Acer Therapeutics, Inc.

The $150 million price tag for Zevra’s PRV is slightly above the $100 million mark that many vouchers have traded at in the past, though it’s in line with several other recent transactions. For Zevra Therapeutics, last year’s approval of the rare lysosomal storage disorder drug Miplyffa appears to be the gift that keeps on giving.After receiving a rare pediatric disease priority review voucher (PRV) in tandem with Miplyffa’s FDA green light in September, Zevra has locked in a deal to sell the PRV for $150 million.Zevra did not disclose the identity of the purchaser. The transaction itself is expected to occur within 30 to 45 days, the company said Thursday.The cash from the sale will be used to fuel R&D, plus the commercial rollouts of Miplyffa and the urea cycle disorder (UCD) medicine Olpruva, which Zevra picked up in its $91 million buyout of Acer Therapeutics in 2023, Zevra’s CFO, LaDuane Clifton, said in a statement.Miplyffa’s FDA approval last fall made it the first drug specifically cleared in the U.S. to treat Niemann-Pick disease type C (NPC), a rare lysosomal storage disorder that prevents the body from moving and using cholesterol and other lipids in cells. The disease can take a significant toll on patients’ speech, cognition, swallowing, movement and fine motor skills.Miplyffa is taken orally three times a day in conjunction with the enzyme inhibitor miglustat. Miglustat, which is sold by Johnson & Johnson as Zavesca, has long been used as the primary, albeit off-label, treatment for NPC patients.Zevra’s first-comer advantage was quickly challenged by Austin, Texas-based IntraBio, which scored an FDA thumbs up for its own NPC drug Aqneursa just one week after Miplyffa’s approval. Still, Zevra hasn’t been sweating the competition so far.“It’s really just hard to believe that you have two approvals within one week after literally decades of nothing available—so we’re thrilled for patients and the NPC community,” Zevra’s chief commercial officer, Josh Schafer, said in an interview last year.Miplyffa’s ability to halt the progression of disease, rather than just treating symptoms, will likely position the drug as the “foundation and the cornerstone” of NPC treatment, Schafer said, adding that he believes additional therapies will be prescribed based on patients’ individual symptoms and needs.The $150 million price tag for Zevra’s PRV is slightly above the $100 million mark that many vouchers have traded at in the past, though it’s in line with several other recent transactions.In late November, PTC Therapeutics said it had lined up a $150 million sale of the rare pediatric disease PRV it picked up through the approval of its gene therapy for the ultra-rare disease aromatic L-amino acid decarboxylase (AADC) deficiency. Earlier that same month, Acadia Pharmaceuticals also bagged a $150 million PRV sale tied to the approval of its Rett syndrome drug Daybue. In Acadia’s case, the company was only able to keep $100 million of the proceeds, with the rest going to the drug's licensor Neuren Pharmaceuticals.The recent voucher sales by Zevra, PTC and Acadia are likely to be among the last in the childhood rare disease space.Back in September, the FDA said it would sunset its rare pediatric disease PRV program beginning Dec. 20, 2024. 

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.

TAIPEI, Feb. 20, 2025 /PRNewswire/ -- NaviFUS Corp. is revolutionizing brain disorder treatments through ultrasound-based solutions, leveraging Taiwan's advanced chip technology. By integrating ICT capabilities into high-end medical devices, NaviFUS enhances signal processing beyond traditional medical devices, positioning itself as a global leader in focused ultrasound treatments. Innovative Technology for Brain Disease Treatment The NaviFUS system uses transcranial focused ultrasound and therapeutic guidance tracking for drug delivery and neuromodulation, offering new hope for patients with brain cancer and drug-resistant epilepsy. Strong Industry Backing and Global Collaborations NaviFUS has secured support from top venture capitalists, including Andy T.C. Chiu (Top Taiwan Venture Capital), Stan Shih (Founder of Acer and Chairman of Himalaya Venture Capital), and Max Wu (Spring Foundation of NCTU). It collaborates with YoungTek Electronics Corp., Genovate Biotechnology Co., Ltd., and the Focused Ultrasound Foundation, led by Dr. Neal Kassell. These partnerships accelerate development and clinical applications. Breakthrough Clinical Achievements NaviFUS completed a Phase II trial in Taiwan with Avastin for recurrent glioblastoma (rGBM), demonstrating: 66.7% progression-free survival at six months (vs. historical 42.6%) Median progression-free survival of 8.9 months (vs. historical 4.2 months) The strong safety and efficacy results have led to a pivotal clinical trial under Taiwan's regulatory agency. In drug-resistant epilepsy, clinical studies show the NaviFUS system significantly reduces seizure frequency and duration, offering a promising treatment for patients unresponsive to medication. Phase II trials are ongoing in Taiwan, with pilot studies in Australia and the U.S. Global Expansion and Strategic Partnerships NaviFUS collaborates with Brainlab, a global leader in surgical navigation, to enhance treatment precision. Brainlab's network of 6,300 hospitals in 120 countries strengthens NaviFUS' global reach. Taiwan's Leadership in Smart Healthcare Chairman Jen Chen emphasized Taiwan's commitment to biomedical advancements, as highlighted by Acer founder Stan Shih during NaviFUS' pre-listing presentation. As Taiwan's only high-end medical device company integrating advanced chip technology, NaviFUS is shaping the future of non-invasive brain treatments. About NaviFUS Corp. NaviFUS develops focused ultrasound (FUS) technology for blood-brain barrier opening, drug delivery, and neuromodulation, improving treatment outcomes for brain diseases like glioblastoma and epilepsy. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED