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.
China’s National Medical Products Administration has given the thumbs up to Junshi Biosciences Co. Ltd.’s NDA for ongericimab, a recombinant humanized anti-PCSK9 monoclonal antibody, marking the third PCSK9 inhibitor to be cleared in China.
Cytotoxic T-lymphocyte antigen 4 (CTLA-4) is a cell surface molecule with immunosuppressive function involved in T-cell immune regulation and proliferation. Researchers from Shanghai Junshi Biosciences Co. Ltd. and collaborators described the preclinical evaluation of JS-007, a humanized monoclonal antibody targeting CTLA-4, in cancer models.
Shares of Scholar Rock Holding Corp. (NASDAQ:SRRK) soared $26.86, or 362%, to close Oct. 7 at $34.28, after the Cambridge, Mass.-based firm disclosed positive top-line data from the phase III Sapphire study testing apitegromab in patients with spinal muscular atrophy (SMA). Apitegromab, which Wainwright analyst Andres Maldonado said will “transform SMA” therapy, met the primary endpoint with statistically significant and clinically meaningful improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded.
Recordati SpA is shelling out $825 million up front for global rights to Enjaymo (sutimlimab), the only therapy approved for treating the rare disease cold agglutinin disease. In the deal with Sanofi SA, which won U.S. FDA approval of the antibody drug in 2022, the Italian pharma agreed to pay up to $250 million more should net sales reach certain thresholds.
The U.S. FDA’s approval of yet another indication for Dupixent (dupilumab), partnered between Regeneron Pharmaceuticals Inc. and Sanofi SA, could mean another $6.4 billion-plus in sales by the end of the decade. Regulators cleared the drug as an add-on maintenance treatment for chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype who are prone to flare-ups. Dupixent, the first-ever biologic for COPD, entered the market in March 2017.
As Eli Lilly and Co. launches its recently approved Ebglyss (lebrikizumab) in an atopic dermatitis market already dominated by established biologic Dupixent (dupilumab, Regeneron Pharmaceuticals Inc.), investors tuned into an Amgen Inc. investor call disclosing positive top-line phase III results for rocatinlimab, a monoclonal antibody that could potentially offer patients a new mechanism of action. While data from the Rocket Horizon study showed rocatinlimab hit all co-primary and secondary endpoints, the early findings fell below expectations in a highly competitive market.
Argenx SE’s ARGX-119 is a monoclonal antibody (MAb) targeting the muscle-specific kinase (MuSK) Frizzled (Fz)-like domain, and has entered early clinical development for the treatment of neuromuscular diseases.
Genentech’s newly approved multiple sclerosis (MS) injection takes about 10 minutes to administer, dramatically reducing the four to six hours required by its intravenous predecessor. The U.S. FDA approved the humanized monoclonal antibody Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) for relapsing MS and primary progressive MS on Sept. 13.
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