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FDA fast tracks novel autism drug
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The FDA recently granted fast track designation status to the tyrosine hydroxylase inhibitor known as L1-79, a potential treatment for autism, according to a press release from Yamo Pharmaceuticals, the drug’s manufacturer.
“The only autism spectrum disorder treatments that are approved are for agitation and irritability in the subset of children with autism who have those symptoms,” J. Thomas Megerian, MD, PhD, Yamo Pharmaceutical’s chief medical officer and senior vice president of clinical development, told Healio Family Medicine. “There are no pharmacotherapies available for treating the core social communication and interaction deficits in autism spectrum disorder ... which is why it is so important for us to develop therapies for this disorder.”
Megerian said a recent phase 2 trial that led the FDA to grant fast track designation consisted of two cohorts of 20 patients (mean age, 16 years) who received a dose of either 100 or 200 mg of L1-79 three times a day. In each cohort, five patients received open label drug, and 15 patients were randomized in a 2:1 ratio to receive the drug or a placebo. No serious adverse events were noted and most such events were mild to moderate.
“Our next planned study is a second, 3-month long, phase 2b trial of approximately 250 male and female patients between the ages of 12 and 21 years. This trial is scheduled to start in early 2019,” Megerian said.
He added that Yamo Pharmaceuticals anticipates submitting L1-79 to the FDA for approval in 2022. – by Janel Miller
Disclosures: Megerian works for Yamo Pharmaceuticals.