NDA Accepted for Troriluzole, FDA Approves Mirdametinib for Neurofibromatosis, New Tablet Formulation for Risdiplam Approved

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

According to a new announcement from Biohaven, the FDA has accepted the company’s new drug application (NDA) for its investigational agent troriluzole, a third-generation prodrug that modulates glutamate, for the treatment of patients with spinocerebellar ataxia (SCA). The agency is expected to have a decision sometime in Q3 of this year and if approved, troriluzole would become the first marketed treatment specific to SCA. Troriluzole’s submission comprised data from the pivotal Study BHV4157-206-RWE (NCT06529146) along with confirmatory and supportive data from studies BHV4157-201 and BHV4157-206 (NCT03701399). Study BHV4157-206-RWE, which met its primary end point, comprised of multiple sources of real-world data, including the Clinical Research Conosrtium for the Study of Cerebellar Ataxia cohort (CRC-SCA), the European Integrated Project on Spinocerebellar Ataxias cohort (EUROSCA), and the 3-year open-label extension (OLE) data from troriluzole-treated patients from the previously completed Study BHV4157-206.

The FDA has granted approval to mirdametinib (Gomekli; SpringWorks Therapeutics) for the treatment of adult and pediatric patients aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) that are not amenable to complete surgical resection. The approval was based on data from the phase 2 ReNeu trial (NCT03962543), in which mirdametinib demonstrated an overall response rate (ORR) of 41% (95% CI, 29-55) in adult patients (n = 58) and 52% (95% CI, 38-65) in pediatric patients (n = 56). The treatment, which is a highly selective MEK inhibitor, offers a new option for patients with NF1 with inoperable PN, marking a significant advancement, particularly for adults who have lacked an FDA-approved therapy.

The FDA has approved a new tablet formulation for risdiplam (Evrysdi; Genentech), one of the few authorized therapies to treat patients with spinal muscular atrophy (SMA), according to a company announcement. With the approval, it becomes the first and only tablet available to this patient population, who often face challenges with digesting and taking medication. The new tablet, which comes in a 5-mg dose to be swallowed whole or dispersed in water, is expected to be available in the coming weeks. Similar to its existing prescribing information, the tablet is applicable for those with the disease aged 2 years or older who weigh more than 44 lbs. Risdiplam, a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier, was originally approved in 2020 as a liquid oral solution, becoming the second approved agent for SMA.

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