3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
200 recruiting
3
Rare Diseases
across 5 areas
0
News (30d)
Quiet
Belief BioMed Limited is a company with 3 orphan drug designations across 3 rare diseases. 5 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 |
|---|---|---|
| Duchenne muscular dystrophy | an adeno-associated virus vector expressing codon-optimized fusion of utrophin and dystrophin (iAAV9.6/shCK.mini-UDYS.spolyA) | Des.TrialAppr. |
| hemophilia A | Adeno-associated Virus Vector Containing an Expression Cassette of the Human Factor VIII Transgene | Des.TrialAppr. |
| hemophilia B | An Adeno-associated Virus Vector Containing an Expression Cassette of the Human Factor IX Transgene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
21
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
5
affecting portfolio
0% of portfolio targets high unmet need diseases
21
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
5
affecting portfolio