Hematology Center of Excellence

Supporting patients living with a rare hematology condition

Orsini’s Hematology Center of Excellence launched in 2016 with our first program for paroxysmal nocturnal hemoglobinuria. Today we support multiple hematology programs across several rare conditions. Our dedicated Therapy Care Teams work with the physician’s office and insurance on a patient’s behalf so that our patients and their family have more time to focus on their treatment and quality of life. We are here to listen, provide counseling on treatment, including side-effect management, and assist with sourcing third-party financial assistance.

Explore Our Hematology Therapies

Benefits

Improving the Patient Experience, Every Time

Dedicated Care Team

Receive personalized support from a dedicated Therapy Care Team, including a primary pharmacy contact, clinical education, side-effects management, and scheduled refill calls.

Home Health Nursing

Access nationwide nursing support for in-home treatment or self-administration training. Our nurses provide clinical support to help patients achieve their treatment goals.

Insurance Coverage

Secure treatment coverage with assistance navigating payor and plan requirements. Our team proactively works with prescribers to obtain prior authorization and support appeals.

Free Care Overnight Shipping

Ensure medication and essential medical supplies arrive when and where patients need them through coordination across prescribers, patients, and care teams to arrange free overnight shipping.

Financial Assistance

Minimize financial exposure through financial assistance programs. We successfully identify and assist in enrolling patients into manufacturer co-pay and foundation support programs.

24/7/365 Access to a Pharmacist

Gain continuous access to a pharmacist, 24/7/365, ensuring ongoing care and clinical support for patients and prescribers to achieve better outcomes.

Support

Hematology Conditions Supported

Explore the rare hematology conditions and therapies Orsini supports below.

About Congenital Thrombotic Thrombocytopenic Purpura (c-TTP)

Congenital thrombotic thrombocytopenic purpura (c-TTP) is a rare and inherited blood disorder characterized by deficient activity of the enzyme ADAMTS13. This leads to the formation of blood clots in small blood vessels throughout the body, causing thrombocytopenia (low platelet count) and other complications. The lack of properly functioning ADAMTS13 impairs the breakdown of a specific blood clotting factor, resulting in the formation of microclots. c-TTP’s symptoms include purpura, neurological abnormalities, and organ damage. This lifelong condition poses challenges in maintaining blood clotting balance.

c-TTP Disease Therapies Supported

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

About Hemophilia B

Hemophilia B, also known as Christmas disease or factor IX deficiency, is a rare, degenerative disease that occurs in approximately 1 in 25,000 male births. People with this condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Hemophilia B can range from mild (factor IX levels between 5 and 40% of normal) to moderate (factor IX levels from 1 to 5% of normal) to severe (factor IX levels less than 1% of normal).

Hemophilia B Therapies Supported

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

About Sickle Cell Disease (SCD)

Sickle cell disease is an inherited blood disorder causing red blood cells to become “sickled,” leading to severe pain, organ damage, and shorter life expectancy. Hallmarked by painful blood vessel blockages (vaso-occlusive crises, or VOCs), SCD can also cause acute chest syndrome, stroke, jaundice, and heart failure symptoms.

SCD Therapies Supported

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	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

About Transfusion-Dependent Beta Thalassemia (TDT)

Transfusion-dependent beta thalassemia is an inherited blood disorder causing anemia, resulting in fatigue, shortness of breath, and, in infants, growth issues and jaundice. Complications can include an enlarged spleen, liver, or heart; misshapen bones; and delayed puberty. Many patients need regular blood transfusions, requiring frequent hospital visits and often causing iron buildup.

TDT Therapies Supported

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

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

Patient Advocacy Partners

The Alex Rezcalla Foundation advances the development of therapies for individuals with Duchenne muscular dystrophy (DMD) through advocacy and research funding. It raises awareness of the DMD experience by hosting events, engaging the public, and building networks of support.

Learn more at alexrezkallafoundation.org

The Amyloidosis Research improves the lives of patients with amyloidosis and accelerates cutting edge research through collaborative, groundbreaking initiatives. ARC provides support, information, and resources to patients and caregivers, empowering them to access the best care possible.

Learn more at aci.org

Cure SMA is dedicated to finding treatments and a cure for spinal muscular atrophy (SMA) by funding research and supporting families. Since 1984, Cure SMA has grown to be the largest U.S.-based network of individuals, families, clinicians, and research scientists working together to advance SMA research, support individuals and families impacted by SMA, and educate public and professional communities about SMA.

Learn more at www.curesma.org

The US Hereditary Angioedema Association (HAEA) is a 501 (c)(3) non-profit advocacy and research organization serving people living with the rare genetic condition, Hereditary Angioedema (HAE), and their families. Founded and staffed by people with HAE and caregivers, the US HAEA is committed to actively engaging our community in a wide variety of grassroots activities that promote HAE education and awareness.

Learn more at haea.org

The HypoPARAthyroidism Association is a non-profit patient association working to improve the lives of people impacted by hypoPARAthyroidism through education, support, research, and advocacy. They raise awareness of hypoPARAthyroidism and bring change to the lives of those affected.

Learn more at hypopara.org

Jett Foundation empowers people and families impacted by Duchenne muscular dystrophy through the development of transformative programming, educational opportunities, and ongoing support for every stage of a Duchenne journey.

Learn more at jettfoundation.org

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease.

Learn more at mda.org

The National MPS Society is committed to making a difference in the lives of MPS families through support, research, education, and advocacy. Families from around the world gain a better understanding of these rare genetically determined diseases through the Society’s assistance in linking them with healthcare professionals, researchers, and, perhaps most importantly, each other.

Learn more at mpssociety.org

The National Niemann-Pick Disease Foundation, Inc. (NNPDF) is a non-profit, patient advocacy and family support organization dedicated to supporting and empowering patients and families affected by Niemann-Pick disease, through education, collaboration and research. Founded in 1993, NNPDF serves families throughout the nation at all stages of their Niemann-Pick journey.

Learn more at nnpdf.org

The National PKU Alliance (NPKUA) works to improves the lives of individuals with PKU, pursue a cure by expanding research, and provide education and support to individuals living with PKU and their caregivers. Since its inception in 2008, NPKUA has supported numerous successful fundraising events, conferences, and internal support programs and has remained steadfast in its support of research endeavors.

Learn more at npkua.org

Parent Project Muscular Dystrophy (PPMD) is the largest nonprofit organization in the United States focused entirely on Duchenne muscular dystrophy. They take a comprehensive approach in the fight against Duchenne—funding research, raising awareness, promoting advocacy, connecting the community, and broadening treatment options.

Learn more at parentprojectmd.org

The Pompe Alliance is a Public Services Organization dedicated to the ideas of support, empowerment, and progress. It seeks to provide supportive services and information to patients, caregivers, medical professionals, and community stakeholders through a number of initiatives: financial assistance, advocacy, education, mentoring, and research recruitment

Learn more at pompealliance.com

The mission of the Pulmonary Fibrosis Foundation is to accelerate the development of new treatments and ultimately a cure for pulmonary fibrosis. Until this goal is achieved, the PFF is committed to advancing improved care of patients with PF and providing unequaled support and education resources for patients, caregivers, family members, and health care providers.

Learn more at pulmonaryfibrosis.org

The United Pompe Foundation was formed to assist patients and/or their families with medical costs and other expenses that these patents and families face and may not be able to cover, or fully cover, through their insurance.

Learn more at unitedpompe.com