Following an unexpected FDA complete response letter (CRL), Alnylam Pharmaceuticals Inc. said it will no longer pursue an expanded indication for Onpattro (patisiran) in the U.S. The RNAi therapeutic was approved in 2018 to treat polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis and seemed well on its way to snagging a second U.S. indication after the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in September that the drug’s benefits outweighed its risks as a treatment for cardiomyopathy of ATTR amyloidosis. The FDA disagreed with the committee, saying in the CRL that patisiran’s clinical meaningfulness had not been established in the proposed indication.

The US FDA is offering sponsors of certain drugs and biologics more agency access as part of a pilot program that will be launching in January 2024 with the mission of accelerating the development of new therapies for rare diseases.

After considering the evidence, the U.S. FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 14-6 Oct. 4 that the data from a single external-controlled trial and well-established preclinical animal models present sufficient evidence to demonstrate that US Worldmeds LLC’s DFMO (eflornithine) improves event-free survival in pediatric patients with high-risk neuroblastoma.

Takeda Pharmaceutical Co. Ltd. said it will work with U.S. regulators on the market withdrawal of Exkivity (mobocertinib), only two years after the oral tyrosine kinase inhibitor gained the FDA’s accelerated approval for use in locally advanced or metastatic non-small-cell lung cancer patients with EGFR exon 20 insertion mutations whose disease has progressed after chemotherapy.

For the second time in two weeks, the extent of regulatory flexibility will be at the heart of a U.S. FDA advisory committee meeting. The Oct. 4 meeting of the Oncologic Drugs Advisory Committee (ODAC) comes exactly a week after the Cellular, Tissue and Gene Therapies Advisory Committee voted overwhelmingly that the evidence presented for Brainstorm Cell Therapeutics Inc.’s amyotrophic lateral sclerosis drug, Nurown (debamestrocel), didn’t meet the agency’s flexibility standard. If the FDA’s briefing document for the ODAC meeting is anything to go by, the outcome for US Worldmeds LLC’s eflornithine, also known as DFMO, could be more positive, even though once again the agency is asking if the evidence from a single trial, along with supportive data, is sufficient.

Three years after Alnylam Pharmaceuticals Inc. gained U.S. FDA approval of the first treatment for rare disease primary hyperoxaluria type 1 (PH1), Novo Nordisk A/S solidified its own marketing clearance for RNAi therapy Rivfloza (nedosiran).

With Tofidence (tocilizumab-bavi), a monoclonal antibody from Biogen Inc., the U.S. FDA has approved the first biosimilar to the Roche Group AG’s Actemra (tocilizumab), a blockbuster with declining numbers. Tofidence was greenlighted Sept. 29 for treating rheumatoid arthritis  in adults, and for treating polyarticular and systemic juvenile idiopathic arthritis in those ages 2 and older. It’s also the first biosimilar approved to treat systemic juvenile idiopathic arthritis.