2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 6 areas
0
News (30d)
Quiet
Elpida Therapeutics SPC is a company with 2 orphan drug designations across 2 rare diseases. 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 |
|---|---|---|
| Krabbe disease | recombinant serotype 9 adeno-associated virus (AAV) encoding a human galactosylceramidase (GALC) transgene (hGALC) | Des.TrialAppr. |
| neuronal ceroid lipofuscinosis 7 | adeno-associated viral vector serotype 9 encoding a codon-optimized human ceroid neuronal lipofuscinosis type 7 (CLN7) transgene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
2
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
2
affecting portfolio