Pitolisant Being Evaluated for Idiopathic Hypersomnia, FDA Approves Ravulizumab for Generalized Myasthenia Gravis, Gender Differences in MS Disease Activity

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A newly initiated phase 3 study, dubbed INTUNE will evaluate the efficacy and safety of pitolisant (Wakix; Harmony Biosciences) in patients with idiopathic hypersomnia (IH), a condition that currently has only 1 FDA-approved medication. Pitolisant, a selective histamine 3 receptor antagonist/inverse agonist, is already FDA-approved to treat excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. INTUNE, a double-blind, placebo-controlled, randomized, withdrawal study that spans across 60 to 80 sites in the US, will include approximately 200 patients with IH. The primary outcome measure of the trial will be the Change in Epworth Sleepiness Scale (ESS) score for pitolisant compared with placebo. In addition to evaluating efficacy and safety as the primary objective, investigators will also assess the impact of pitolisant on other symptoms of IH, such as sleep inertia and cognitive impairment. Other outcomes, including patient impression of overall change in symptoms, investigator assessment of overall disease activity, and functional status will also be evaluated in the study.

The FDA has approved ravulizumab (Ultomiris; Alexion), a terminal compliment C5 inhibitor, for the treatment of patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. With the decision, ravulizumab becomes the first approved long-acting C5 complement inhibitor for this patient population.The approval was based on data from the phase 3 CHAMPION MG trial in which treatment with ravulizumab resulted in rapid and sustained improvement of symptoms in patients with gMG for up to 26 weeks. At the end of the treatment period, investigators observed statistically significant improvements on the primary end point of Myasthenia Gravis Activities of Daily Living (MG-ADL) total score compared with placebo. Primary investigator James F. Howard, MD said in a statement that, “despite recent advances, managing gMG is complex. Earlier intervention can preserve function and quality of life. This approval offers patients, including those with milder symptoms, a long-acting C5 inhibitor with early onset and reliable efficacy.

Findings from a nationwide study of patients with relapsing multiple sclerosis (MS) showed statistically significant differences between the sexes, as women demonstrated more inflammatory disease activity while men accrued more disability with time than women. The difference in inflammatory disease activity subsided following the age of 50 years, suggesting that sex hormones may play a role in the earlier stages.The trial included 3028 men and 6619 women on a disease-modifying therapy from the Danish MS Registry (DMSR) who had clinical onset of relapsing MS or clinically isolated syndrome (CIS) since 1996. Patients were indexed by date of birth, date of onset, and date of entry, which was the date of the commencement of the first DMT. From this time, relapses and Expanded Disability Status Scale (EDSS) scores were recorded systematically. Men demonstrated a higher cumulative probability of reaching the confirmed and sustained end points of EDSS 3.0, EDSS 4.0, and EDSS 6.0 than women, regardless of whether onset or age 20 years was the starting point. The cumulative probability of not reaching EDSS 2.0 by year 20, or what the investigators defined as “benign MS,” was 22.5% (95% CI, 21.0-24.0) in women and 13.1% (95% CI, 10.7-15.4) in men

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