If everything goes according to the current plan, the U.S. FDA would get the final report of a confirmatory trial for Acrotech Biopharma Inc.’s Folotyn (pralatrexate) and Beleodaq (belinostat) in 2030 – more than two decades after Folotyn received accelerated approval to treat relapsed or refractory peripheral T-cell lymphoma and 16 years after Beleodaq was granted accelerated approval for the same indication.
In a show of bipartisan solidarity, members of the U.S. Senate Special Committee on Aging voiced their support Oct. 26 for a new regulatory pathway to quicken access to new drugs for rare diseases that have no approved treatments.
As it continues its crackdown on accelerated approval, the FDA continues to stress that successfully completing confirmatory trials should be the top priority for sponsors of drugs that enter the U.S. market via accelerated approval.
The dark cloud of what the U.S. FDA called potential “systemic bias” rained on Amgen Inc.’s bid for full approval of Lumakras (sotorasib), a KRAS-G12C inhibitor that was granted accelerated approval in May 2021 for locally advanced or metastatic non-small-cell lung cancer after at least one systemic therapy.
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.
Since accelerated approvals first began to be granted in 1992, their pace for cancer indications has increased dramatically but a revolution in science has made it tough for the U.S. FDA to find its balance.
Sarepta Therapeutics Inc.’s balloting March 12 from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (OTAT) in favor of gene transfer therapy SRP-9001 (delandistrogene moxeparvovec) in Duchenne muscular dystrophy (DMD) had Wall Street mulling the odds for others in the space.
Despite congressional concerns about accelerated approval, the U.S. FDA’s use of the pathway is not slowing down. If anything, it’s picked up pace since Congress gave the agency stronger authority last year to monitor drugs approved based on a surrogate endpoint and to ensure that confirmatory trials are progressing in a timely way.
U.S. Centers for Medicare & Medicaid Services (CMS) Administrator Chiquita Brooks-LaSure made her first appearance April 26 before the House Energy and Commerce’s Subcommittee on Health, ostensibly to discuss legislative solutions to increase transparency and competition in health care. But member after member, regardless of political party, demanded answers about why CMS continues to severely restrict access to Eisai Co. Ltd.’s Alzheimer’s drug, Leqembi (lecanemab), especially since another government agency is covering it for all veterans that meet the labeling requirements.
The long-running Makena saga came to a close April 6 with the U.S. FDA announcing its decision to immediately withdraw approval of the drug and its generics – the only drugs indicated in the U.S. to reduce the risk of preterm birth.
