4
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
6
Rare Diseases
across 10 areas
0
News (30d)
Quiet
Shire Human Genetic Therapies is a company with 4 orphan drug designations across 6 rare diseases.
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 |
|---|---|---|
| burn | icatibant | Des.TrialAppr. |
| burn goodship syndrome | icatibant | Des.TrialAppr. |
| choanal atresia-hearing loss-cardiac defects-craniofacial dysmorphism syndrome | icatibant | Des.TrialAppr. |
| metachromatic leukodystrophy | recombinant human arylsulphatase A | Des.TrialAppr. |
| metachromatic leukodystrophy, adult form | recombinant human arylsulphatase A | Des.TrialAppr. |
| metachromatic leukodystrophy, juvenile form | recombinant human arylsulphatase A | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
2
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
0
affecting portfolio