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:
Company CEO: Travis C. Mickle
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count: 1
Recent & Upcoming Milestones
- Clinical Outcomes Reported - Zevra presented P3 Niemann-Pick Disease, Type C results on 2026-02-05 for ARIMOCLOMOL CITRATE
- 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.
Highest Development Phases
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
2015-004438-93 |
Investigate study drug Arimoclomol with patients diagnosed with Niemann Pick disease type C. | P3 |
Completed |
Unknown |
2024-10-31 |
2025-06-30 |
Treatments |