Zevra Therapeutics
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2006-01-01
- Employees
- 69
- Market Cap
- $379.3M
- Website
- http://zevra.com
- Introduction
Zevra Therapeutics, Inc. is a commercial-stage rare disease company that combines science, data and patient needs to create therapies for diseases with limited or no treatment options. The firm focuses on the treatment of rare neurological and sleep conditions such as certain Urea Cycle Disorders, Niemann Pick Disease Type C and Idiopathic Hypersomnia. The company receives royalties for AZSTARYS from its commercial partner Corium. The company was founded by Christal M. M. Mickle and Travis C. Mickle on October 30, 2006 and is headquartered in Celebration, FL.
Zevra Therapeutics' MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment
Zevra Therapeutics announces FDA approval of MIPLYFFA, the first treatment for Niemann-Pick disease type C (NPC), indicated for neurological manifestations in adults and pediatric patients ≥2 years, in combination with miglustat. The company also receives a rare pediatric disease priority review voucher and launches AmplifyAssist™ patient support program.
FDA approves arimoclomol as first drug to treat Neimann-Pick disease, type C
FDA approves arimoclomol, in combination with miglustat, as the first treatment for Niemann-Pick disease, type C (NPC) in adults and children aged 2 and older. Arimoclomol demonstrated efficacy in slowing disease progression in a 12-month trial, with common side effects including upper respiratory tract infection, diarrhea, and decreased weight.
Related Clinical Trials:
FDA Approves First Treatment for Niemann-Pick Disease, Type C
FDA approves Miplyffa (arimoclomol), the first drug for treating Niemann-Pick disease, type C (NPC), in combination with miglustat for neurological symptoms in adults and children 2 years and older.
Arimoclomol Becomes First FDA Approved Treatment for Neimann-Pick Disease Type C
Arimoclomol, an oral medication, was approved by the FDA for treating Niemann-Pick disease, type C (NPC), in combination with miglustat. This marks the first FDA-approved treatment for NPC, addressing neurological symptoms in adults and children aged 2 years or older.
FDA Approves Miplyffa (arimoclomol) for the Treatment of Niemann-Pick Disease Type C
FDA approves Miplyffa (arimoclomol) for Niemann-Pick disease type C (NPC), the first NPC drug. Indicated for neurological manifestations in adult and pediatric patients 2 years and older, in combination with miglustat. Zevra Therapeutics receives rare pediatric disease priority review voucher.
FDA Action Alert: BMS, Merck, Sanofi/Regeneron and More
FDA decision deadlines include Vanda's tradipitant for gastroparesis (Sept 18), Zevra's arimoclomol for Niemann-Pick disease type C (Sept 21), Heron's extended-release needle for Zynrelef (Sept 23), Merck's Keytruda for pleural mesothelioma (Sept 25), and Bristol Myers Squibb's KarXT for schizophrenia (Sept 26). Sanofi and Regeneron await FDA verdict on Dupixent for COPD (Sept 27).
FDA Advisory Committee Votes Favorably that the Data Support Arimoclomol as Effective Treatment for Patients with Niemann-Pick Disease Type C
FDA's GeMDAC voted favorably (11 yes, 5 no) that arimoclomol is effective for Niemann-Pick disease type C (NPC). Zevra Therapeutics remains confident in arimoclomol's clinical benefit and its path to approval, with a PDUFA action date of September 21, 2024.
Related Clinical Trials:
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