FDA Grants Breakthrough Therapy Designation to Expedite Potential Pediatric Insomnia Drug

Key takeaways:

• The FDA granted Breakthrough Therapy Designation for daridorexant to treat insomnia disorder in patients aged 10 to under 18 years.
• A phase 2 study demonstrated that a single 50 mg dose increased total sleep time by 50 minutes overall and by 64 minutes in patients with neurodevelopmental disorders.
• A pediatric Phase 3 clinical development program is expected to begin in the second half of next year.

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to daridorexant for the treatment of insomnia disorder in patients 10 to under 18 years of age.

Developed by Idorsia, daridorexant is a dual orexin receptor antagonist (DORA) designed to selectively reduce wake drive rather than broadly inhibiting brain activity. The designation provides for intensive FDA guidance and closer collaboration to expedite the drug’s development for the pediatric population, which currently has no FDA-approved pharmacological treatments for insomnia.

“This Breakthrough Therapy Designation underscores both the significant unmet medical need faced by children and adolescents living with insomnia disorder and the encouraging clinical evidence generated with daridorexant to date,” says Amer Joseph, MD, Idorsia chief medical officer and head of global clinical development, in a release. “Daridorexant was specifically designed with an optimized pharmacokinetic and tolerability profile, allowing it to be largely cleared by the next morning and avoiding accumulation with repeated dosing.”

The FDA’s decision is supported by data from a multicenter, randomized, double-blind, placebo-controlled phase 2 dose-finding study. The trial evaluated doses of 10, 25, and 50 mg of daridorexant, or placebo, over two weeks in 165 children and adolescents. Analysis of the primary endpoint demonstrated a statistically significant dose-dependent improvement in objective total sleep time from baseline on day 1.

According to the trial results, a single 50 mg dose increased sleep time by 50 minutes overall and by 64 minutes in children and adolescents with neurodevelopmental disorders. The study also confirmed the safety and tolerability profile of the drug in pediatric patients, noting no adverse events denoting drug abuse during treatment and no indication of withdrawal symptoms upon treatment discontinuation.

Pediatric insomnia affects approximately 8% to 12% of children and adolescents, and the burden is particularly high in those with neurodevelopmental disorders, including autism spectrum disorder and attention-deficit/hyperactivity disorder.

Idorsia is currently consolidating supportive comments from global health authorities on the protocol for the pediatric phase 3 clinical development program and expects to initiate the new program in the second half of next year.

More in Pediatric Insomnia Treatment:

ID 171097951© Sergiomonti| Dreamstime.com