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 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, the drug received a “breakthrough” therapy designation from the Food and Drug Administration, indicating preliminary evidence of potential for clinical benefit. Enrollment is underway for children, teens and adults with autism to test the drug in clinical trials, company officials said.
A second drugmaker, Yamo Pharmaceuticals, received “fast-track” designation from the FDA in May for its autism medication L1-79 that targets the central nervous system to improve speech and socialization. Compared to breakthrough therapies, fast-track designations have a lower standard to meet, requiring only that a drug treats a serious condition and fills an unmet medical need. Chuck Bramlage, Yamo’s CEO, said the company planned to meet with the FDA this fall to finalize details for a clinical trial set to start in early 2019.
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. The special designations from the FDA indicate the drugs show some early promise in a field with a lack of treatment options, said Dr. David Gortler, associate professor of pharmacology at Georgetown University and a former FDA medical officer.
“What they’re saying is, we believe there’s enough potential here to give it attention,” Gortler said. “They don’t do that randomly. There must be something they found early in the trial that actually does make a difference.”
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 and has since mainly been used for cancer drugs. But the designation is no guarantee of success. Of the 264 drugs to earn the status, 116 have gone on to be approved by the FDA, according to Friends of Cancer Research, a nonprofit involved in the creation of breakthrough designations.
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.
“Fast-track status is trying to take barriers out of the way, but it doesn’t mean that it works,” said Thomas Frazier, chief science officer for Autism Speaks. “If (the drugs) don’t work or they’re not safe, they shouldn’t be used, and we don’t know that yet.”
One potential roadblock in Yamo’s quest for approval of fast-tracked L1-79 is the company’s link to Michael Cohen, President Donald Trump’s former attorney who pleaded guilty to eight federal counts of fraud and campaign finance violations. Cohen made a pitch to drug company Novartis last year to help fund the development of L1-79 at the request of a client, a Yamo stakeholder. In a July letter to the U.S. Senate finance committee, a lawyer for Yamo denied any relationship with Cohen and urged federal authorities to allow the company’s autism research to continue unabated.
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.
“It is too early to discuss (balovaptan’s) availability on the market,” said Bob Purcell, a spokesman for Roche subsidiary Genentech, adding that “we understand the concerns that people living with serious conditions have about accessing new medicines as soon as possible.”