1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
4
Active Trials
200 recruiting
4
Rare Diseases
across 10 areas
0
News (30d)
Quiet
AskBio Inc. is a company with 1 orphan drug designation across 4 rare diseases. Active clinical trials in 4 indications.
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 |
|---|---|---|
| Parkinson disease | - | Des.TrialAppr. |
| glycogen storage disease II | non-replicating single stranded recombinant adeno-associated viral vector with a thyroxine hormone binding globulin (THBG) promoter expressing the modified human acid alpha-glucosidase gene (hGAA) | Des.TrialAppr. |
| multiple system atrophy | - | Des.TrialAppr. |
| muscular dystrophy | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
23
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
23
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio