Specializing in the development of therapies for rare diseases, the company has one orphan drug designation and is active in treating Gaucher disease and several cancers.
1
Orphan Designations
0 approved, 1 designated
18
FDA Approvals
Latest: BONCRESA (2025)
0
Active Trials
200 recruiting
6
Rare Diseases
across 13 areas
0
News (30d)
Quiet
Amneal Pharmaceuticals LLC is a company with 1 orphan drug designation across 6 rare diseases, including 18 FDA-approved therapies. 6 active policy bills affect their portfolio.
Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.
Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA
| Disease | Drug(s) | Stage |
|---|---|---|
| Gaucher disease | Eliglustat Tartrate | Des.TrialAppr. |
| Gaucher disease type I | Eliglustat Tartrate | Des.TrialAppr. |
| Gaucher disease type III | Eliglustat Tartrate | Des.TrialAppr. |
| cervical cancer | ALYMSYS | Des.TrialAppr. |
| colorectal cancer | ALYMSYS | Des.TrialAppr. |
| renal cell carcinoma | ALYMSYS | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
11
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
6
affecting portfolio
0% of portfolio targets high unmet need diseases
11
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
6
affecting portfolio