Drugs

FDA OKs Leucovorin for Ultra-Rare Brain Disorder, But Not Autism

FDA has approved an expanded use of GSK’s Wellcovorin (leucovorin calcium) tablets for treating cerebral folate deficiency in adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (CFD-FOLR1). The approval fell short of agency and HHS expectations communicated in September that hinted the drug could be a potential treatment for autism.

Those earlier comments were controversial because the evidence supporting leucovorin for autism was limited — coming from small, preliminary studies — and the largest study had been retracted. Autism advocacy and medical groups cautioned that the science was weak and anecdotal.

Despite the limited evidence, prescriptions for leucovorin surged after the September White House event. A recent study in The Lancet found that prescriptions for children ages 5 to 17 increased by 71% in the three months following the announcement. FDA officials say the agency is allowing imports of leucovorin from foreign manufacturers to help maintain supply. The drug was originally marketed in the U.S. by GSK, which does not plan to relaunch its version.

FDA officials now say their review focused only on patients with a rare genetic condition that disrupts the transport of folate—a form of vitamin B—into the brain. The agency estimates the disorder affects fewer than one in a million people in the U.S. Patients with the condition targeted by the approval can experience seizures, movement disorders and other neurological symptoms that may resemble autism.

The approval was based on a systematic review of the published literature on the topic, including published case reports with patient-level information, as well as mechanistic data. "The approval of leucovorin for FOLR1-related cerebral folate transport deficiency (CFD- FOLR1) demonstrates the FDA’s commitment to rapidly identifying effective treatments for ultra rare diseases while maintaining the same evidentiary standards for approval,” CDER acting director Tracy Beth Hoeg is quoted in a release as saying. “It also provides a good example of how observational or ‘real world’ evidence can lead to an FDA approval when the product is shown to provide clear clinical benefit compared with what is seen with the natural history of the disease.”

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

Read more

Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome