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.
The U.S. development path for rare disease treatments is strewn with numerous challenges, not least of which are the regulatory hurdles. For companies developing promising candidates to treat ultra-rare diseases and the patients who are running out of time, the regulatory obstacles in the U.S. may seem almost insurmountable. And new concerns about drug development in general could make those barriers even higher.
Despite pleas from patient advocacy groups and bipartisan pressure from the U.S. Congress, the Centers for Medicare & Medicaid Services (CMS) isn’t budging on its national coverage determination for amyloid-targeting monoclonal antibodies approved to treat Alzheimer’s.
Now that the U.S. FDA has granted accelerated approval for Biogen Inc./Eisai Co. Ltd.’s early Alzheimer’s drug, Leqembi (lecanemab), the Centers for Medicare & Medicaid Services (CMS) is being pressured to rethink its coverage of amyloid-targeting monoclonal antibodies.
Eli Lilly and Co.’s complete response letter (CRL) from the U.S. FDA relating to accelerated approval of Alzheimer’s disease (AD) candidate donanemab set off a round of speculation regarding not only what the move might mean for the pharma giant but also for others in the embattled therapeutic space and beyond. The answer, if you believe analysts: not much.
In the wake of ongoing criticism over the U.S. FDA’s 2021 accelerated approval of Biogen Inc.’s Alzheimer’s drug, Aduhelm, the percentage of novel drugs receiving accelerated approval last year was the lowest it’s been since 2018.
Shares of Oncopeptides AB dropped 35% Dec. 7 on the U.S. FDA’s request to withdraw marketing authorization of Pepaxto (melflufen), a drug that had gained accelerated approval in early 2021 for use in relapsed/refractory multiple myeloma. The move followed a negative advisory committee vote in September 2022 and is based on the outcome of the confirmatory phase III Ocean study.
In a bit of déjà vu, the U.S. FDA’s Obstetrics, Reproductive and Urologic Drugs Advisory Committee once again voted that Makena (hydroxyprogesterone caproate) should be withdrawn from the U.S. market while a second confirmatory trial is designed and conducted. But this time around, the committee’s 14-1 vote was much more decisive than its 9-7 vote in 2019.
U.S. lawmakers concerned about unconfirmed clinical benefit of drugs with accelerated approval got more fodder for their arguments in a new report from the Department of Health and Human Services Office of Inspector General (OIG). According to that report, which was released Sept. 29, Medicare and Medicaid have spent more than $18 billion over the past few years covering 18 drugs granted accelerated approval that haven’t completed their confirmatory trials even though the trial completion dates have passed.
Although it wasn’t a shutout, Spectrum Pharmaceuticals Inc. didn’t get the support it needed from a U.S. FDA advisory committee Sept. 22 for its non-small-cell lung cancer candidate, poziotinib, which it has proposed marketing as Pozenveo.
