5
Orphan Designations
0 approved, 5 designated
0
FDA Approvals
0
Active Trials
200 recruiting
5
Rare Diseases
across 11 areas
0
News (30d)
Quiet
AI Therapeutics, Inc. is a company with 5 orphan drug designations across 5 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 |
|---|---|---|
| amyotrophic lateral sclerosis | Apilimod | Des.TrialAppr. |
| bronchiolitis obliterans syndrome | Sirolimus | Des.TrialAppr. |
| follicular lymphoma | apilimod dimesylate | Des.TrialAppr. |
| sarcoidosis | Sirolimus | Des.TrialAppr. |
| tuberous sclerosis | sirolimus | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
7
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
7
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio