1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
1
Active Trials
30 recruiting
2
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Myrtelle, Inc. is a company with 1 orphan drug designation across 2 rare diseases. Active clinical trials in 1 indication. gene therapy candidates in 1 disease.
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 |
|---|---|---|
| Canavan disease | recombinant adeno-associated virus containing human ASPA cDNA (rAAV-Olig001-ASPA) | Des.TrialAppr. |
| leukodystrophy | - | Des.TrialAppr. |
100% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
1/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
100% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
1/2
candidate diseases
0
avg importance: 0
0
affecting portfolio