3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
200 recruiting
2
Rare Diseases
across 5 areas
0
News (30d)
Quiet
Biohaven Pharmaceutical Holding Company, Ltd. is a company with 3 orphan drug designations across 2 rare diseases.
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 |
|---|---|---|
| Rett syndrome | [(S)-1-(L-valyl)-N_((S)-1-phenyl-2-(pyridin-2-yl)ethyl)pyrrolidine-2-carboxamide fumarate] | Des.TrialAppr. |
| amyotrophic lateral sclerosis | riluzole orally disolving tablet | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio