ABBV-951 Gets Another CRL, Pitolisant Approved for Pediatric Narcolepsy, FDA Clears Efgartigimod for CIDP

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

The FDA has again issued a complete response letter (CRL) to AbbVie's new drug application (NDA) for ABBV-951 (foscarbidopa/foslevodopa) for the treatment of motor fluctuations in adults with advanced Parkinson disease (PD). In the CRL, the agency cited observations identified during inspection of a third-party manufacturer listed in the NDA. The inspection at the facility did not involve ABBV-951 or any of the company's medicine. The CRL did not identify any issues in terms of safety, efficacy, or labeling of ABBV-951, including the device. Furthermore, the letter did not request that the company perform additional efficacy and safety studies related to the drug or device-related testing.

Months after being granted priority review designation by the FDA, the agency has granted approval to Harmony Biosciences' pitolisant (Wakix), a selective histamine H3 receptor inverse agonist, for the treatment of excessive daytime sleepiness (EDS) in pediatric patients aged 6 years and older with narcolepsy.1 In addition, the agency issued pitolisant a complete response for the treatment of cataplexy in pediatric patients with narcolepsy. Pitolisant's supplemental new drug application was supported by findings from a phase 3 trial (NCT02611687), which demonstrated significant reductions in EDS and cataplexy in pediatric patients aged 6 to 17 years with narcolepsy treated with 5 mg to 40 mg a day of the therapy. These findings suggested that pitolisant is a safe and effective treatment for children and adolescents with narcolepsy.

The FDA has approved Argenx's coformulation therapy efgartigimod alfa and hiya-luronidase-qvfc (Vyvgart Hytrulo) as a new treatment for adults with chronic inflammatory demyelinating polyneuropathy (CIDP). The therapy is the first and only neonatal Fc receptor (FcRn) blocker approved for the treatment of CIDP, and will be available as a once-weekly, 30- to 90-second subcutaneous injection. The therapy was approved based on data from the pivotal phase 3 ADHERE study (NCT04281472) in which treatment with the agent significantly reduced relapse risk compared with placebo. All told, patients treated with Vyvgart Hytrulo had a 61% attenuated risk relative to placebo (P = .00039), with a safety profile that was consistent with previous studies.

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