Product Description
Arimoclomol, taken at a dose of 200 mg three times daily will improve survival as defined by time to death, tracheostomy or permanent assisted ventilation. In addition, it will be safe and well tolerated in subjects with SOD1 positive familial ALS. (Sourced from: https://clinicaltrials.gov/ct2/show/NCT00706147)
Mechanisms of Action: HSP70 Modulator
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Fast Track - Amyotrophic Lateral Sclerosis *
Approval Status: Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: KemPharm
Company Location: CELEBRATION FL 34747
Company CEO: Travis C. Mickle
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Recent & Upcoming Milestones
- Zevra presented long-term real-world data supporting arimoclomol for Niemann-Pick Disease Type C at FDA's upcoming PDUFA date.
- The PDUFA action date for Arimoclomol's NDA is June 21, 2024, bringing it closer to potential approval for NPC.
- Zevra Therapeutics resubmitted NDA for arimoclomol for Niemann-Pick Disease Type C, awaiting FDA's PDUFA date confirmation within 30 days.
Highest Development Phases
Phase 3: Myositis, Inclusion Body
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|
| 2019-000749-11 | P3 |
Unknown status |
Myositis, Inclusion Body |
2023-09-09 |
2022-03-13 |
Treatments |
|
| 2017-004903-33 | P3 |
Unknown status |
Myositis, Inclusion Body |
2022-05-18 |
2022-03-13 |
Treatments |