Weekly Rundown: FDA approves first treatment for rare cerebral folate deficiency Unexpected breakthroughs in drug manufacturing, lawsuits, regulatory updates, and more led the news this week.

https://www.drugdiscoverynews.com/weekly-rundown-fda-approves-first-treatment-for-rare-cerebral-folate-deficiency-17069 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