Concomitant ASM Dosages With Stiripentol, FDA Pushes Givinostat Decision Back, Soticlestat Improves Caregiver Satisfaction

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

Based on data from a retrospective analysis of historical and current records of US prescribing practices from a singular specialty pharmacy, prescriptions of stiripentol tended to be lower than the approved dose level, with almost half those prescribed were not also taking concomitant clobazam. Stiripentol was approved by the FDA in 2018 and was available via compassionate use up until 2019, and the indication was expanded in 2022 to include the treatment of seizures associated with Dravet syndrome (DS) in individuals aged 6 months of age or older who weigh 15 pounds or more, and who are also being treated with clobazam. Notably, no clinical data yet support the use of stiripentol alone in DS.

Weeks before a decision was expected to be made for Italfarmaco’s investigational agent givinostat, the FDA informed the company it has extended the review process, with a new scheduled PDUFA of March 21, 2024. Givinostat, a proprietary HDAC inhibitor, is currently in development as a treatment for Duchenne muscular dystrophy (DMD), a severe neuromuscular genetic disease. The FDA accepted the new drug application (NDA) submission for givinostat earlier this year, with data from the phase 3 EPIDYS trial (NCT02851797) as the supporting evidence. Givinostat is designed to inhibit HDACs, which are enzymes that prevent gene translation by changing the 3-dimensional folding of DNA in the cell. In the latest update, Italfarmaco noted that the agency needs more time to review the application, but that there was no issues with the data submitted.

New 2-year data from the open-label extension (OLE) of the phase 2 ENDYMION 1 study showed that treatment with soticlestat, an antiseizure medication, was associated with high rates of caregiver-reported satisfaction. Across non-seizure symptoms/impacts faced by patients with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS), the highest levels of caregiver-reported improvement were in verbal and non-verbal communication. ENDYMION 1 is a multi-site, open-label, extension study assessing the long-term safety and tolerability of soticlestat, also known as TAK-935, in patients with developmental and epileptic encephalopathies like LGS and DS. In the trial, patients receive soticlestat twice a day, with site visits every 2-6 months throughout the study.

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