Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
FDA approves first treatment for rare cerebral folate deficiency
The FDA has approved the first treatment for cerebral folate transport deficiency, a rare genetic disorder that impairs folate transport into the brain. Wellcovorin (leucovorin calcium) tablets have traditionally been used to reduce symptoms from high-dose chemotherapy but are now approved for adult and pediatric patients with a confirmed FOLR1 (folate receptor alpha 1) gene variant. This provides the first FDA-sanctioned option for a condition that can cause severe developmental delays, seizures, movement disorders, and other neurological complications. The approval was supported by a systematic review of 26 published case reports and case reviews from 2009 to 2024, covering 46 patients treated through various administration routes. Among 27 patients receiving oral leucovorin, 24 showed clinical improvements, including reduced seizure severity, better motor function, and enhanced communication or behavior. FDA Commissioner Marty Makary called the decision a “significant milestone” for patients who previously had no approved therapies, while officials noted that observational and real-world evidence played a key role in demonstrating clinical benefit. – Bree Foster
Senator launches investigation into rare disease therapy rejections by FDA
On March 2, uniQure announced that they were issued complete response letters for their Huntington’s disease gene therapy, AMT-130, with the FDA insisting on an additional sham surgery-controlled Phase 3 trial. UniQure raised ethical concerns over creating sham burr holes in the skulls of control patients, which were echoed by Republican Senator Ron Johnson, who is launching an investigation into the FDA’s rejections of multiple rare disease drugs. On Monday, Johnson told Bloomberg News, “You’re expecting people to go through sham surgeries where they get holes drilled in their heads? ... That’s just unbelievable.” However, a senior FDA official disputed uniQure’s characterization of the new trial specifics, telling reporters in a conference call only “one to three nicks in the scalp” are required. The FDA official was later revealed to be Vinay Prasad, which comes on the heels of the announcement of his upcoming departure from the FDA in April 2026. Prasad’s 10-month tenure has been tumultuous, and this is not his first departure from the FDA because of rare disease drug regulatory decisions — he was briefly ousted following a dispute over Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, Elevidys. Johnson was personally involved in getting patients access back to that gene therapy. – Melissa Kay
Failed experiment leads to breakthrough in safer drug manufacturing
Scientists at the University of Cambridge have turned a laboratory setback into a potential game-changer for drug development, discovering a new light-powered chemical reaction that could make medicines safer, cheaper, and greener to produce. The unexpected finding, published in Nature Synthesis, allows researchers to modify complex drug molecules at late stages of production without toxic chemicals or heavy metal catalysts. The anti-Friedel–Crafts reaction, powered by simple LED light at room temperature, can forge critical carbon-carbon bonds with high precision, cutting months off conventional multi-step processes and reducing environmental impact. The breakthrough emerged when PhD researcher David Vahey noticed a control experiment worked even without the intended catalyst, prompting the team to explore the surprising result. Researchers say the method could accelerate the optimization of existing drugs and the creation of new ones, while collaborations with AstraZeneca and AI-driven predictive models suggest it could scale efficiently for industrial use. – Bree Foster
Novo Nordisk drops lawsuit as Hims switches to approved obesity drugs
Novo Nordisk and telehealth company Hims & Hers have reached a deal to make the diabetes and weight loss drugs Ozempic and Wegovy available through the Hims’ platform, ending a legal dispute and regulatory scrutiny over compounded versions of the medications. Under the agreement, Hims will offer the FDA-approved medicines at standard telehealth prices and stop promoting unapproved alternatives, while existing patients can switch to the branded treatments. In exchange, Novo will drop its patent infringement lawsuit against Hims, though it reserves the right to refile in the future. The move marks a major shift for Hims, which had previously sought to undercut the market with lower-cost knockoffs amid shortages of GLP-1 obesity drugs. Shares of Hims surged more than 40 percent on the news. – Bree Foster
Xenon shares breakthrough Phase 3 results in epilepsy treatment
Xenon Pharmaceuticals recently announced successful topline results from its Phase 3 X-TOLE2 study, which demonstrated that azetukalner significantly reduced the frequency of focal onset seizures. The study met its primary endpoint with a 53.2 percent median reduction in seizures for the 25mg dose group — a result that outperformed previous Phase 2b data and showed a highly significant lead over the placebo group’s 10.4 percent reduction. The drug maintained a favorable safety profile consistent with earlier trials, with common side effects such as dizziness and headache being generally well tolerated. Because azetukalner utilizes a unique KV7 potassium channel mechanism that requires no titration and offers once-daily dosing, it represents a potentially first-in-class treatment for patients with treatment-resistant epilepsy. Following these results, the company plans to submit a New Drug Application to the FDA in the third quarter of 2026. – Andrea Corona
FDA launches unified adverse event monitoring platform
The FDA has launched a new platform designed to consolidate and modernize how adverse event reports are accessed across regulated products. The FDA Adverse Event Monitoring System brings reports for drugs, biologics, vaccines, cosmetics, and animal food into a single searchable dashboard, replacing a patchwork of legacy databases that previously handled about six million reports each year. FDA Commissioner Marty Makary said the earlier systems were fragmented and difficult to search, limiting the usefulness of postmarket safety data. Over the coming months, the agency will migrate historical records and transition additional product centers to the platform, with real-time adverse event reporting for all FDA-regulated products expected by May 2026 while maintaining protections for identifiable patient information. Officials estimate the consolidation will save about $120 million over five years by replacing seven systems that cost roughly $37 million annually to maintain. FDA Chief AI Officer Jeremy Walsh said the rollout represents the largest technical transformation in the agency’s history. The platform will replace systems including FAERS for drugs and biologics, VAERS for vaccines, which areco-managed with the Centers for Disease Control and Prevention, and databases covering animal drugs and foods, with additional reporting systems for devices, foods, and tobacco products scheduled to migrate in May.– Andrea Corona
