A biopharmaceutical company focused on developing and providing treatments for rare diseases, with 27 FDA-approved drugs and active involvement in 16 rare conditions, including various forms of mastocytosis.
2
Orphan Designations
27
FDA Approvals
16
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Aicardi-Goutieres syndrome 7 | Silibinin-C-2',3-dihydrogensuccinate, disodium salt | Orphan Designation | - |
| Alstrom syndrome | Silibinin-C-2',3-dihydrogensuccinate, disodium salt | Orphan Designation | - |
| Gaucher disease | Silibinin-C-2',3-dihydrogensuccinate, disodium salt | Orphan Designation | - |
| Mycobacterium tuberculosis, susceptibility | PRETOMANID | - | FDA Approved |
| Smouldering systemic mastocytosis | GASTROCROM | - | FDA Approved |
| abdominal tuberculosis | PRETOMANID | - | FDA Approved |
| chronic obstructive pulmonary disease | YUPELRI | - | FDA Approved |
| extrapulmonary tuberculosis | PRETOMANID | - | FDA Approved |
| isolated bone marrow mastocytosis | GASTROCROM | - | FDA Approved |
| mastocytosis | GASTROCROM | - | FDA Approved |
| pediatric acute-onset neuropsychiatric syndrome | Revatio | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Revatio | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Revatio | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Revatio | Orphan Designation | - |
| pediatric-onset Graves disease | Revatio | Orphan Designation | - |
| pulmonary arterial hypertension | Revatio | Orphan Designation | - |