CELEBRATION, Fla. - Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a rare disease therapeutics company, announced today the FDA approval of MIPLYFFA (arimoclomol) for Niemann-Pick disease type C (NPC). MIPLYFFA, the first FDA-approved treatment for NPC, is indicated for use with miglustat to treat neurological symptoms in adults and children aged two and older.
The FDA's decision is based on a 12-month placebo-controlled trial and a 48-month open-label extension study. The trial showed that MIPLYFFA, combined with miglustat, halted disease progression at 12 months, compared to patients treated with miglustat alone.
NPC is a rare, progressive neurodegenerative disorder with approximately 900 known cases in the U.S. It leads to severe physical and cognitive impairments, and until this approval, there were no FDA-approved treatments available.
Zevra also received a rare pediatric disease priority review voucher alongside the drug's approval. The company launched AmplifyAssist™, a patient support program providing resources such as insurance education, co-pay assistance, and therapy management.
MIPLYFFA capsules, administered orally three times daily, will be available commercially in the U.S. within eight to twelve weeks. The dosages range from 47 mg to 124 mg based on body weight.
The approval marks a significant advancement for NPC patients and the medical community. Zevra will host a conference call and webcast on Monday to discuss the approval.
This article is based on a press release statement from Zevra Therapeutics.
In other recent news, Zevra Therapeutics has received significant FDA approval for its treatment of a rare genetic disorder, marking a major milestone for the company. This approval is the first of its kind for this particular condition, offering new hope for affected patients and families. In the financial sphere, Canaccord Genuity has revised its outlook on Zevra Therapeutics, reducing the price target but maintaining a buy rating following a recent capital raise. Despite this, the firm forecasts first commercial sales in the United States in 2025, with a potential launch in the European market in 2026.
Roth/MKM also maintains a buy rating on Zevra shares, citing promising trial results. These clinical trials include a Phase 2/3 trial of arimoclomol, a drug under investigation for Niemann-Pick disease type C. The firm's optimism is bolstered by the potential for near-term FDA approval of arimoclomol.
In their Q2 2024 earnings call, Zevra Therapeutics reported a net revenue of $4.4 million and a net loss of $19.9 million, alongside a successful public offering that raised approximately $64.5 million. The company also highlighted the continuation of its celiprolol Phase III trial and preparations for the potential launch of arimoclomol and OLPRUVA. These are the latest developments in Zevra Therapeutics' commitment to addressing unmet medical needs in the rare disease sector.
InvestingPro Insights
In light of Zevra Therapeutics' recent FDA approval for MIPLYFFA, investors and stakeholders are closely monitoring the company's financial health and stock performance. According to InvestingPro, Zevra is currently trading with a market capitalization of approximately $402 million. Despite the positive news, analysts have expressed concerns, anticipating a sales decline and expecting net income to drop in the current year. These projections may be tied to the high costs associated with launching a new drug and the niche market for rare disease treatments.
InvestingPro data shows that Zevra's Price / Book ratio stands at a high 12.38, which could suggest that the stock is currently valued above the company's net asset value. Additionally, Zevra has been operating with a significant operating income margin deficit of -315.77% over the last twelve months as of Q2 2024. This could be indicative of the company's heavy investment in research and development, as well as the costs related to bringing MIPLYFFA to market.
On a more positive note, Zevra has experienced a strong return over the last three months, with a 79.59% price total return, and analysts have revised their earnings upwards for the upcoming period. This optimism may reflect investor confidence in the long-term potential of MIPLYFFA and the company's pipeline. For investors interested in a deeper dive, there are additional InvestingPro Tips available, providing further insights into Zevra's financial metrics and stock performance.
For those seeking to make informed decisions, InvestingPro offers a comprehensive set of 12 additional tips on Zevra Therapeutics, accessible at https://www.investing.com/pro/ZVRA. These tips could provide valuable guidance in evaluating the company's prospects in the wake of this significant regulatory milestone.
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