With the U.S. FDA approval of Attruby (acoramidis) for transthyretin amyloidosis with cardiomyopathy (ATTR-CM), Bridgebio Pharma Inc. is taking on an industry giant. The next-generation, oral, small-molecule stabilizer of transthyretin will take on Pfizer Inc.’s Vyndamax (tafamidis, Vyndaqel), a TTR stabilizer approved in 2019 for ATTR-CM but expected to lose exclusivity in late 2028. The approval was based on a phase III study that showed Attruby significantly reduced death and cardiovascular-related hospitalizations. The NDA, which had a Nov. 29 PDUFA date, was approved Nov. 22.
Ultromics Ltd. received clearance from the U.S. FDA for Echogo Amyloidosis, its artificial intelligence-enhanced software that helps with the early diagnosis of cardiac amyloidosis, enabling timely intervention that can significantly improve patients' quality of life and extend survival.
Zai Lab and Pfizer Inc. are teaming up in China to fight the deadly carbapenem-resistant Acinetobacter baumannii (CRAB) infection with novel antibacterial drug Xacduro (sulbactam-durlobactam). Through the collaboration, Zai Lab will leverage Pfizer’s commercial infrastructure in the anti-infective space to widen access to Xacduro in mainland China. Terms of the deal were not released.
Allovir Inc., which has struggled since late last December, will merge with privately held Kalaris Therapeutics Inc. to develop therapies for treating neovascular and exudative retinal diseases. Kalaris already has an anti-VEGF treatment in a phase I study with a data readout set for the third quarter of 2025. Once the deal closes, the combined company name will be Kalaris Therapeutics Inc. and shares will trade on Nasdaq as KLRS. The new company said it plans to drive development of TH-103 for treating neovascular age-related macular degeneration (nAMD), diabetic macular edema and retinal vein occlusion. The phase I study is for treatment of nAMD.
Gastric inhibitory polypeptide receptor (GIPR) antagonists potentially useful for the treatment of type 2 diabetes and obesity have been disclosed in a Pfizer Inc. patent.
Pfizer Inc. has divulged AMP-activated protein kinase (AMPK) activators reported to be useful for the treatment of autoimmune diseases as well as inflammatory and gastrointestinal disorders.
Camurus AB received a complete response letter (CRL) from the U.S. FDA for CAM-2029 (octreotide), its extended-release injection for acromegaly, due to “facility-related deficiencies” identified during a cGMP inspection of a third-party manufacturer. The Lund, Sweden-based company noted that the CRL did not indicate any concerns related to clinical efficacy or safety. Camurus will work with the FDA and the third-party manufacturer to address the concerns, said Fredrik Tiberg, president and CEO.
The U.S. FDA has approved the second hemophilia drug in nearly six months from Pfizer Inc. This one, Hympavzi (marstacimab), is for preventing or reducing bleeding in those age 12 and older with hemophilia A and B. Hympavzi heralds a couple of market boundary breakers: it’s the first and only anti-tissue factor pathway inhibitor approved in the U.S. for hemophilia A or B and the first hemophilia medicine approved in the U.S. to be administered using a pre-filled, auto-injector pen.
