Federal regulators put two experimental drugs targeting autism’s core symptoms on a fast track for testing and approval this year, but they’re still a long way from getting approval and arrival on pharmacy shelves. Balovaptan, from drugmaker Roche, aims to improve communication and social skills for people with autism by signaling a hormone linked to behavior. In January of this year, the drug received a special designation as a “breakthrough” therapy from the Food and Drug Administration, indicating that there is preliminary evidence of potential for clinical benefit. Enrollment into studies is underway for children, teens and adults with autism to test the drug in clinical trials. A second drugmaker, Yamo Pharmaceuticals,also received a special designation as a “fast-track” from the FDA in May of last year for its autism medication L1-79 that targets the central nervous system to improve speech and socialization.
Currently, just two drugs — risperidone and aripripazole — are FDA approved to treat irritability associated with the developmental disorder. If approved, balovaptan and L1-79 would be the first to address autism’s core symptoms, which the two previously indicated drugs do not. The special designations from the FDA indicate the drugs show some early promise in a field with a lack of treatment options. Balovaptan is thought to be the first autism drug to receive the breakthrough designation, which was created in 2012 by the FDA Safety and Innovation Act. At least two other drugs aimed at autism have received fast-track status, which has been granted to 283 drugs since 1998, according to the FDA. Curemark’s CM-AT received the designation in 2010 and is still being studied. Neuropharm earned fast-track status for the antidepressant Prozac as an autism treatment in 2008, but clinical trials did not show improvement in repetitive behaviors. The FDA also granted fast-track status this year to Ovid Therapeutics’ gaboxadol for the treatment of fragile X syndrome. Yamo’s L1-79 is in phase two of the drug testing process, and only one-third of experimental drugs make it to phase three. Clinical trials on balovaptan, which are in phase two and phase three, are expected to be completed in 2023 or beyond.