CELEBRATION, Fla. - Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a rare disease therapeutics company, announced today that MIPLYFFA™ (arimoclomol), the first U.S. Food and Drug Administration (FDA) approved treatment for Niemann-Pick disease type C (NPC), is now available. MIPLYFFA is prescribed in conjunction with miglustat to treat neurological symptoms in NPC patients aged two and older.
Early demand for MIPLYFFA has surpassed expectations, and the treatment is now being shipped to patients. Zevra's Chief Commercial Officer, Josh Schafer, expressed satisfaction with the rollout pace, aligning with the company's eight to 12-week post-approval distribution plan.
The company's AmplifyAssist™ program offers support to patients and caregivers, including assistance with insurance coverage, copay, and funding alternatives, as well as educational resources and therapy management. The program aims to minimize treatment barriers and ensure consistent medication access.
MIPLYFFA works by increasing transcription factors EB (TFEB) and E3 (TFE3) activation, leading to the upregulation of genes associated with lysosomal expression and regulation (CLEAR). The medication has also demonstrated a reduction in unesterified cholesterol in lysosomes of human NPC fibroblasts, although the clinical significance of this finding is not fully understood.
The pivotal phase 3 trial for MIPLYFFA showed a halt in disease progression over a one-year period as measured by the NPC Clinical Severity Scale. The treatment has received Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation from the FDA, and Orphan Medicinal Product designation from the European Medicines Agency (EMA) for NPC treatment.
NPC is a rare, progressive, neurodegenerative disorder characterized by the body's inability to transport cholesterol and other lipids within cells, leading to accumulation and affecting various cell types, including neurons. The disease can cause significant physical and cognitive impairments, often resulting in early mortality.
Zevra Therapeutics focuses on creating therapies for rare diseases with limited treatment options. The company's approach integrates scientific research, data, and patient needs. Zevra also operates expanded access programs, subject to its policy and jurisdictional regulations.
This news is based on a press release statement from Zevra Therapeutics.
In other recent news, Zevra Therapeutics announced its third-quarter financial results, highlighting the successful launch of MIPLYFFA, the first treatment for Niemann-Pick disease Type C, despite facing challenges with the launch of OLPRUVA for urea cycle disorders. The company reported a net loss for the quarter, with net revenue of $3.7 million, but emphasized a strong cash runway extending into 2027, supported by a $64.5 million net proceeds from a public offering. The firm's plans include the monetization of a rare pediatric disease priority review voucher associated with MIPLYFFA and moving KP1077 for idiopathic hypersomnia toward Phase III trials.
Zevra Therapeutics is also focusing on commercial excellence, pipeline innovation, talent development, and corporate foundation for 2024, with plans to expand into the EU market for MIPLYFFA. The company is monitoring the market for potential monetization of the Priority Review Voucher. Despite the challenges faced with OLPRUVA, Zevra Therapeutics remains optimistic about its growth prospects and is actively working to address the issues. These are among the recent developments in the company's activities.
InvestingPro Insights
As Zevra Therapeutics (NasdaqGS: ZVRA) launches its groundbreaking treatment MIPLYFFA™ for Niemann-Pick disease type C, investors may be interested in the company's financial performance and market position. According to InvestingPro data, Zevra's market capitalization stands at $487.32 million, reflecting the market's valuation of this rare disease therapeutics company.
The company's revenue growth is noteworthy, with a 47.85% increase over the last twelve months as of Q3 2024. This aligns with the positive reception of MIPLYFFA and the company's focus on addressing unmet needs in rare diseases. However, it's important to note that Zevra is not yet profitable, with an operating income margin of -354.22% for the same period.
InvestingPro Tips highlight that Zevra's stock price movements are quite volatile, which is not uncommon for biotechnology companies, especially those in the early stages of product commercialization. The stock has shown strong performance, with a 98.89% price total return over the past year, and is currently trading near its 52-week high.
Investors should be aware that analysts anticipate a sales decline in the current year, which may seem counterintuitive given the launch of MIPLYFFA. This projection could be due to various factors, including the typical ramp-up period for new drug launches and the small patient population for rare diseases like NPC.
For those interested in a deeper analysis, InvestingPro offers 14 additional tips for Zevra Therapeutics, providing a more comprehensive view of the company's financial health and market position.
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