Product Description
AP-103 is a preclinical drug candidate to treat epidermolysis bullosa from Amyrt Pharma. (Sourced from: https://amrytpharma.com/pipeline/)
Mechanisms of Action: Gene Therapy, COLVII
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: Injection
FDA Designation: Orphan Drug - Epidermolysis Bullosa *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Company: Chiesi
Company Location: Europe
Company Founding Year: 1935
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Recent & Upcoming Milestones
Highest Development Phases
Phase 0: Epidermolysis Bullosa
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
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Recent News Events
Date |
Type |
Title |
|---|---|---|
|
01/05/2023 |
News Article |
Amryt Announces Positive Top Line Results from Phase 3 Pediatric Trial of Lomitapide in HoFH |
|
12/19/2022 |
News Article |
COMP Adopts Positive Opinion on Orphan Designation for Mycapssa® for the Treatment of Carcinoid Syndrome Associated with Neuroendocrine Tumors |
|
12/05/2022 |
News Article |
European Commission approves Mycapssa® for the treatment of Acromegaly |
|
11/30/2022 |
News Article |
Amryt Announces New Patent for Mycapssa® |