NDA Submitted For CBD in Tuberous Sclerosis Complex, Ataluren Improves Walking Ability in DMD, Lasmiditan Available in Pharmacies

This week's Neurology News Network covered the new drug application for a CBD oral solution to treat seizures associated with tuberous sclerosis complex, as well as a trial that demonstrated an increase in walking ability for patient's with Duchenne muscular dystrophy. Additionally, NNN details lasmiditan's emergence to the shelves of pharmacies.

Marco: Welcome to Neurology News Network. I’m Marco Meglio. Let’s get into the news from this week.

GW Pharmaceuticals announced that it has submitted a supplemental new drug application for its cannabidiol oral solution to expand its indication to include seizures associated with tuberous sclerosis complex. The therapy was originally approved for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome for patients age 2 and older in 2018. It was rescheduled by the DEA to Schedule 5 in September of that year. Those who took CBD were more likely to experience a ≥50% reduction in seizures, observed in 36% of the 25 mg group and 40% of the 50 mg group compared to 22% for placebo. Caregivers reported an overall improvement in 69% of those taking 25 mg of CBD, 62% of those taking 50 mg, and 40% of those who received placebo.

An interim analysis of data from the STRIDE registry showed that boys with nonsense mutation Duchenne muscular dystrophy who received ataluren and standard of care preserved the ability to walk for years longer, compared to those on SoC alone. Those treated with ataluren as part of the STRIDE registry were able to walk independently for an additional 3.5 years compared with a propensity-score matched cohort from the CINRG DNHS. Investigators noted a median age at loss of ambulation was 14.5 years in the STRIDE registry compared to 11 years in CINRG DNHS, respectively. the final data from the STRIDE registry are expected in 2025.

Eli Lilly has announced that lasmiditan, an oral medication approved for the acute treatment of migraine with or without aura, is now available for prescription and will be available in pharmacies in the US in the next few days. Lasmiditan, which is the first molecule in the ditan drug class to be approved, is a non-opioid/non-narcotic, Schedule V medication that can be prescribed to patients in 50 mg, 100 mg, and 200 mg doses. The product will be available in 50 mg and 100 mg tablets. In October 2019, the FDA approved lasmiditan for the acute treatment of migraine with or without aura in adults. The approval was based off results from the SAMURAI and SPARTAN phase 3 trials, in which lasmiditan demonstrated a high rate of pain-freedom in patients with migraine, as well as relief from the most bothersome symptom.

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