The U.S. FDA’s green lighting of Omeros Corp.’s Yartemlea (narsoplimab) makes it the first approved treatment for hematopoietic stem cell transplant-associated thrombotic microangiopathy, a life-threatening complication of hematopoietic stem cell transplantation. The BLA for narsoplimab, a fully human monoclonal antibody that inhibits the enzyme mannan-binding lectin-associated serine protease-2, had a Dec. 26 PDUFA date. Narsoplimab has breakthrough therapy and orphan drug designations in the U.S. for TA-TMA, and orphan drug designation by the EMA for HSCT. The Seattle-based company’s stock (NASDAQ:OMER) had jumped strongly at midday, with shares going for $15.17 each, an increase of 73% from the opening bell.

Sanofi buying Dynavax for $2.2B; tolebrutinib CRL arrives

Shares of Dynavax Technologies Corp. (NASDAQ:DVAX) were trading at $15.44, up $4.31, or 38% on word that Sanofi SA plans to acquire the company’s outstanding shares for $15.50 each in cash, reflecting a total equity value of about $2.2 billion. Sanofi, of Paris, brings aboard Emeryville, Calif.-based Dynavax’s adult hepatitis B vaccine Heplisav-B, approved by the U.S. FDA in November 2017. Separately, Sanofi said the agency issued a complete response letter (CRL) related to tolebrutinib, the brain-penetrant Bruton’s tyrosine kinase inhibitor to treat non-relapsing secondary progressive multiple sclerosis in adults. The CRL, Sanofi said, represents “a significant and meaningful change in direction from the feedback the agency previously provided” on the drug.

Hanx Bio sees stock drop 46% in $75M Hong Kong IPO debut

Hanx Biopharmaceuticals (Wuhan) Co., Ltd. raised HK$586 million (US$75.37 million) in its IPO on the Hong Kong Securities Exchange (HKEX:3378) on Dec. 23 to advance its clinical stage immuno-oncology pipeline. The global offering consisted of 18.321 million H shares priced at HK$32 per share. The Dec. 23 debut on the main board of the Hong Kong Exchange saw its stock drop 46.25% after starting trading at HK$28.90 per share and rising to a high of HK$29.48 and finally closing at HK$17.20. 

CMS crossing threshold of obesity drug coverage

Following through on its commitment to break through the obesity barrier, the U.S. CMS rolled out a voluntary test of a model designed to enable Medicare Part D plans and state Medicaid programs to cover GLP-1 drugs prescribed for weight management. Under the new Better Approaches to Lifestyle and Nutrition for Comprehensive Health (BALANCE) Model, CMS will negotiate directly with manufacturers, such as Eli Lilly and Co. and Novo Nordisk A/S, for lower net prices and standardized coverage terms for their GLP-1 drugs. The BALANCE Model ”builds upon our historic most-favored-nations drug pricing deals’ goal of democratizing access to weight-loss medication, which has been out of reach for so many in need,” CMS Administrator Mehmet Oz said in announcing the coverage program late yesterday.

Trump trade and pricing policies piled pressure on Europe in 2025

After an all-night negotiating session that concluded after 5 a.m. on Dec. 12, political agreement was finally reached on the long-awaited EU pharmaceutical legislation. The aim of the new rules is to improve patient access and increase the competitiveness of the sector, but for the industry, it was too little too late in terms of the incentives, and potentially damaging in the measures to improve access. 

Gene therapy genie back in the bottle?  

Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether. 

MASHup: Liver disease brings diverse players to table 

The year 2025 proved especially active with regard to deals that centered on the indication previously known as nonalcoholic steatohepatitis (NASH). NASH, a subtype of nonalcoholic fatty liver disease (NAFLD), was changed to MASH (metabolic dysfunction-associated steatohepatitis) to reduce stigma and, more specifically, to indicate the cause of the affliction, emphasizing the link to metabolic dysfunction such as diabetes and obesity. NAFLD, too, was changed to MASLD (metabolic dysfunction-associated steatotic liver disease). By any moniker, MASH is continuing to intrigue pharma and biotech alike.

Holiday notice

BioWorld's offices will be closed in observance of Christmas. No issues will be published Thursday, Dec. 25 or Friday, Dec. 26.

Also in the news

Agenus, Amneal, Avicanna, Bioeq, Biogen, Cassava, Dexcel, Dogwood, Dyne, Formycon, GSK, Hope Biosciences, Invivyd, Mannkind, Neurocrine, Novo Nordisk, Orexo, Regeneron, Reviva, Samsung Bioepis, Samsung Biologics, Sanofi, Seismic, Vaccinex, Windtree, Zai Lab, Zydus