On Monday, Zevra Therapeutics, Inc. (NASDAQ:ZVRA) saw its stock price target increased by H.C. Wainwright to $20.00, up from $18.00, while the firm maintained a Buy rating on the stock. The adjustment follows the recent FDA approval of Zevra's drug arimoclomol, now branded as Miplyffa, for the treatment of Niemann-Pick disease type C (NPC) on September 20, a day before the anticipated August 21 Prescription Drug User Fee Act (PDUFA) date.
The approval came after an 11-5 Advisory Committee vote in favor of the drug, marking arimoclomol as the first therapy ever approved for NPC. Analysts anticipate that this milestone will be met with excitement from the NPC community, which includes approximately 70 U.S. patients currently accessing arimoclomol through the Expanded Access Program (EAP), and the wider group of around 350 patients receiving active treatment in the United States.
The approval is seen as a transformative event for Zevra, with projections for peak arimoclomol sales reaching approximately $250 million. This estimate is significant against Zevra's current revenue base of less than $20 million and does not yet account for the potential market outside the U.S., where about 250 patients are in EAPs.
Moreover, the approval is expected to grant Zevra a rare pediatric disease priority review voucher (PRV), valued at a minimum of $100 million, with recent sales nearing $160 million. This value may rise due to the PRV program's impending sunset at the end of September.
The firm's projections for arimoclomol's success are above the consensus, citing underestimation by the Street of the potential conversion of the EAP patient base to paid therapy and the number of patients "warehoused" by physicians waiting for access to the drug. Zevra's existing commercial infrastructure for orphan diseases is anticipated to facilitate a smooth launch, with the product expected to be commercially available by December.
In other recent news, Zevra Therapeutics has made significant strides in the biopharmaceutical industry. The company has received FDA approval for its drug MIPLYFFA, marking the first approved treatment for Niemann-Pick disease type C (NPC), a rare neurodegenerative disorder. The approval is based on a 12-month placebo-controlled trial and a 48-month open-label extension study, which demonstrated halted disease progression when used in conjunction with miglustat.
In financial developments, Canaccord Genuity revised its outlook on Zevra, reducing the price target but maintaining a buy rating. The firm forecasts the first commercial sales in the US in 2025, with a potential European market launch in 2026. Roth/MKM also maintains a buy rating on Zevra, citing promising trial results.
In their Q2 2024 earnings call, Zevra 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. These are the latest developments in Zevra Therapeutics' commitment to addressing unmet medical needs in the rare disease sector.
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