Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors.
Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors, and joins the small but growing group of non-factor therapy options, with the advantage of a broad label and a convenient dosing regimen.
Acrivon Therapeutics Inc. provided updated phase II data for checkpoint kinase candidate ACR-368, highlighting promising response rates for biomarker-positive patients with endometrial cancer and raising the possibility of an accelerated pathway in the second-line setting.
Acrivon Therapeutics Inc. provided updated phase II data for checkpoint kinase candidate ACR-368, highlighting promising response rates for biomarker-positive patients with endometrial cancer and raising the possibility of an accelerated pathway in the second-line setting.
Ushering in a new class of antibiotics, the U.S. FDA approved GSK plc’s gepotidacin for use in uncomplicated urinary tract infections. Branded Blujepa, the oral triazaacenaphthylene bacterial topoisomerase inhibitor is indicated for treating female adults and adolescents, 12 and older.
Shares of Unity Biotechnology Inc. (NASDAQ:UBX) sank 28.8% to close at $1.30 March 24 as a top-line readout of its phase IIb Aspire study testing UBX-1325 head-to-head against aflibercept in diabetic macular edema (DME) fell short of statistical noninferiority on the primary analysis endpoint. But that’s not the full story, according to company executives, who have plans for moving the senolytic Bcl-xL inhibitor into late-stage studies.
Black Diamond Therapeutics Inc. is getting $70 million up front and could earn up to $710 million in milestone payments in a deal with Servier for phase I-stage BDTX-4933, a small-molecule candidate targeting both RAS mutations and RAF alternations for treating solid tumors, including non-small-cell lung cancer.
While data on functional endpoints are still to come, Avidity Biosciences Inc. executives said the firm is moving ahead with plans for a BLA filing by the end of 2025 for del-zota, an antibody-oligonucleotide conjugate, in Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44), based on positive top-line data that analysts say bode well for Avidity’s other late-stage programs targeting rare neuromuscular diseases.
The first treatment for macular telangiectasia type 2 is set to enter the market following U.S. FDA approval of NT-501 (revakinagene taroretcel) from Neurotech Pharmaceuticals Inc., a privately held company that has been quietly advancing its encapsulated cell therapy platform for more than two decades.
