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' SWOT analysis: rare disease focus drives stock potential
Zevra Therapeutics' MIPLYFFA approved by FDA for Niemann-Pick disease type C, projected to reach $230 million in U.S. sales by 2028. MIPLYFFA's efficacy contrasts with IntraBio's AQNEURSA, positioning Zevra as a key player in rare disease therapeutics. The company's $115 million in pro forma cash supports operations until 2027, with a diversified portfolio including OLPRUVA for urea cycle disorders and pipeline candidates KP1077 and Celiprolol. Challenges include competition from AQNEURSA and regulatory hurdles for pipeline products.
Biotech Stocks Facing FDA Decision In October 2024
September saw FDA approvals for two Niemann-Pick Disease Type C drugs, an OTC hearing aid software, and upcoming October decisions include Biofrontera's Ameluz dosage increase, Zealand Pharma's Dasiglucagon for hypoglycemia, Bristol Myers Squibb's Opdivo for lung cancer, Amgen's LUMAKRAS for colorectal cancer, Camurus' Oclaiz for acromegaly, Iterum Therapeutics' Oral Sulopenem for UTIs, PharmaTher's Ketamine for anesthesia, and Lexicon Pharma's Zynquista for diabetes.
Levacetylleucine Receives FDA Approval as Stand-Alone Therapy for Niemann-Pick Disease Type C
FDA approves levacetylleucine (Aqneursa) for treating neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients weighing at least 15 kg, based on a clinical trial showing significant neurological symptom improvement within 12 weeks.
Related Clinical Trials:
FDA Approves Stand-Alone Therapy Levacetylleucine for Niemann-Pick Disease Type C
FDA approves IntraBio's levacetylleucine (Aqneursa) for treating neurological manifestations in Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg, based on phase 3 trial data showing significant improvements in ataxia and neurological symptoms.
Related Clinical Trials:
FDA Approves Zevra Therapeutics' Arimoclomol as First Treatment for Niemann-Pick Disease Type C
FDA approves Zevra Therapeutics' arimoclomol (Miplyffa) for treating Niemann-Pick disease type C (NPC) in adults and children 2 years and older, marking the first FDA-approved drug for this condition. The approval is based on a 12-month trial showing arimoclomol, combined with miglustat, slowed NPC progression. The treatment comes with warnings for hypersensitivity reactions and is not recommended for pregnant women.
FDA approves levacetylleucine for the treatment of Niemann-Pick disease, type C
The FDA approved levacetylleucine (Aqneursa; IntraBio) for treating neurological symptoms of Niemann-Pick disease, type C (NPC) in adults and pediatric patients weighing at least 15 kg. This follows the recent approval of arimoclomol (Miplyffa; Zevra Therapeutics) for NPC, marking the second treatment approved within a week. The approval underscores the FDA's commitment to rare disease treatments.
FDA approves second Niemann-Pick disease type C treatment
The FDA approves Aqneursa, an oral treatment for neurological symptoms of Niemann-Pick disease type C in adults and children weighing at least 15 kg, marking the second treatment approved for the condition in a week.
FDA Approves Levacetylleucine for Treatment of Niemann-Pick Disease Type C
The FDA approved levacetylleucine (Aqneursa) for treating neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing 15 kg or more, making it the first stand-alone therapy for NPC in the U.S. The approval was based on the IB1001-301 trial, which demonstrated significant improvements in ataxia scores and quality of life measures. Levacetylleucine was well-tolerated with common adverse events being abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
Related Clinical Trials:
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