1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 3 areas
0
News (30d)
Quiet
AMMTeK is a company with 1 orphan drug designation across 3 rare diseases. 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 |
|---|---|---|
| neonatal diabetes mellitus with congenital hypothyroidism | glibentek (glibenclamide oral suspension) | Des.TrialAppr. |
| neonatal diabetes, congenital sensorineural hearing loss and congenital cataracts | glibentek (glibenclamide oral suspension) | Des.TrialAppr. |
| permanent neonatal diabetes mellitus | glibentek (glibenclamide oral suspension) | Des.TrialAppr. |
100% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
100% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
1/3
candidate diseases
0
avg importance: 0
0
affecting portfolio