Product Description
For Duchenne Muscular Dystrophy; (Sourced from: https://www.chugai-pharm.co.jp/english/profile/media/conference/files/181024ePresentation.pdf)
Mechanisms of Action: GDF8 Inhibitor
Novel Mechanism: Yes
Modality: Peptide/Protein
Route of Administration: Subcutaneous
FDA Designation: Fast Track - Muscular Atrophy, Spinal|Muscular AtrophyOrphan Drug - Muscular Atrophy, Spinal|Muscular Atrophy *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Pfizer
Company Location: NEW YORK NY 10017
Company CEO: Albert Bourla
Additional Commercial Interests: Bristol-Myers Squibb
Clinical Description
Countries in Clinic: Belgium, Czech Republic, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Active Clinical Trial Count: 3
Recent & Upcoming Milestones
Highest Development Phases
Phase 3: Muscular Atrophy, Spinal|Spinal Diseases
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
2022-000193-25 |
RESILIENT | P3 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2025-11-02 |
2025-05-23 |
Treatments |
|
NCT05337553 |
RESILIENT | P3 |
Active, not recruiting |
Muscular Atrophy, Spinal|Spinal Diseases |
2024-09-25 |
26% |
2025-06-19 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
2024-511852-42-00 |
BHV2000-301 | P3 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2025-10-08 |
2025-05-02 |
Treatments |