Vinay Prasad

Regenxbio announced it will submit its Duchenne muscular dystrophy gene therapy for accelerated approval, despite the FDA previously requesting an additional trial. The FDA appears to be reversing recent rejections of gene therapies, including a Hunter syndrome treatment reconsidered after key officials, Marty Makary and Vinay Prasad, left the agency.

The FDA is considering Moderna's first mRNA-based flu vaccine, mFlusiva, for people 50 and older. A study showed it reduced flu cases by 27% compared to a standard vaccine, but data on frail older adults and those with weak immune systems is lacking. A prior FDA official disputed the vaccine's comparison to a high-dose brand, but the application was later accepted.

The FDA advisory panel is evaluating Moderna's first mRNA-based flu vaccine, mFlusiva, for approval in adults 50 and older. The vaccine, which showed a 27% reduction in flu cases compared to a standard-dose vaccine in a 40,000-person study, faces scrutiny over its study design but has received a favorable FDA review with no safety concerns noted.

The FDA is considering Moderna's first mRNA-based flu vaccine, mFlusiva, for adults 50 and older. A study showed it reduced flu cases by 27% compared to a standard vaccine, but lacks data on very frail older adults. The decision follows a dispute with a former FDA official who questioned the study's design.

The FDA has reversed its opposition to UniQure's experimental Huntington’s disease treatment AMT-130, allowing the company to file for U.S. approval. UniQure plans to submit a marketing application in Q3 for accelerated approval, citing a three-year analysis of early-stage study data as acceptable to the FDA, despite previous objections from former agency officials.

The article claims the CDC promoted mask-wearing during COVID using low-quality research, citing a study by Vinay Prasad and Tracey Beth Hoeg. Their analysis found 77 CDC studies on masks published after 2019, with 30% lacking comparative groups and none being randomized trials, suggesting flawed evidence.

Dr. Tracy Beth Hoeg, an FDA official, has been removed from her role leading the agency's drug program and will be replaced by Dr. Mike Davis. Hoeg was involved in scrutinizing the safety of COVID-19 vaccines and antidepressants. Her departure is the latest in an ongoing shake-up at the FDA.

Dr. Marty Makary, FDA commissioner, is resigning after a rocky tenure that drew complaints from health industry executives and anti-abortion activists. He struggled to manage the FDA's bureaucracy and failed to win the confidence of its staff. His resignation comes after just over a year leading the agency.

The FDA has agreed to reconsider a treatment for a rare blood cancer called Ebvallo after initially rejecting it in January. The drug's manufacturers, Pierre Fabre Pharmaceuticals and Atara Biotherapeutics, had a meeting with FDA officials in late April. The agency will now review the already completed clinical trial.

The Pharmaceutical Research and Manufacturers of America is launching a seven-figure ad campaign targeting the 340B Drug Pricing Program, while the US Food and Drug Administration has seen an increase in rejection rates for cell and gene therapies. The program requires manufacturers to provide drugs at reduced prices to health providers caring for low-income patients. The ad campaign aims to manipulate the program's benefits.

The FDA's shifts are a top concern for the gene therapy field, even at a meeting in Rome. Recent approvals of rare disease treatments have been highlighted as reasons for optimism. The departure of Vinay Prasad, the FDA's vaccine chief, is also seen as potentially promising.

The FDA has appointed Katherine Szarama as the acting director of its vaccines and biologics unit, replacing Vinay Prasad. A Department of Health and Human Services official confirmed the change to The Hill.