After saying it would refuse to halt shipments of its Duchenne gene therapy Elevidys, Sarepta reversed course this week and opted to voluntarily stop shipping it. The about-face came after the FDA said it would use its “full regulatory authority” to enforce the shipping pause. At least one large children’s hospital had already stopped treating patients by the time Sarepta announced its reversal.

Later in the week, a senior FDA official said Sarepta would have to conduct new studies to get Elevidys back on the market, Endpoints News exclusively reported. The official, who is directly familiar with the discussions, spoke on condition of anonymity. Dosing or manufacturing changes might be made, testing it on 10 or 12 boys to show whether liver function tests were significantly lower. 

The developments are a continuation of the quandary Sarepta finds itself in after three individuals died after taking their treatments. Two were young boys who had received Elevidys, while the third was an adult man participating in Sarepta’s clinical trial for limb-girdle muscular dystrophy. Both gene therapies used the same viral vector, and all individuals experienced the same liver side effects. Read more about how Sarepta continues to evolve in this report from Jared Whitlock and Lei Lei Wu. 

💉 Some of HHS Secretary Robert F. Kennedy Jr.’s biggest fans in the Make America Healthy Again movement are beginning to express doubt, specifically over whether Kennedy intends to get rid of the mRNA vaccines used during the Covid-19 pandemic. In the six months since President Donald Trump’s inauguration, three mRNA-based vaccines were approved. Shannon Joy, a self-described “medical freedom advocate,” told pharma reporter Max Bayer that the approvals were shocking.

The FDA did recently instruct Moderna and Pfizer to add an enhanced warning for the potential side effects. But Kennedy said in his confirmation hearing that he would not try to remove existing drugs or vaccines. 

As the infighting continues, Trump loyalty enforcer Laura Loomer is also taking aim at one of the FDA’s top leaders. Loomer, a conservative provocateur who traffics in conspiracy theories, leveled accusations at CBER Director Vinay Prasad of being a “progressive leftist saboteur” working to undermine Trump’s agenda. It’s not the first time Loomer has gone after FDA officials and MAHA personalities — earlier this year, she also targeted Calley and Casey Means after the latter’s nomination as surgeon general. 

🔬 CAR-T pioneer Carl June and colleagues finally unveiled the solid tumor cell therapy biotech that they’ve been cooking up since 2022. Philadelphia and San Francisco startup Dispatch Bio broke cover with $216 million to create a “universal treatment” for solid tumors. Cell therapies have transformed the treatment of liquid tumors like leukemia and lymphoma, but solid tumors have proven trickier to crack.

They joined forces with the Parker Institute for Cancer Immunotherapy and ARCH Venture Partners. The plan is to give patients a viral vector that places a synthetic antigen on cancer cells, supporting the immune system and making way for an autologous CAR-T therapy to then rid the body of the deadly cells.

Meanwhile, circular RNA startup Orbital Therapeutics provided an early look at how it hopes to enter the in vivo CAR-T cell therapy race. The well-funded biotech, loaded with $270 million in 2023, is starting with autoimmune diseases. The goal is to erase disease-causing B cells and return patients to a healthy state by delivering a simple infusion of lipid nanoparticles that carry RNA molecules. Those molecules are expected to reprogram T cells to hunt and kill the bad actors.

FDA Commissioner Marty Makary is overhauling leadership positions at the agency. Whereas CDER and CBER directors have often been career federal employees, Makary is instead opting for outsiders who could significantly change agency norms. With the appointment this week of George Tidmarsh as CDER director, Makary is shifting the roles from their traditional status as government professionals to political appointees.

Makary also convened a panel billed as a discussion of safely using SSRIs during pregnancy. But many of the panelists have long questioned antidepressant use, with some linking the drugs to autism and school shootings and calling the entire class a “placebo.” The panel lasted for about two hours on Monday and often veered into controversial and outlandish territory, though Makary only stayed for the first few minutes. 

🚫 The biotech received a complete response letter for RP1, which was being studied in second-line advanced melanoma. But the agency described its clinical trial as not “adequate and well-controlled” because of the “heterogeneity” of the patient population. Replimune shares fell more than 75% on Tuesday.