Between a high-profile FDA shakeup, mounting safety concerns in gene therapy, Trump-era policy ghosts rising from the grave, and a tariff war threatening supply chains, the summer of 2025 keeps bringing the plot twists. Mayo Clinic Venture Partner and Alliance for Regenerative Medicine board member Audrey Greenberg discusses the latest cliffhangers.
What happened with Sarepta and Elevidys?
The Elevidys story serves as a cautionary tale for the entire medicine-making ecosystem.
In March, Sarepta reported that a patient had died of acute liver failure after treatment with its Duchenne muscular dystrophy gene therapy, Elevidys. While liver toxicity is a known risk with adeno-associated virus (AAV) vectors, the subsequent deaths including one in another Sarepta program for limb-girdle muscular dystrophy amplified public and regulatory concern.
Following the third death, the FDA requested Sarepta suspend all Elevidys shipments in the US After initial resistance, the company complied. Roche, which controls ex-U.S. rights, followed by suspending supply to countries whose approvals were anchored in the FDA decision.
The agency has since recommended lifting the hold but only for ambulatory patients. Sarepta CEO Doug Ingram called the decision a vote of confidence, stating that commercial shipments had already resumed. However, non-ambulatory patients remain on pause, pending further risk mitigation measures.
This might be a signal that the AAV platform may no longer be afforded the benefit of the doubt. Developers must grapple with evolving safety standards, shifting physician sentiment, and wary investors. Analysts have already flagged near-term commercial softness, and payers may begin requiring stronger real-world evidence before approving reimbursement.
The Sarepta episode underscores a broader truth: manufacturing innovation must be matched by clinical responsibility and proactive risk management. In gene therapy, the science is spectacular but the margin for error is unforgiving.
Is MFN drug pricing really coming back?
Despite headlines suggesting Trump is resurrecting Most Favored Nation (MFN) pricing, there is no viable legislative path for implementation. Republican leadership, including Senator Bill Cassidy, has dismissed the proposal. While MFN remains a campaign soundbite, it faces fierce opposition from biopharma and is unlikely to be revived via executive order.
That said, the Inflation Reduction Act’s drug pricing provisions remain in effect, and additional pricing pressures are expected in FY26 budget negotiations. The upcoming election cycle will determine whether these pricing levers are expanded, reversed, or reframed.
What’s going on inside the FDA?
In a plot twist even by Washington standards, Vinay Prasad has been reinstated as director of CBER less than two weeks after stepping down. His short-lived departure followed internal backlash over his oversight of gene therapy programs and polarizing public statements. The reversal came after FDA Commissioner Marty Makary and HHS Secretary Robert F. Kennedy Jr. pushed for his return, framing it as essential to the agency’s mission.
So, is there any good news?
ARPA-H is still functioning with over $1 billion committed and multi-year funding secured to support platform technologies, distributed clinical trials, and biothreat preparedness programs. But broader science and health innovation funding, particularly related to the Inflation Reduction Act, is politically vulnerable. Support may be scaled back depending on the outcome of FY26 budget negotiations. The current momentum around AI-enabled drug discovery and public-private partnerships is still attracting private capital and industry collaboration.
What’s happening with tariffs and pharma supply chains?
The US has implemented 25 percent tariffs on APIs from China and 20 percent on generics from India. Trump has floated escalating those tariffs to 150 percent – possibly 250 percent. Meanwhile, Europe is now in the crosshairs too. Medicines from the EU face a new 15 percent tariff under a “reciprocal” trade policy.
These moves are already disrupting global supply chains. Manufacturers are scrambling to diversify sourcing to Mexico, Eastern Europe, and Southeast Asia. But shifting isn’t simple due to GMP revalidation, regulatory filings, and logistics costs all being real friction points. This is a system-level shock to how medicine is made and moved worldwide. Pharma is at the center of global economic warfare.
What can industry players do?
We can walk the halls of Congress, speak truth to power, and push for better. Science needs advocates because patients can’t wait. Silence is compliance. More than 160 attendees from 28 states joined with me recently to meet with 105 congressional offices and 12 members of congress to discuss NIH funding, Reauthorization of the Rare Pediatric Disease Priority Review Voucher program, passage of the Accelerating Kids’ Access to Care Act, and MFN pricing. We can show up with our feet, voices, and votes. When policy threatens science, innovation, or access to care, we don’t sit on the sidelines.
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