Patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment now have a new oral treatment option, with the U.S. FDA’s approval of Novartis AG’s Rhapsido (remibrutinib). A Bruton’s tyrosine kinase (BTK) inhibitor, Rhapsido offers a new mechanism of action, shown to work by inhibiting the release of histamine and other proinflammatory mediators by targeting BTK, with convenient administration that offers an advantage over existing injectables. It marks the second approval of a new CSU drug this year, following an FDA nod for Regeneron Pharmaceuticals Inc.’s Dupixent (dupilumab), even as analysts look ahead to the KIT inhibitors advancing in late-stage development.

CRL blocks Menkes disease treatment CUTX-101

A U.S. FDA complete response letter (CRL) citing manufacturing concerns has stepped into the way of a rare genetic disorder treatment. Fortress Biotech Inc. and its subsidiary, Cyprium Therapeutics Inc., said the letter in response to the NDA for CUTX-101, an injectable formulation of copper histidinate, noted cGMP deficiencies at the facility where the Menkes disease treatment is manufactured. The company said the CRL didn’t mention any other problems and didn’t identify efficacy or safety data concerns. Sentynl Therapeutics Inc., which is handling development and commercialization duties, said it will ask for a meeting with the FDA to determine next steps. The NDA for treating pediatric patients had a Sept. 30 PDUFA date. Fortress’ stock (NASDAQ:FBIO) dropped by 32% at midday, with shares at $2.50 each.

Pharma pools structural data to boost power of AI in drug discovery

Pharma companies are collaborating to boost the power of artificial intelligence (AI) in drug discovery by allowing access to proprietary structural data to train a large language model. Each of the partners is contributing data from several thousand experimentally determined protein:ligand interactions, creating one of the most diverse datasets and the richest chemistry assembled to date for model training. The companies involved, Abbvie Inc., Johnson & Johnson Inc., Bristol Myers Squibb Co., Takeda Pharmaceutical Co. Ltd. and the Otsuka Pharmaceutical Co. Ltd. subsidiary Astex Pharmaceuticals Ltd., are members of AI Structural Biology, a network, which enables the collaboration to take place without moving or exposing data. 

Kyorin snags Hinge Bio’s multispecific antibody

Kyorin Pharmaceutical Co. Ltd. is in-licensing Hinge Bio Inc.’s multispecific antibody-based therapy HB-2198 in Japan for multiple autoimmune indications, starting with systemic lupus erythematosus (SLE). Tokyo-based Kyorin will make an up-front payment of $10 million to Hinge Bio, and Hinge is eligible to receive development- and sales-based milestone payments of up to $95 million, plus sales-based royalties for the SLE indication. Burlingame, Calif.-based Hinge Bio will lead global development activities, and Kyorin will fund development, regulatory, marketing and commercialization activities in Japan and contribute a portion of investment to global development.

Full-Life nabs $77M for radiopharmaceuticals, Belgium GMP site

Full-Life Technologies Ltd. announced Sept. 29 the completion of a $77 million financing, comprising $50 million in series C equity and $27 million in debt financing, which will be used to advance a portfolio of radiopharmaceuticals and radionuclide-drug conjugates (RDC) worldwide. The funds raised will notably help progress Full-Life’s global phase I clinical trial of its lead RDC asset, 225AC-FL-020. The latest financing will also support Full-Life’s construction of a manufacturing facility in Belgium, where the company acquired a 17,000-square-meter of land in the Walloon region in March 2023.

New FDA submissions stalled during funding lapse

Caught between the start of fiscal 2026 and a congressional standoff that shut down several parts of the U.S. government at midnight, the FDA cannot accept 2026 user fees, and thus any regulatory submissions associated with those fees, until Congress authorizes funding by enacting either a continuing resolution or a 2026 appropriations bill. In the duration, the agency will be limited to “activities necessary to address imminent threats to the safety of human life and activities funded by carryover user fee funds,” according to a notice on the FDA website. Those carryover fees will allow the agency to continue reviewing drug and device applications submitted prior to the new fiscal year.

Also in the news

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