FDA Accepts NDA for Tofersen, Serious Falls Increased With Hypnotic Medications, Racial Disparities in Sleep of Pregnant Women


Neurology News Network for the week ending July 30, 2022. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

Transcript below.

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

Ionis has announced that the FDA has accepted the new drug application (NDA) for tofersen, an investigational antisense medicine being evaluating for the potential treatment of SOD1 amyotrophic lateral sclerosis (ALS). Additionally, the agency granted priority review and set a PDUFA date of January 25, 2023.There are currently no approved treatments for patients with SOD1 mutations, and only a handful of approved therapies for ALS in general. Tofersen will be seeking approval under the accelerated approval pathway based on data that showed changes in neurofilament, a biomarker of neuronal degeneration. The basis of the NDA included data from a phase 1 study of healthy volunteers, a phase 1/2 dose ascending study, the phase 3 VALOR study, and its open label extension. In VALOR, the most recently reported study, treatment with tofersen resulted in reductions in neurofilament light, accompanied by trends toward reduced disease progression and reductions in SOD1 protein in patients with SOD1 ALS.

Using data from the REGARDS study, recently published findings showed a higher incidence of serious falls among participants taking vs not taking a prescribed hypnotic medication regardless of whether they had an insomnia diagnosis. Notably, the combination of insomnia diagnosis and use of a hypnotic medication accounted for the highest cumulative incidence of serious fall injury.Among a cohort of 9087 individuals aged 65 years or older, 1660 (18.3%) participants had a serious fall injury over a median of 6.8 years. The incidence of a serious fall injury was highest among those with both insomnia and on hypnotics (+insomnia/+hypnotics), with 46.6 per 1000 person-years, followed by those without insomnia and on hypnotics, with 32.6 per 1000 PY; those with insomnia without hypnotics, with 28.8 per 1000 PY, and those without insomnia nor on hypnotics, with 24.8 per 1000 PY. The reference group, those without insomnia or on hypnotics, had a lower risk for serious fall injury relative to all other groups after initial adjustment for age, sex, race, geographic region of residence, education, household income, and current cigarette smoking.

As previous data have linked poor sleep to adverse maternal outcomes, new findings from the Child Health Outcomes (ECHO) program identified stark racial differences among pregnant women in terms of sleep health, and more specifically, sleep duration and sleep disturbances.The complete dataset, published in SLEEP, came less than a month after the first results were presented at the 2022 SLEEP Annual Meeting, June 4-8, in Charlotte, North Carolina. Among the key findings, non-Hispanic Black/African American (nHB/AA) participants reported shorter sleep duration and more disturbances but not worse subjective sleep quality compared with non-Hispanic White (nHW) participants. Between non-Hispanic Asian (nHA) and nHW participants, there were no significant differences in any sleep domain across trimesters. The investigators conclude that the "results from this study could inform the identification of modifiable factors and targeted interventions to improve barriers to sleep health with potential positive effects for downstream health outcomes among pregnant people and their children." They also noted that that further research is warranted to investigate the determinants of sleep disparities.

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