Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat)
| 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 |