FDA Approves Programmable Pump System for Spasticity, Revised Treatment Recommendations for Spinal Muscular Atrophy, OnabotulinumtoxinA Trial Results

This week Neurology News Network covered the FDA approval for the Promentra II Programmable Pump System to treat severe spasticity, as well as the revised recommendations for treatment of spinal muscular atrophy. Finally, we discuss onabotulinumtoxinA the trial results for the migraine treatment.

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

The FDA has given market approval to Flowonix Medical’s Prometra II Programmable Pump System for use with intrathecal baclofen, which is used by patients with severe spasticity of cerebral or spinal origin. This indication expansion follows the company’s prior market entry in November 2019, when the Prometra II 40 mL pump was introduced. This offers the choice between 20 mL and 40 mL capacities, Flowonix noted in a statement. It announced that the device with the new indication will be immediately marketed. The Prometra Pump was originally approved by the FDA in 2012 to deliver Infumorph. Prior to that, it was approved in the United Kingdom and the European Union in 2011. To date, the system has been implanted in more than 8000 patients globally.

Cure SMA has announced that it has revised its recommendations for treating newborns with spinal muscular atrophy to include immediate treatment for those who have 4 copies of the SMN2 gene. The revised recommendations were published in the Journal of Neuromuscular Diseases. Originally published in May 2018, the recommendations, titled “Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening,” had previously recommended immediate treatment for individuals with 2 or 3 copies of SMN2, typically corresponding to the SMA type 1 and 2 clinical phenotypes. At the time, the multidisciplinary working group was unable to reach a consensus on immediate treatment in patients with 4 copies of SMN2, ultimately recommending that the decision to treat be made on a case by case basis with input from health care providers and parents.

Despite failing to meet its efficacy end points, onabotulinumtoxinA was deemed well-tolerated in an adolescent population with chronic migraine, with prior data in the adult population providing enough to push for additional, modified study in adolescents. All told, none of the 125-patient cohort discontinued treatment due to adverse events, with no serious adverse events considered related to treatment. OnabotulinumtoxinA was originally approved for the prevention of chronic migraine in adult patients in 2010. The study authors concluded, “To fully assess the benefits of treatment in adolescents, we suggest that future pediatric trials evaluate at least 2 treatment cycles of onabotulinumtoxinA, enroll larger numbers of patients, and explore the use of a placebo run‐in or crossover design.”

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