After 12 years on the market under an accelerated approval, the application holder for Makena has failed to provide sufficient efficacy data to the FDA to support continued marketing. The FDA said “[T]oday, the U.S. Food and Drug Administration announced the final decision to withdraw approval of Makena.” The 26 page final decision (here) can […]
CMS realizes that some drugs approved through the FDA’s accelerated-approval process won’t prove to be safe and effective in final analysis. It wants to balance its payment for products that might not pan out in confirmatory trials by trying to make firms sprint, rather than crawl, to finalizing such studies. How? Well, we know that […]
It is always interesting to see how the Agency works when one action could trigger another action. In the Federal Register pre-publication page (here) this morning, the “Food and Drug Administration (FDA) [announced that it] is withdrawing approval of new drug application (NDA) 019832 for SULFAMYLON® (Mafenide Acetate, USP) Powder for 5% Topical Solution, held […]
As the pace of accelerated approvals has increased over the past few years, and as the number of withdrawals of accelerated approvals for some indications and products has also increased, the latest FDA move to seek removal of Makena, the premature birth preventative, for lack of efficacy could spell trouble for firms that cannot demonstrate […]
With the demise of the Alzheimer’s drug aducanumab after the FDA’s accelerated approval and CMS’s actions to raise Medicare premiums in anticipation of huge demands based on its proposed pricing, we may now face another similar situation and that is with a drug for amyotrophic lateral sclerosis (ALS). After a 7-2 favorable vote by the […]
There is no question that the Alzheimer’s drug Aduhelm has stirred up a lot of controversary. The action that CMS took related to reimbursement for the drug for Medicare patients and the initial yearly projected cost soured many stakeholders on the product, from prescribers to patients, as well as caregivers and healthcare providers, not to […]
Three FDA Advisory Committee members resigned over the FDA’s decision to grant accelerated approval to aducanumab for treating Alzheimer’s Disease. One member said, when he resigned, that “this is the worst decision the FDA has ever made.” I get it. When the entire committee voted against approval (full approval), the FDA kind of backdoored its […]
January was a gangbuster month for the FDA’s Center for Biologics Evaluation and Research (CBER) with regard to publication of gene therapy (GT) guidance documents! A total of six final guidelines and one draft guideline on GT products were issued. These included: Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug […]
Just look around, watch the television, read the newspaper, and what do you see? Seems like very few reasons to be thankful. School and workplace shootings, the political scene is as nasty as I ever remember in my 70 years on earth. Climate change is threatening the future of our children, and the environmental controls […]
Sue Sutter of the Pink Sheet wrote an article on September 23 entitled “Makena’s Accelerated Approval Will Hang In Balance at US FDA Panel Meeting In October” (here [subscription required]). The crux of the article deals with how FDA will handle accelerated approval when the required postmarketing confirmatory study fails to meet its endpoints. Ms. […]
