3
Orphan Designations
1 approved, 2 designated
4
FDA Approvals
Latest: DAYBUE STIX (2025)
1
Active Trials
200 recruiting
5
Rare Diseases
across 12 areas
1
News (30d)
Acadia Pharmaceuticals Inc. is a company with 3 orphan drug designations across 5 rare diseases, including 4 FDA-approved therapies. Active clinical trials in 1 indication. 2 active policy bills affect their portfolio. 1 news article in the past 30 days.
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 |
|---|---|---|
| Alzheimer disease | - | Des.TrialAppr. |
| FOXG1 disorder | Daybue Stix | Des.TrialAppr. |
| Prader-Willi syndrome | carbetocin (nasal spray) | Des.TrialAppr. |
| Rett syndrome | Daybue Stix | Des.TrialAppr. |
| fragile X syndrome | Trofinetide | Des.TrialAppr. |
50% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/5
candidate diseases
1
avg importance: 70
2
affecting portfolio
50% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/5
candidate diseases
1
avg importance: 70
2
affecting portfolio