5
Orphan Designations
0 approved, 5 designated
0
FDA Approvals
1
Active Trials
96 recruiting
6
Rare Diseases
across 12 areas
0
News (30d)
Quiet
Healx Limited is a company with 5 orphan drug designations across 6 rare diseases. Active clinical trials in 1 indication. 2 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 |
|---|---|---|
| Angelman syndrome | 4,5,6,7-tetrahydroisoxazolo(5,4-c)pyridin-3-ol | Des.TrialAppr. |
| chromosome 17q11.2 deletion syndrome, 1.4Mb | Nitroxoline | Des.TrialAppr. |
| fragile X syndrome | IbudilastSulindacGaboxadol | Des.TrialAppr. |
| neurofibromatosis | - | Des.TrialAppr. |
| neurofibromatosis type 1 | Nitroxoline | Des.TrialAppr. |
| neurofibromatosis-Noonan syndrome | Nitroxoline | Des.TrialAppr. |
100% of portfolio targets high unmet need diseases
10
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
2
affecting portfolio
100% of portfolio targets high unmet need diseases
10
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
2
affecting portfolio