Brainstorm Cell Therapeutics Inc.’s Nurown got a thumbs down from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee Sept. 27, as the committee voted 1-17, with one abstention, that the data presented demonstrated substantial evidence of effectiveness for treatment of mild to moderate amyotrophic lateral sclerosis.
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.
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.
The U.S. FDA is going straight to final guidance with its “Considerations for the conduct of clinical trials of medical products during major disruptions due to disasters and public health emergencies [PHEs].”
The U.S. FDA is going straight to final guidance with its “Considerations for the conduct of clinical trials of medical products during major disruptions due to disasters and public health emergencies [PHEs].”
A Sept. 21 U.S. FDA advisory committee meeting will either be a “Hail Mary” or a last gasp of life for ITCA-650, a twice-yearly implantable exenatide-device combination product intended to improve glycemic control in adults with type 2 diabetes.
If the U.S. FDA has its way, biosimilars and interchangeable biosimilars would no longer be a difference with a distinction – at least when it comes to labeling. Instead of distinguishing between the two, the agency is recommending that the labeling for both follow-ons include a “biosimilarity statement.”
The U.S. FTC put brand drug companies on notice Sept. 14 when the commissioners unanimously voted, 3-0, to issue a policy statement recognizing that improperly listed patents in the FDA’s Orange Book “may constitute an unfair method of competition.”
Supporting their conclusions with data from the same phase III study, the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for extending the use of Oncopeptides AB’s Pepaxti (melflufen) to earlier lines of treating relapsed, refractory multiple myeloma even as the FDA dug in its heels about withdrawing the drug from the U.S. market.
