Former FDA chiefs rebuke vaccine official’s push for stricter review standards
Twelve former FDA commissioners criticized current Chief Medical and Scientific Officer Vinay Prasad for proposing sweeping changes to the agency’s vaccine review process, arguing in a New England Journal of Medicine article that his framework relies on selectively interpreted evidence and would undermine long-standing scientific standards. Prasad’s memo calls for large randomized trials for all new vaccine applications — including updates to existing flu and COVID-19 shots — a shift the prior commissioners say would slow access to improved vaccines, raise costs, and disadvantage smaller biotech firms. They also rebuked Prasad for citing unverified Vaccine Adverse Event Reporting System (VAERS) data to claim that COVID-19 vaccines caused at least 10 child deaths and for invoking nonexistent federal vaccine mandates to justify his position. According to the former commissioners, VAERS data alone cannot establish causality, and prior FDA and CDC reviews of the reported cases reached different conclusions, making Prasad’s rationale insufficient for overhauling a system designed to ensure timely, safe, and effective immunizations. – Andrea Corona
FDA outlines plan to cut back primate testing in some drug trials
In another move to reduce animal testing, the FDA released new guidelines on Tuesday signalling that the six-month non-human primate toxicity testing can be dropped or decreased for monoclonal antibody trials. The release noted that these types of preclinical trials can include more than 100 non-human primates with a cost of around $50,000 per animal. It also pointed towards the use of human-relevant models in the FDA’s decision making instead, like computational toxicology and organoid systems. “Modern science has given us far more effective and humane ways of evaluating drug safety than animal testing. This reform may reduce the amount of time it takes to bring a drug to market and lower research and development costs, which can translate into lower drug prices,” said FDA Commissioner Marty Makary in the press announcement. – Allison Whitten
UK pharmaceutical exports to US protected under new deal
The UK and the US have struck a landmark deal to keep tariffs on British pharmaceutical exports to America at zero for the next three years. Under the agreement, the NHS will pay more for medicines, marking the first increase in over 20 years, while UK drugmakers gain protection against US import taxes, safeguarding exports worth at least £5 billion annually. The deal comes after President Trump threatened tariffs of up to 100 percent on branded drug imports, a move that had raised concerns about investment in the UK’s life sciences sector. As part of the deal, the NHS will raise its spending on medicines, increasing the threshold for new treatments and capping paybacks from drug companies, with a projected boost of three to five extra drugs approved annually. Industry leaders welcomed the agreement, highlighting its potential to secure billions in investment and promote UK competitiveness, while analysts cautioned on the potential financial strain on the NHS. – Bree Foster
Capricor to seek FDA approval for Duchenne cell therapy again after Phase 3 results
Capricor Therapeutics announced new results from their Phase 3 HOPE-3 trial testing deramiocel, a first-in-class cell therapy, to treat cardiomyopathy in Duchenne muscular dystrophy (DMD). Their team reported that the drug significantly improved skeletal and cardiac endpoints with a favorable safety and tolerability profile. Stocks of Capricor soared after the announcement, which follows an earlier FDA rejection of the drug in July. Back then, the FDA cited that it “does not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data.” In the press release, Capricor said that they plan to include the new Phase 3 data in their response to the FDA’s Complete Response Letter. “We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 OLE studies, position us to address the clinical issues in the Complete Response Letter received earlier this year, consistent with prior FDA guidance that HOPE-3 results should be sufficient to support regulatory approval,” said Linda Marbán, CEO of Capricor, in the release. – Allison Whitten
New study maps how anti-seizure drugs work at molecular level
A team of researchers from St. Jude Children’s Research Hospital and UT Southwestern Medical Center has, for the first time, revealed how key epilepsy drugs interact with their target protein, offering new insights for drug development. Using cryo-electron microscopy, the scientists mapped the structural changes that occur in SV2A (synaptic vesicle glycoprotein 2A), a protein found in nearly all neurons, when anti-seizure medications like levetiracetam and brivaracetam bind. The study also identified an alternative allosteric site on SV2A that experimental modulators can target to boost drug potency. Understanding these interactions could help design more effective and specific therapies while reducing side effects. The findings, published in Nature Communications, shed light on a poorly understood but clinically critical protein and may guide the next generation of anti-epileptic treatments. – Bree Foster
Regeneron and Tessera partner on AATD gene editing therapy
Regeneron and Tessera Therapeutics have inked a global collaboration to codevelop and commercialize TSRA-196, an investigational in vivo gene-editing therapy designed to precisely correct the mutation that drives alpha-1 antitrypsin deficiency (AATD), an inherited lung and liver disease affecting roughly 200,000 people in the US and Europe. Tessera will receive $150 million in upfront cash and equity, with both companies sharing development costs and future profits 50/50, and Tessera is set to file an IND and multiple clinical trial applications by year-end. The partnership combines Regeneron’s longstanding genetics and clinical development expertise with Tessera’s Gene Writing and non-viral delivery platforms, with Tessera leading the first-in-human study before Regeneron takes over global development and commercialization. Preclinical data presented earlier this year showed durable, high-fidelity editing of the SERPINA1 (Serpin family A member 1) gene in animal models, supporting advancement into clinical testing pending routine regulatory clearances. – Andrea Corona
