Despite Staff Opposition, FDA Official OKs Sarepta’s Duchenne Gene Therapy

Stat: Top FDA Official Peter Marks Overruled Staff To Approve Sarepta Gene Therapy
For a third time, Sarepta Therapeutics has convinced a top Food and Drug Administration official to overrule the prevailing view of their staff and approve a drug for Duchenne muscular dystrophy. (Mast and Herper, 6/20)

AP: Gene Therapy May Cure Rare Diseases. But Drugmakers Have Few Incentives, Leaving Families Desperate 
Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not available to him. ... Collectively, about 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual disorders affects perhaps a few in a million people or less. There’s little commercial incentive to develop or bring to market these one-time therapies to fix faulty genes or replace them with healthy ones. (Ungar, 6/21)

In other pharmaceutical news —

Reuters: US FDA Proposes To Remove Switching Study Requirement For Biosimilars 
The U.S. Food and Drug Administration has proposed that biosimilar drugs seeking agency's interchangeable designation will no longer need studies showing the impact of switching between them and the branded drug. (6/20)

Reuters: Industry Groups Back Drugmakers' Appeal In Zantac Cancer Lawsuits
A bid by GSK and other drugmakers to stop more than 70,000 lawsuits in Delaware over discontinued heartburn drug Zantac has received the backing of leading U.S. industry groups, including the United States Chamber of Commerce and Pharmaceutical Research and Manufacturers of America. (Pierson, 6/20)

CIDRAP: Study Suggests Cancer Patients Should Stay Current On COVID-19 Boosters
Yesterday in Nature Communications a study shows cancer that patients who are up to date on vaccines and have received COVID-19 boosters are more protected against death and serious complications than unvaccinated patients. Cancer patients were not included in key randomized clinical trials on vaccine efficacy (VE), the authors of the study write, but they are at increased risk of death and serious illness from COVID-19 infections. (Soucheray, 6/20)

CIDRAP: Rebyota Fecal Transplant Offers Durable Protection After Antibiotic Exposure, Study Finds
A microbiota-based live biotherapeutic for treating recurrent Clostridioides difficile infection (rCDI) remained effective after subsequent antibiotic exposure, researchers reported this week in Open Forum Infectious Diseases. In a post-hoc analysis of a phase 2 trial evaluating the safety, efficacy, and durability of Rebyota fecal microbiota transplantation for preventing rCDI, the researchers evaluated patients who received non-CDI antibiotics for up to 2 years after Rebyota administration. (Dall, 6/20)

Los Angeles Times: Antibiotics Wreak Havoc On The Gut. Can We Kill The Bad Bugs And Spare The Good Ones? 
Inside every human is a thriving zoo of bacteria, fungi, viruses and other microscopic organisms collectively known as the microbiome. Trillions of microbes live in the digestive tract alone, a menagerie estimated to contain more than 1,000 species. This ecosystem of tiny stuff affects our health in ways science is only beginning to understand, facilitating digestion, metabolism, the immune response and more. But when serious infection sets in, the most powerful antibiotics take a merciless approach, wiping out colonies of beneficial bacteria in the digestive tract and often prompting secondary health problems. (Purtill, 6/20)

Also —

Reuters: US, China Hold High-Level Talks On Anti-Narcotics Cooperation
The United States and China held high-level talks on anti-narcotics cooperation on Thursday, following a breakthrough in bilateral work this week that saw them jointly go after a major drug-linked money laundering operation. The U.S. and China restarted talks on counter-narcotics and law enforcement cooperation at the start of the year and China's public security department has lauded the case as a successful example of anti-drug cooperation between the two superpowers. (Slodkowski, 6/20)