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The rare paediatric disease PRV program aims to incentivise drug development for rare paediatric diseases. Credit: MagicVectorCreation via Getty Images. Zevra has sold its US Food and Drug Administration (FDA) priority review voucher (PRV) for $150m to an undisclosed party. The US-based company received the PRV when its Niemann-Pick disease type C drug Miplyffa (arimoclomol) won FDA approval in September 2024. Zevra plans to use the funds from the voucher sale to support the commercial launches of Miplyffa and the company’s urea cycle disorder drug Olpruva, which it inherited as part of the $91m acquisition of Acer Therapeutics in 2023. Niemann-Pick disease type C is a progressive, neurodegenerative lysosomal storage disorder caused by the body’s inability to transport cholesterol and other lipids within cells. This leads to lipid accumulation in tissues, including the brain, resulting in severe physical and cognitive decline. Miplyffa was the first therapy approved for Niemann-Pick disease type C in the US. It is indicated for use alongside Johnson & Johnson’s (J&J’s) Zavesca (miglustat). Zavesca has long been used as the primary, but off-label treatment for Niemann-Pick disease type C patients in the US. It is however approved for the disease in the UK and Germany. Miplyffa works by inducing the heat shock response to address Niemann-Pick disease type C protein misfolding. Miplyffa’s approval followed a turbulent regulatory journey . The FDA initially rejected the drug in 2021, issuing a complete response letter (CRL) to its then-owner Orphazyme due to insufficient clinical data. The agency was not convinced by the clinical results and requested additional data. Orphazyme later sold the drug’s rights to KemPharma, which rebranded it as Zevra. According to GlobalData’s Pharma Intelligence Center, Miplyffa is forecast to generate $273m in global sales by 2030. GlobalData is the parent company of Pharmaceutical Technology. The FDA’s PRV programme was introduced to incentivise innovation in areas with limited commercial incentives, such as rare paediatric diseases and neglected tropical diseases. In February 2024, Valneva sold its PRV for $103m after securing FDA approval for its chikungunya vaccine, Ixchiq, in November 2023. However, not all companies have benefited as expected. In 2023, bluebird bio faced financial and strategic difficulties after failing to secure a PRV for Lyfgenia, a gene therapy it anticipated would qualify for a voucher. The company had pre-arranged a $103m sale of the voucher to Novartis, only for the FDA to deny it, citing similarities to the active ingredient in another of bluebird’s gene therapies called Zynteglo, which had already earned bluebird a PRV on its approval in 2022.

SOPA Images/LightRocket via Getty Images The proposed acquisition by global investment firms Carlyle and SK Capital Partners could net shareholders $3 per share plus potential CVR dollars and provide bluebird bio with primary capital to expand the commercial reach of its gene therapies. After months of holding a tenuous grip on financial solvency, bluebird bio has found a solution. Friday, the gene therapy–focused biotech announced it will go private in an acquisition by global investment firms Carlyle and SK Capital Partners. The transaction—which will see bluebird shareholders receive $3.00 per share in cash and a contingent value right (CVR) equaling $6.84 per share if the company’s current product portfolio achieves $600 million in net sales in any 12-month period prior to Dec. 31, 2027—comes as bluebird was just about to run out of money . The deal values the company at roughly $30 million. David Meek, the former chief executive of Mirati Therapeutics, will take over as CEO of bluebird. He said in the statement that the new team is “committed to unlocking [the company’s] full potential for patients.” Investors did not react favorably to the deal, however, as bluebird’s stock lost 33% of its value as the markets opened Friday, falling to $4.65. It’s been a difficult stretch for bluebird, whose cash position has been in freefall the past few years—plummeting from a high of just over $1 billion in the first quarter of 2021 to $118.7 million in Q3 2024. Bluebird expected the latter amount to see the company into the first quarter of this year. Executives on a Q3 2024 earnings call in November told investors they expected to eke out cash break even in the second half of 2025—though it was unclear at the time exactly how the company would make it to that point. Bluebird had been counting on an increased number of patient starts for its gene therapy portfolio—including Lyfgenia, Zynteglo and Skysona. Executives in November said the company had doubled treatment starts for these products. “This trajectory continues to support the potential path to cash flow breakeven in the second half of 2025 as those starts convert to deliveries and infusions,” Tom Klima, chief commercial & operating officer, said in November. But William Blair analysts at the time expressed concern that bluebird would miss its patient start guidelines in the fourth quarter. Ultimately, after a “comprehensive review of bluebird’s strategic alternatives,” the company’s board of directors determined that the deal with Carlyle and SK Capital Partners “is the only viable solution to generate value for stockholders,” according to Friday’s news release. The strategic review included meeting with more than 70 potential investors in the past five months and making a third failed appeal for a priority review voucher, bluebird revealed Friday. “For more than a decade, bluebird has been at the forefront of gene therapy,” CEO Andrew Obenshain said in a statement. “However, as our financial challenges mounted, it became clear that securing the right strategic partner was critical to maximizing value for our stockholders and ensuring the long-term future of our therapies.” In December 2023, the FDA approved bluebird’s Lyfgenia for sickle cell disease. The greenlight was somewhat overshadowed at the time by the concurrent FDA approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy as the first-ever CRISPR-based gene editing therapy, for the same disease. Bluebird also markets Zynteglo, a one-time gene therapy for beta-thalassemia, and Skysona, a one-time treatment for cerebral adrenoleukodystrophy (CALD). As part of the deal, Carlyle and SK Capital Partners will provide bluebird with primary capital to expand the commercial reach of these therapies, according to Seeking Alpha . The acquisition is expected to close during the first half of 2025 and will transition bluebird to a privately held company.