FDA withdraws autism drug approval
The FDA has withdrawn approval of a drug promoted as an autism treatment, while generics of that product will remain available according to the reporting source. The action is notable because it shows the agency can use approval withdrawal—not only labeling or enforcement letters—when it determines the approval is no longer justified. (theepochtimes.com)
The Food and Drug Administration just erased the approval for Wellcovorin, a branded leucovorin tablet from GlaxoSmithKline, even though the same medicine is still sold as generics. The withdrawal took effect on April 10, 2026, and it covers the 5 milligram and 25 milligram tablet versions in New Drug Application 018342. (federalregister.gov) This was not a safety recall. GlaxoSmithKline told the agency the product was no longer being marketed, and the Food and Drug Administration said approval was being withdrawn at the company’s request under the voluntary withdrawal rule in 21 Code of Federal Regulations 314.150(c). (federalregister.gov) The odd part is timing. Wellcovorin had already been off the market for decades, but the Food and Drug Administration announced on September 22, 2025 that it was reviving the old approval so the label could be updated for cerebral folate deficiency, a rare condition that blocks folate from getting into the brain. (fda.gov) Leucovorin is a prescription form of folinic acid, which is a usable version of vitamin B9. In people with cerebral folate deficiency, the problem is less like “not enough fuel” and more like “the fuel truck cannot get through the gate,” so doctors try a form that can bypass part of that bottleneck. (fda.gov) That rare disorder is not autism. On March 10, 2026, the Food and Drug Administration approved leucovorin for people with cerebral folate deficiency who have a confirmed variant in the folate receptor 1 gene, while agency officials also said they did not have enough data to show the drug works for autism more broadly. (statnews.com) That March decision was a retreat from what top officials had said six months earlier. Commissioner Marty Makary said on September 22, 2025 that the agency was going to approve leucovorin “for the treatment of autism,” and Health and Human Services Secretary Robert F. Kennedy Jr. called it a therapy that might help large numbers of children. (fda.gov, theepochtimes.com) The evidence never fully caught up with that rhetoric. STAT reported that the biggest study claiming benefit in autism was retracted in January 2026, and the agency narrowed its review to the much smaller group with a specific folate-transport disorder instead of autism as a whole. (statnews.com) So what changed on April 10 was the legal status of one brand, not access to the underlying drug. The Food and Drug Administration told reporters that generic leucovorin remains available for patients with cerebral folate deficiency, and the Federal Register notice says any remaining Wellcovorin inventory can still be dispensed until it runs out or expires. (theepochtimes.com, federalregister.gov) What this leaves behind is a paper trail showing how far the agency went, and then backed away. In September 2025 it used an unusual process to revive a long-discontinued 1983 drug approval, and in April 2026 it used another formal process to withdraw that approval altogether when the sponsor said the product was not being sold. (fda.gov, federalregister.gov, thefdalawblog.com)
