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Zevra Therapeutics

Zevra Therapeutics logo
🇺🇸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.

finance.yahoo.com
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Zevra's MIPLYFFA™ (arimoclomol) Receives Approval from U.S. Food and Drug

XOMA Royalty announces FDA approval of MIPLYFFA™, the first therapy for NPC, entitling XOMA to a mid-single digit royalty on sales and up to $52.6 million in milestones, making it the sixth commercial asset in their portfolio.

FDA approves Zevra Therapeutics' Miplyffa for Niemann-Pick disease

Zevra Therapeutics announces FDA approval for Miplyffa (arimoclomol) to treat Niemann-Pick disease type C (NPC) in patients aged two years and older, in conjunction with miglustat. The approval is based on comprehensive data from a 12-month trial demonstrating Miplyffa's safety and effectiveness, halting disease progression over 12 months. A 48-month open-label extension study further confirmed improved outcomes. Miplyffa is set to launch in the US market by autumn/winter 2024.
devdiscourse.com
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Regulatory Milestones and Health Advances: Key Developments in the Pharmaceutical Sector

EMA recommends Novo Nordisk's Wegovy for heart failure in obese individuals, expanding its use beyond weight loss. FDA approves Zevra Therapeutics' drug for a rare genetic disorder and AstraZeneca's nasal spray flu vaccine. FTC sues top U.S. pharmacy benefit managers over insulin price inflation. UNICEF stresses stigma combat in Burundi's mpox outbreak.
medicaldialogues.in
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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. Miplyffa targets neurological symptoms in NPC patients aged 2 and older, showing efficacy in slowing disease progression in a 12-month trial. Common side effects include upper respiratory tract infection, diarrhea, and decreased weight.
devdiscourse.com
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Key Developments in Health: Novo Nordisk's Wegovy, Zevra's Breakthrough, and More

Recent health news highlights include: EMA endorses Novo Nordisk's Wegovy for heart failure in obesity, FDA approves Zevra Therapeutics' treatment for a rare genetic disorder, FTC sues pharmacy benefit managers over insulin pricing, U.N. official suggests mpox in Burundi can be tackled within weeks, Sanofi's tolebrutinib shows promise in MS, Missouri healthcare worker contracts bird flu, FDA approves AstraZeneca's nasal spray flu vaccine, early studies show potential in rejuvenating older eggs for IVF and preventing newborn jaundice, U.S. appeals court revives lawsuit against Medicare drug price negotiation, and FDA approves Sanofi's Sarclisa for multiple myeloma treatment.
devdiscourse.com
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Breaking Health News: Major Drug Approvals and Controversies

Novo Nordisk's Wegovy endorsed for heart failure in obese patients. FDA approves Zevra Therapeutics' treatment for a rare genetic disorder. FTC sues pharmacy benefit managers over insulin prices. Sanofi's multiple sclerosis drug tolebrutinib shows 31% delay in disease progression. Advances in flu vaccines, IVF, and jaundice prevention.
devdiscourse.com
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Major Updates in Health: Drug Approvals, Legal Battles, and Health Innovations

Recent health news includes EMA endorsing Novo Nordisk's Wegovy for heart failure in obese individuals, FDA approving Zevra Therapeutics' treatment for a rare genetic disorder, and FTC suing major pharmacy benefit managers over insulin price manipulation. Other updates cover Sanofi's MS drug trial success, efforts to end mpox in Burundi, advancements in older egg IVF, and FDA approval of AstraZeneca's nasal spray flu vaccine.

US FDA approves Zevra's treatment for rare genetic disease

The FDA approved Zevra Therapeutics' Miplyffa, the first treatment for Neimann-Pick disease type C, expected to be available in 8-12 weeks. The drug, in combination with miglustat (Zavesca), treats neurological symptoms in adults and children 2 years and older. Analysts estimate the drug's annual net price between $500,000 and $600,000, with potential peak sales of $250 million in the U.S.
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