The U.S. FDA named the first nine recipients of the recently unveiled commissioner’s national priority voucher (CNPV) program aimed at addressing unmet public health needs by shortening regulatory review times to as little as one to two months. For one of those firms, Disc Medicine Inc., which submitted an NDA for bitopertin for rare genetic disorder erythropoietic protoporphyria (EPP) in September, that means a potential approval before the end of 2025. Disc already had sought an accelerated pathway when it submitted the NDA, seeking clearance of the orally administered glycine transporter 1 inhibitor for use in patients, ages 12 and older, with EPP, including those with X-linked protoporphyria (XLP). But the CNPV option offers an even speedier route, as the news sent shares of Disc (NASDAQ:IRON) up 23% at midday. Also on the CNPV list is DB-OTO, a gene therapy for deafness from Regeneron Pharmaceuticals Inc., which anticipates a U.S. regulatory submission later this year, pending discussions with the FDA, and RMC-6236, a RAS blocker from Revolution Medicines Inc., in phase III testing in pancreatic cancer.

Immune and neurology biopharma deals drive value in Q3

Biopharma companies announced $73.38 billion in deals from 240 transactions during the third quarter (Q3) of 2025, bringing the year-to-date total to $212.44 billion, up from $149.87 billion in the same period of 2024. The total marks the highest deal value through Q3 ever recorded by BioWorld. Deal value has risen steadily throughout the year, climbing from $67.6 billion in Q1 to $71.47 billion in Q2 and $73.38 billion in Q3. The largest biopharma deal of Q3 2025 was Hengrui Pharmaceuticals Co. Ltd. out-licensing its phase I phosphodiesterase (PDE) 3 and 4 inhibitor, HRS-9821, and 11 additional development programs, to GSK plc in an agreement worth $500 million up front and up to $12 billion in potential milestones.

Mixed EMA news for Sanofi: Wayrilz yay, Rezurock nay

There was a curate’s egg for Sanofi SA from this month’s meeting of the EMA’s Committee for Medicinal Products for Human Use (CHMP), with a recommendation to approve one of the French pharma’s drugs – and the rejection of another. Getting a nod was Wayrilz (rilzabrutinib), a treatment for immune thrombocytopenia. But it was a thumbs down from CHMP for Rezurock (belumosudil) as a third line treatment for chronic graft vs. host disease.

Bring it all back home: Astellas opts to not license Taysha’s gene therapy

The full rights to Taysha Gene Therapies Inc.’s lead gene therapy candidate is coming back to the company. In 2022, Astellas Pharma Inc. invested $50 million in Taysha for 15% of the company and the exclusive option to license TSHA-102 for treating the rare disease Rett syndrome. That option has expired, according to Taysha. The gene therapy has the U.S. FDA’s breakthrough therapy designation and has been through a phase I/II study that Taysha said produced “compelling data.” Taysha’s stock (NASDAQ:TSHA) had dropped 10.6% at midday, with shares going for $4.54 each.

MFN drug pricing getting a foothold in the US

Caught between the rock and the hard place of most-favored nation (MFN) pricing and the threat of a hefty biopharma sector tariff, drug companies marketing in the U.S. are exploring their options. Several large firms already have committed millions and billions of dollars in investment in new or expanded U.S.-based manufacturing facilities to avoid tariffs on finished drugs. In addition to their U.S. manufacturing commitments, Astrazeneca plc and Pfizer Inc. have entered into agreements with the Trump administration to provide MFN pricing for their products through Medicaid and to offer a direct-to-consumer (DTC) cash program. Merck KGaA’s EMD Serono is the latest to jump on board, announcing with President Donald Trump on Oct. 16 DTC sales of its in vitro fertilization therapies at an 84% discount, and shielding itself from tariffs with a separate deal to invest in U.S.-based manufacturing and research.

Also in the news

Actor, Aculys, Advancell, Aicuris Alzinova, Anti-infective Cures, Aptose, Aqilion, C4, Candel, Conexeu, Delix, Enterome, Evoq, Formycon, Jacobio, Jacoray, Johnson & Johnson, Inmed, Ketabon, Klinge, Megalabs, Merck, Novartis, Peptilogics, Pharmther, Praxis Precision, Remegen, Repair, Sanofi, Spyre, Veradermics, Viatris, Vor