| The Food and Drug Administration is rolling back drug rejections following a leadership shakeup at the agency, easing concerns from industry and patient groups about an unpredictable approval process. The latest decision came Monday, when Regenxbio announced the FDA had agreed to a path forward for gene therapy for an ultra-rare disease called Hunter syndrome, months after rejecting the treatment and saying that the company needed to complete additional studies. Last Wednesday, the agency did a 180 and gave biotech company uniQure the green light to apply for accelerated approval for its Huntington’s disease treatment based on existing clinical data after previously pressing for additional evidence. The company, which agreed to do a confirmatory study, said in a statement that the outcome is the result of its “continued regulatory engagement with FDA.” That’s a contrast from November, when uniQure said it was “surprised” by feedback received during a meeting with the agency, in which the FDA had allegedly raised the expectations for what would be needed to submit the rare disease treatment for approval. Months of drama between regulators and the company had ensued prior to last week’s announcement. Key context: The decisions came following the departure of Marty Makary as FDA commissioner and several of his deputies, some of whom had sought to overhaul how medications and immunizations were approved and argued that some products were being authorized based on insufficient evidence. “Upon resubmission, the FDA will comprehensively review the data to assess the product’s safety, effectiveness and quality,” said Emily Hilliard, a spokesperson for the Department of Health and Human Services, in an emailed statement in response to an inquiry about the reversals since the agency’s leadership shakeup. “The agency remains committed to working closely with Regenxbio and the rare disease community to help advance promising therapies while maintaining the rigorous scientific standards that patients and families depend on,” Hillard added. In a statement, Regenxbio President and CEO Curran Simpson emphasized being “encouraged” by new leadership at the FDA. OTHER CHANGES - The FDA recently approved a new use for Sanofi’s Tzield after months of internal debate over the Type 1 diabetes treatment, including reported interference from Tracy Beth Høeg, the former top drug regulator who was fired after Makary’s departure.
- Replimune’s advanced melanoma therapy — which the FDA had twice rejected and requested additional data — has also been granted a renewed path toward resubmission following Makary’s departure.
In an announcement, the company said the application for its treatment, called RP1, would be resubmitted “following collaborative communications” with the FDA. Just days earlier, prior to leaving the agency, Makary defended the FDA’s decision to reject the treatment. - In a note to clients Monday, Chris Meekins, an analyst at Raymond James, pointed to comments from acting FDA leaders aimed at reassuring career staff and restoring confidence in the agency’s scientific review process.
“I respect your judgment and your dedication to the mission as we navigate this transition together,” acting FDA Commissioner Kyle Diamantas told staff in a note after taking over for Makary. Mike Davis, who is serving as the acting leader of the FDA office that regulates new medicines, said at an industry conference that “our credibility depends on our people.” Those comments, Meekins said, “may signal a return to a more traditional review model.” Why it matters: Last year, drug companies argued that the nation’s top drug and biologics regulators imposed increasingly unpredictable requirements needed for approval — in a process that some critics said had been plagued by politicization. Drugmakers, Wall Street investors and patient advocates had been similarly frustrated with the FDA’s repeated roadblocks when evaluating certain new medicines, while Makary publicly touted several efforts to streamline parts of the drug and biosimilar approval process. I wrote about this more in-depth in a November issue of Health Brief. - Comparatively, the more recent “decisions would not have been out of place in any previous administration in the past 20 years,” Paul Kim, a principal of Kendall Square Policy Strategies, who advises pharmaceutical companies and patient groups, tells me.
“This is a clear and welcome return to normalcy — live and in real time,” Kim said. The Trump administration announced a broad initiative to speed clinical trials for new drugs that HHS says could reduce early-trial timelines by six months to a year — an effort to shift more biotech investment back to the United States. “America should be the best place in the world to develop new medicines, yet we have built a system that drives too much clinical research overseas,” Health Secretary Robert F. Kennedy Jr. said Monday in a statement. “America led the world in medical innovation before. We will lead again.” The plan spans FDA, the National Institutes of Health, the Office of the National Coordinator for Health Information Technology, Advanced Research Projects Agency for Health (ARPA-H), and other agencies — and targets several bottlenecks involved in the development of new drugs, including during the earlier-stage trial initiation and the process of enrolling patients in clinical trials. - For example, the FDA has issued draft guidance clarifying that drug companies may be able to secure approval with one high-quality, late-stage study instead of multiple large trials, provided there is additional evidence supporting the findings.
- At NIH, officials are pushing researchers to conduct stronger studies and make greater use of AI, human cell-based models, real-world patient data and newer research tools that it says could speed the development of promising therapies “without compromising scientific rigor.”
- The HHS Office of Inspector General is reviewing whether anti-kickback rules are discouraging participation in clinical trials, and whether researchers should have more flexibility to offer financial support, such as travel reimbursements or stipends. It’s accepting public comment on the request for information over the next 60 days.
The pharmaceutical industry has been calling upon regulators to reduce barriers to clinical research while also increasing its overseas investments — particularly in China. But more on the latter topic below. “At a time when China is able to conduct clinical trials faster and cheaper than the U.S., it is imperative we advance solutions that enable America to continue leading in biopharmaceutical innovation,” said Chanse Jones, a spokesperson for the Pharmaceutical Research and Manufacturers of America, which represents some of the world’s largest drugmakers. “We applaud FDA’s efforts to accelerate early-stage clinical trials in the U.S. while maintaining rigorous oversight and safety protections for patients,” Jones said. The Trump administration said that “the entire U.S. research enterprise will need to do its part to streamline their processes” in order for the initiative to be successful. The announcement about bolstering domestic clinical trials comes when a growing share of the world’s drug research is moving through China. WP Intelligence Lead Health Care Analyst Rebecca Adams has a breakdown of the data about the competitive landscape, including battles over price, speed and China’s growing pipeline of new drugs. → As Rebecca notes, it’s been attracting attention from pharmaceutical giants, and concern among lawmakers and regulators in Washington. A key figure: China’s share of global early-stage clinical trial starts surged from 1 percent in 2009 to 30 percent in 2024, according to the IQVIA Institute for Human Data Science. → That’s in part because clinical trials are becoming materially cheaper and faster in China. - Phase 1 studies cost at least 30 percent less and finish an average of seven months faster than in the United States, generating roughly $2.5 million in savings per trial, according to GlobalData.
- The use of Chinese sites in global clinical trials nearly doubled, rising from 9 percent of worldwide trials in 2019 to 17 percent in 2025. By contrast, North America’s share remained flat, at roughly 20 percent over the same period.
- China’s innovation pipeline is expanding rapidly. McKinsey found China accounted for 24 percent of novel drugs in development worldwide in 2023, climbing to 30 percent by 2025.
→ Dealmaking between Western drugmakers and Chinese biotech firms has hit record levels, according to Rebecca’s report. Licensing activity and acquisition deals reached an all-time high in 2025, with nearly half involving U.S. companies. - The value of these partnerships is growing quickly. Up-front payments in cross-border biotech licensing deals increased from $1.1 billion in 2022 to more than $3.8 billion through mid-May 2026 alone. Deal volume rose from 43 transactions in 2022 to 92 in 2025.
→ Recent headline deals with Chinese companies underscore the trend: - AstraZeneca agreed to pay $1.2 billion upfront to partner with CSPC Pharmaceuticals on obesity and diabetes drugs, in a deal potentially worth more than $18 billion, while separately announcing a $15 billion investment in China through 2030, specifically to expand drug manufacturing and research and development.
- AbbVie committed $650 million upfront to RemeGen for the development, manufacturing and commercialization of a cancer therapy, with up to $5 billion in milestone payments.
- Bristol Myers Squibb struck a potential $15 billion agreement with Hengrui Pharma on cancer, hematology and immunology drugs.
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