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.
FDA Approves Levacetylleucine for Niemann-Pick Disease Type C
The FDA approved IntraBio’s levacetylleucine (Aqneursa) for treating neurological symptoms of Niemann-Pick disease type C in adults and pediatric patients ≥15 kg. This marks the second NPC treatment approval in a week, following Zevra Therapeutics’ arimoclomol. Levacetylleucine is the only stand-alone therapy approved for NPC, based on a pivotal clinical trial showing significant neurological improvement within 12 weeks. Common adverse reactions include abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
IntraBio Wins FDA Nod for Ultra-Rare Neurodegenerative Disease on Heels of Competitor's Approval
FDA approves IntraBio’s Aqneursa for Niemann-Pick disease type C, showing improved neurological symptoms in a 12-week study. Common side effects include vomiting and abdominal pain. Aqneursa follows Zevra Therapeutics’ Miplyffa approval, priced $40,000-$106,000/month, launching in 8-12 weeks.
FDA approves Zevra's Miplyffa as first drug for Niemann-Pick disease
The FDA approved Zevra Therapeutics’ Miplyffa (arimoclomol) for Niemann-Pick disease type C (NPC), the first drug for this ultra-rare disorder. Miplyffa is approved for use with miglustat to treat neurological symptoms in adults and children aged two and older. The drug aims to increase molecular chaperone proteins to aid lipid metabolism. A trial showed the Miplyffa/miglustat combination slowed disease progression.
FDA Approves IntraBio's Aqneursa For Niemann-Pick Disease, Type C
The FDA approved IntraBio's Aqneursa for treating neurological symptoms of Niemann-Pick disease type C in adults and pediatric patients weighing at least 15 kg. This follows the approval of Zevra Therapeutics' Miplyffa. Aqneursa's efficacy was tested in a 24-week study, showing improved functional SARA scores. Common side effects include abdominal pain and difficulty swallowing.
FDA OKs First Treatment for Rare, Deadly Genetic Disorder
The FDA approved the first treatment for Niemann-Pick disease type C, a rare genetic disorder affecting less than 1,000 people in the U.S. The drug, arimoclomol marketed as Miplyffa, is approved for ages 2 and up and works in combination with miglustat to slow disease progression. Common side effects include upper respiratory tract infection, diarrhea, and decreased weight.
Miplyffa bags FDA approval for ultra-rare disease
Miplyffa, an oral treatment for NPC, boosts TFEB and TFE3 proteins to regulate lipids, approved in combination with miglustat. Zevra plans to launch it at $85,000/month, with AmplifyAssist support. Arimoclomol, previously rejected by FDA, was acquired by Zevra. IntraBio and Azafaros also have NPC treatments in development.
Zevra Therapeutics to Present Top-Line Data from the Phase 2 Clinical Trial of KP1077
Zevra Therapeutics presents KP1077 Phase 2 trial data at Sleep Europe 2024, showing safety and efficacy for idiopathic hypersomnia.
FDA approves Miplyffa for Niemann-Pick disease type C treatment
Zevra Therapeutics secures FDA approval for Miplyffa, the first drug for Niemann-Pick disease type C in the US, to be used with miglustat for neurological symptoms in patients aged two and older.
FDA clears first drug for Niemann-Pick disease
Zevra Therapeutics' Miplyffa, in combination with miglustat, is the first FDA-approved treatment for neurological symptoms in NPC patients aged 2 and older. The approval follows a phase 2/3 trial showing slowed disease progression and a favourable safety profile. Miplyffa will be available in the US within 8-12 weeks.
Zevra's MIPLYFFA™ (arimoclomol) Receives Approval from U.S. Food and Drug
XOMA Royalty announces FDA approval of MIPLYFFA™, the first therapy for NPC, expanding its portfolio to six commercial assets. MIPLYFFA™, in combination with miglustat, treats NPC in adults and children aged 2 and older.
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