As biosimilar development expands beyond monoclonal antibodies to more complex biologics, the flexibility built into regulatory paths across the world will become more essential. Rather than making wholesale changes to those pathways, regulators need to follow the science in exercising the flexibility they already have, Leah Christl, executive director of global biosimilars regulatory affairs and R&D policy at Amgen Inc., told BioWorld. In doing so, “we do need to look forward to what might be coming down the pipeline,” in addition to looking backwards at what types of biosimilars have already been approved, she said.

How flexible should the U.S. FDA evidentiary standards be for a therapy addressing a significant unmet need in a disease such as amyotrophic lateral sclerosis (ALS)? That’s the question the agency’s Cellular, Tissue and Gene Therapies Advisory Committee will ponder Sept. 27 as it looks at the data for Brainstorm Cell Therapuetics Inc.’s Nurown (debamestrocel), a mesenchymal stromal cell therapy targeting ALS. Nurown is going into the adcom with a bit of a checkered history that includes a refuse-to-file letter and a single phase III trial that failed to demonstrate efficacy for the primary endpoint and all key secondary efficacy endpoints, according to the FDA briefing document.

Approvals from the U.S. FDA are up more than 16% from last year, with 113 drugs approved in the first eight months of 2023. New approvals include two different treatments for relapsed or refractory multiple myeloma, two treatments for ultra-rare diseases and a biosimilar to Biogen Inc.’s Tysabri. In August the FDA greenlighted 13 drugs, approving three NDAs, six BLAs, two supplemental NDAs and two supplemental BLAs.

The U.K.’s Competition Appeal Tribunal has rejected appeals by drugmakers, including Actavis plc and Allergan Inc., against a ruling by regulators that they hiked the price of a life-saving adrenal insufficiency drug excessively for almost a decade, saddling the companies with fines amounting to nearly £130 million (US$159 million).

Safety concerns overrode benefit when the U.S. FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted unanimously, 19-0, Sept. 21 that the potential risks of Intarcia Therapeutics’ ITCA-650 outweighed the compliance and A1C-lowering benefits the twice-yearly implantable exenatide-device combination product could provide for adults with type 2 diabetes.