1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
2
Active Trials
200 recruiting
3
Rare Diseases
across 6 areas
0
News (30d)
Quiet
Insmed Gene Therapy LLC is a company with 1 orphan drug designation across 3 rare diseases. Active clinical trials in 2 indications. 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 | single-stranded, non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) vector containing alpha-myosin-heavy-chain/murine muscle creatine kinase 7 (MHCK7) promoter and micro-dystrophin elements | Des.TrialAppr. |
| amyotrophic lateral sclerosis | - | Des.TrialAppr. |
| muscular dystrophy | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
43
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
5
affecting portfolio
0% of portfolio targets high unmet need diseases
43
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
5
affecting portfolio