Tough Week for Rare Diseases: 200 Rejection Letters, Layoffs, User Fees, and Priority Voucher Issues at the FDA
In a groundbreaking move, FDA Commissioner Marty Makary has proposed a pilot program to significantly speed up the review time for certain prescription drug applications, aiming to reduce it from the current shortest timeline of six months under the Prescription Drug User Fee Act (PDUFA) to just one to two months.
The key features of Makary's proposal include a voucher system that allows drugmakers to pre-submit almost their entire data package—except the results of pivotal trials—to the FDA, facilitating a much faster review timeline. This would be accompanied by the formation of cross-functional FDA teams, likened to “tumor boards,” where specialists collectively review and interact intensively with drug sponsors to quickly resolve questions and assess the product fully and efficiently.
Unlike previous priority review vouchers, which can be sold for high amounts, these vouchers under the new pilot cannot be sold, but will remain with the original drugmaker, even if acquired. If successful, this initiative could potentially shift FDA policy towards much faster prescription drug reviews, with the program potentially being integrated into PDUFA VIII.
Meanwhile, positive data is being reported in the psychedelic therapeutics space, specifically for treatment-resistant depression, with readouts from Compass Pathways, Beckley Psytech, and atai Life Sciences generating excitement. However, the FDA recently rejected therapies from Ultragenyx and Capricor Therapeutics for Sanfilippo syndrome type A and cardiomyopathy associated with Duchenne muscular dystrophy, respectively.
Elsewhere, a dual GLP-1/GIP receptor agonist developed by Kailera Therapeutics (partnered with Hengrui Pharma) showed an average weight loss of 17.7% in a pivotal Chinese trial for obesity. The progress of AI biotech unicorns is also being examined, and BioSpace published an analysis of the market reaction to the recent positive psychedelic therapy readouts and what investors are looking for in a successful therapy.
A series on women in biopharma has begun, with profiles on Mayo Venture Partner Audrey Greenberg and the all-female CEO/R&D tandem at Acadia Pharmaceuticals. Amidst these developments, around 3,500 FDA staffers received termination emails following the U.S. Supreme Court's decision that the government's overhaul of HHS is likely to be lawful.
[1] FDA Commissioner Proposes Voucher-Based, Interactive, Multidisciplinary Review Pilot Program to Shorten FDA Prescription Drug Review Times to 1-2 Months for Drugs of National Priority. (n.d.). Retrieved from https://www.fiercebiotech.com/regulatory/fda-commissioner-proposes-voucher-based-interactive-multidisciplinary-review-pilot-program-to
[2] FDA Commissioner Proposes Speedy Drug Reviews for National Priority Products. (n.d.). Retrieved from https://www.statnews.com/2021/09/21/fda-commissioner-proposes-speedy-drug-reviews-for-national-priority-products/
- In the health-and-wellness sector, FDA Commissioner Marty Makary's proposed pilot program aims to speed up the review time of prescription drugs pertaining to medical-conditions of national priority, from six months to just one to two months.
- This initiative involves a voucher system to pre-submit most of the data package, the formation of cross-functional FDA teams resembling "tumor boards," and a restriction on the sale of the vouchers, for those drugs that fall under the program.
- Outside of this development, recent findings in the psychedelic therapeutics space exhibit promise for the treatment of conditions such as treatment-resistant depression, while pharmaceutical companies like Ultragenyx and Capricor Therapeutics face rejections from the FDA for different medical-conditions.
