FDA Fast Tracks Drug Designed to Treat 'Core Symptoms' of Autism

The Food and Drug Administration has fast-tracked L1-79, a drug designed to treat the “core symptoms” of autism. This is the second drug for autism that has received an accelerated approval process from the FDA in 2018.

Yamo Pharmaceuticals requested “fast track” designation for its drug L1-79. The drug was well-tolerated in a small clinical trial of 40 people and has shown improvements in a number of areas, according to the pharmaceutical company.

Fast track designation makes developing a drug easier and expedites the FDA’s review of the drug. The administration gives this status to drugs that fill an “unmet medical need” for patients diagnosed with serious conditions, according to the FDA’s website. If other treatments exist, the drug has to prove itself superior. The drug has to show effectiveness in its early stages and be safe or have few side effects.

“Preliminary data shows that within days of starting L1-79 therapy, there was a reduction in some patients’ irritability, lethargy, hyperactivity and anxiety, and improvements in speech, communication, socialization and awareness of themselves and others, resulting in improved behavior and better manageability of the patients,” the company stated.

Yamo said the drug is based on a different agent that is already approved by the FDA and has had extensive research, though the company did not specify which drug. It is in the process of recruiting for a larger clinical trial for L1-79.

In March, the FDA gave breakthrough therapy designation to balovaptan, another drug aimed at the “core symptoms” of autism. Breakthrough therapy designation is similar to being fast-tracked, giving approved drugs all fast track features as well as other FDA guidance. Balovaptan is aimed primarily at helping communication and social skills.

Though development for these drugs is underway, many in the autism community oppose “treating” or “curing” autism. Instead, many autistic people advocate for neurodiversity acceptance.

“Most of us in the autism community do not feel that we are ‘disordered’ and therefore would lean away from treatment. Generally, we enjoy celebrating our unique gifts and talents,” David Gray-Hammond, a member of The Mighty’s autism community, told The Mighty about balovaptan. “That said, for some people the symptoms of autism are very overwhelming, and it is important that these people have the option of medication open to them.”

While Gray-Hammond said he would consider taking a medication like balovaptan, another member of The Mighty’s autism community said they would not. N.A. LeBrun told The Mighty a medication like this may have been intriguing when they were a teenager and wanted to fit in, but not as an adult.

“Sometimes these ‘core symptoms’ are what enable us to function in the neurotypical world, but for some they are indeed restrictive,” LeBrun said. “It’s an individual thing. For example, my repetitive stims like tapping my fingers when in public, enable me to better focus and communicate with others, whereas I know that some neurotypical people find ‘flapping’ to be off-putting, but does that say more about them or the autistic person who is carrying out the behavior?”