Zevra Therapeutics, Inc. (NASDAQ:ZVRA) is turning a quiet FDA approval into a broader comeback story. HC Wainwright initiated coverage on the rare disease drugmaker with a Buy rating and a $26 price target, pointing to strong early traction for its newly launched NPC treatment and a revamped commercial strategy that could drive long-term value.
Analyst Brandon Folkes argues the stock remains undervalued—not because of weak fundamentals, but because investors haven't yet caught up to the company's transformation.
Zevra has successfully launched Miplyffa (arimoclomol) in the U.S. for Niemann-Pick disease type C (NPC). The strong early performance shows that the Zevra team can exceed expectations in bringing drugs to market.
In September 2024, the U.S. Food And Drug Administration (FDA) approved Miplyffa (arimoclomol), an oral medication for type C (NPC).
Changes in the NPC1 and NPC2 genes cause Type C NPC. With these changes, the body lacks the proteins necessary to move and utilize cholesterol and other lipids within cells. Cholesterol and other lipids build up in the cells of the liver, spleen, or lungs.
HC Wainwright writes, “The company has successfully reworked itself over the past few years, and is now positioned to reward investors as a leading rare disease company with commercial and development expertise.”
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Folkes says Zevra’s stock is still undervalued, mainly because many investors don't fully understand the company's story or growth potential. He writes that this isn't due to any problems with how the company is performing or its future outlook.
“We view a very favorable risk-reward for investors at the current enterprise value,” Folkes wrote.
HC Wainwright states that Miplyffa has demonstrated strong long-term use in real-world settings through its expanded access programs, confirming steady revenue from early patient adoption.
This provides a reliable base for adding new growth drivers beyond Miplyffa in the U.S., with the most promising near-term opportunity likely coming from the EU market.
Zevra's stock is trading at 2.8 times its estimated 2026 enterprise value-to-sales (EV/Sales), factoring in cash from its priority review voucher sale.
In April, Zevra closed the sale of its Rare Pediatric Disease Priority Review Voucher for gross proceeds of $150 million.
Because Zevra focuses on rare diseases, analysts believe it could be valued at a higher EV/Sales multiple—between 4.0x and 8.0x. That means investors could see upside from the U.S. opportunity for its drug Miplyffa.
But Zevra's growth potential goes beyond the U.S. Miplyffa market. The company plans to file for approval in the EU later this year and could enter that market as early as 2026.
The biotech posted a net loss of 6 cents per share, beating analyst estimates of a 20-cent loss, a turnaround from a loss of 40 cents.
Revenue soared to $20.4 million—up nearly 500% year-over-year—driven primarily by sales of Miplyffa and reimbursements for arimoclomol.
Price Action: ZVRA stock is up 5.06% at $9.40 at the last check on Wednesday.
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