A subsidiary of Pfizer Inc., this company focuses on developing treatments for rare diseases, with 15 FDA-approved drugs and active involvement in 11 rare conditions, including hemophilia B and autism.
1
Orphan Designations
15
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| COVID-19–associated multisystem inflammatory syndrome in children | BeneFix | Orphan Designation | - |
| autism | BeneFix | Orphan Designation | - |
| bilirubin encephalopathy | BeneFix | Orphan Designation | - |
| febrile infection-related epilepsy syndrome | BeneFix | Orphan Designation | - |
| hemophilia B | BeneFix | Orphan Designation | - |
| intellectual developmental disorder, X-linked, syndromic, Pilorge type | BeneFix | Orphan Designation | - |
| intellectual disability-severe speech delay-mild dysmorphism syndrome | BeneFix | Orphan Designation | - |
| nut midline carcinoma | BeneFix | Orphan Designation | - |
| postmenopausal osteoporosis | PREMARIN | - | FDA Approved |
| renal cell carcinoma | TORISEL | - | FDA Approved |
| spinal muscular atrophy | BeneFix | Orphan Designation | - |