |
| Phone Number |
847-849-1588 |
| Fax Number |
877-860-1726 |
| Website |
zynteglohcp.com |
