A new analysis from Jefferies shows that drugs receiving breakthrough designations sail through the regulatory process more quickly, on top of frequently winning approval.
The vast majority of drugs granted breakthrough designation by the FDA—more than 94%—go on to earn priority reviews from the agency, according to a new report from Jefferies analysts.
That report follows a previous analysis from the firm, published in October, that showed 72% of drugs given breakthrough designation (BTD) between 2013 and 2022 went on to win approval, buffing the agency’s ability to identify strong drug candidates early on in the development pipeline.
“We have maintained a (+) outlook on the state of the FDA, despite ongoing leadership changes,” the analysts wrote Thursday morning, pointing to the resignation of Center for Drug Evaluation and Research Director (CDER) George Tidmarsh and Richard Pazdur’s subsequent appointment, as well as to Vinay Prasad, who stepped into his role as director of the Center for Biologics Evaluation and Research in May, left in July and returned in August.
Taking the two reports together, more than two thirds of drugs that win breakthrough designation also get priority review and then go on to win approval.
Between 2013 and 2022, in fact, few if any BTD-designated drugs went through the normal review period. In two years, 2014 and 2022, every single drug to secure breakthrough designation—a total of 48—went on to accelerated review.
The success of breakthrough designations, as well as the continued work of FDA advisory committees and the recent decision to release troves of complete response letters, led Jefferies to conclude that the regulatory environment at the FDA remains “largely intact.”
Priority reviews shorten a drug’s review time from a standard 10 months to six. A host of sponsors currently hold breakthrough designations that could lead to priority reviews, Jefferies said, including Axsome Therapeutics and its oral drug Auvelity for Azheimer’s disease–related agitation and Savara’s Molbreevi, an inhaled drug for autoimmune pulmonary alveolar proteinosis (aPAP), a rare autoimmune disorder affecting the lungs.
Interestingly, a number of drugs holding breakthrough designation were also recently awarded the Commissioner’s National Priority Review Voucher: Achieve Life Sciences’ cytisinicline for nicotine dependance, Revolution Medicines’ daraxonrasib for RAS-addicted cancers, Boehringer Ingelheim’s Hernexeos for non-small cell lung cancer and GSK’s Jemperli for endometrial cancer.
