Five FDA decisions to watch before the year ends

The Food and Drug Administration heads into the final quarter of the year with a crowded docket and a review staff that is, by the agency's own count, about 9% smaller than it was eighteen months ago. So far the deadlines have held. Of the 41 goal dates that fell between January and September, the agency met 38.

The fourth quarter will test that record. Five decisions stand out, either because of what they mean for the companies involved or because of the precedent they could set for the drugs lined up behind them.

1. Halvern's bispecific for small cell lung cancer

Goal date: Oct. 28. Halvern Therapeutics is asking for accelerated approval of tovaritamab on the strength of a single-arm study in 212 patients whose disease had returned after chemotherapy. Tumors shrank in 38% of them, and responses lasted a median of 7.4 months.

Reviewers have twice asked for additional safety analyses, both related to a neurological side effect seen in roughly one in eight participants. The company says the events were mostly mild and reversible. An advisory committee was not convened.

A clean label would make this the reference product for a class that at least six other developers are chasing.

2. Nordlicht's twice-yearly cholesterol injection

Goal date: Nov. 14. Nordlicht Pharma's lerosiran lowered LDL cholesterol by 52% in its main trial, in line with the two long-acting injectables already on the market. The question for the agency is narrower than efficacy: whether a cardiovascular outcomes study that will not finish until 2028 is needed before the drug can carry a broad label.

3. A second attempt for Corvane Bio

Goal date: Nov. 30. The FDA rejected Corvane's enzyme replacement therapy for a rare metabolic disorder last year, citing problems at a contract manufacturing site rather than the clinical data. The plant has since been reinspected. A second rejection would leave the company with less than a year of cash.

4. Quillon's oral option in ulcerative colitis

Goal date: Dec. 9. Quillon Biosciences is seeking approval of a once-daily pill in a market dominated by injectable antibodies. Analysts expect an approval; the open question is whether the label carries the boxed warning attached to older drugs in the same class.

5. An expanded label for a gene therapy

Goal date: Dec. 19. Tessaly Genomics wants to extend its hemophilia B gene therapy to adolescents. The application rests on 31 patients followed for two years, and will be an early signal of how the agency's cell and gene therapy office weighs small datasets under its new leadership.

Goal dates are targets rather than guarantees, and the agency can act early or extend a review by three months if a sponsor submits substantial new information.