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.
First Drug Approved for Niemann-Pick Disease Type C
FDA approves arimoclomol (Miplyffa) as the first treatment for Niemann-Pick disease type C (NPC), based on a phase II/III trial showing slower disease progression with arimoclomol combined with miglustat. The approval addresses a significant unmet need, with an estimated 900 U.S. patients affected by NPC.
FDA Approves First Treatment for Niemann-Pick Disease Type C
The FDA has approved arimoclomol (Miplyffa) for treating Niemann-Pick disease type C (NPC) in patients aged 2 years and older, in combination with miglustat. This approval is based on a phase 2/3 trial showing arimoclomol met its primary outcome measure of improving the NPC Clinical Severity Scale score. Arimoclomol, an orally delivered medication, increases the activation of transcription factors EB and E3, leading to upregulation of lysosomal genes. Zevra Therapeutics plans to launch the therapy in the U.S. within 8 to 12 weeks.
Related Clinical Trials:
FDA Approves First Treatment for Niemann-Pick Disease, Type C
FDA approves Miplyffa (arimoclomol), the first drug for Niemann-Pick disease, type C (NPC), in combination with miglustat to treat neurological symptoms in adults and children 2 years and older.
FDA Approves Arimoclomol, First Drug for Niemann-Pick Disease, Type C
The FDA approved Zevra Therapeutics’ arimoclomol (Miplyffa) for treating neurological symptoms of Niemann-Pick disease, type C (NPC) in adults and children aged ≥2 years, marking the first drug approved for NPC. Arimoclomol, in combination with miglustat, demonstrated efficacy in slowing disease progression in a 12-month trial. Common side effects include upper respiratory tract infection, diarrhea, and decreased weight.
Zevra Therapeutics gains FDA approval of arimoclomol for rare neurological disease
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FDA approves Miplyffa for Niemann-Pick disease type C
FDA approves Miplyffa, the first drug for treating neurological symptoms of Niemann-Pick disease type C in patients aged 2 and older, based on a 12-month trial showing efficacy.
FDA Approval Makes a Zevra Drug the First Therapy for Rare and Fatal Metabolic Disease
Zevra Therapeutics' drug, Miplyffa (acrimoclomol), is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), a fatal inherited metabolic disorder. The drug, taken three times daily, aims to slow NPC's progression, with FDA approval specifically for neurological effects. Miplyffa should be used with miglustat, and common side effects include upper respiratory tract infection, diarrhea, and weight loss. Zevra expects the drug to be available in the U.S. within 8-12 weeks.
US FDA approves Zevra's genetic disorder treatment
The FDA approved Zevra Therapeutics' Miplyffa, the first treatment for Neimann-Pick disease type C, a fatal genetic disorder affecting the nervous system. Miplyffa, in combination with miglustat, treats neurological symptoms in adults and children aged 2 and older. The drug carries a warning for hypersensitivity reactions.
Zevra Therapeutics' MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment
FDA approves MIPLYFFA, the first treatment for Niemann-Pick disease type C (NPC), in combination with miglustat for neurological manifestations in adults and pediatric patients 2 years and older. Zevra Therapeutics receives a rare pediatric disease priority review voucher and launches AmplifyAssist™ patient support program. Conference call scheduled for Sept. 23, 2024.
FDA Approves Zevra's Miplyffa for Niemann-Pick Disease Type C
Zevra Therapeutics wins FDA approval for Miplyffa, the first drug for Niemann-Pick disease type C (NPC), in combination with miglustat for neurological symptoms in patients aged two and older. NPC is a rare neurodegenerative disorder with a life expectancy of about 13 years. Zevra acquired the arimoclomol program from Denmark's Orphazyme, which initially failed to gain FDA approval in 2021. The FDA previously granted rare-pediatric-disease designation to arimoclomol, potentially qualifying Zevra for a priority-review voucher.
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