Vatiquinone Recieves CRL, RAINBOWFISH Study Published in NEJM, Hearing Aids Show Protective Effects Against Dementia

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

The FDA has handed PTC Therapeutics a complete response letter (CRL) for its investigational therapy vatiquinone as a new treatment for both pediatric and adult patients with Friedriech ataxia (FA), a rare neuromuscular condition. In the CRL, the agency noted that the evidence of efficacy was not substantial enough for vatiquinone, and that another well-controlled study would be needed to support new drug application (NDA) resubmission. Vatiquinone was aiming to become the second marketed treatment for FA, second to Biogen’s omaveloxolone, an FcRn inhibitor, which gained green light in early 2023 for patients 16 years and older with FA.

Investigators have published the final dataset of the phase 3 RAINBOWFISH trial (NCT02913482), a long-term study testing the safety and efficacy of risdiplam (Evrysdi; Roche), an FDA-approved RNA splicing modifier, as a treatment for presymptomatic spinal muscular atrophy (SMA). Published in the New England Journal of Medicine, results from the trial showed that infants up to 6 weeks of age with genetically diagnosed SMA who received treatment before signs or symptoms appeared had better functional and survival outcomes at 12 and 24 months than untreated infants in natural history studies. RAINBOWFISH, which included 26 infants with 2, 3, or 4 or more copies of SMN2, was the basis behind the drug’s expanded approval in presymptomatic SMA back in May 2022.

A study recently published in JAMA Neurology revealed that patients with hearing loss (HL) with hearing aids had reduced risk for dementia among those younger than 70 years at the time of hearing evaluation, when followed up for up to 20 years. Overall, these data point to the importance of early intervention for HL for possible prevention of dementia. Among those who developed all-cause dementia at the 2-decade follow-up, 42% (n = 245) were younger than 70 years at hearing assessment. Compared with participants with HL without hearing aids, those with HL with hearing aids had a 61% lower risk (HR, 0.39; 95% CI, 0.17-0.89; P = .03) for incident all-cause dementia among those younger than 70 years at HL diagnosis. Of note, participants with no HL had a 29% lower risk (HR, 0.71; 95% CI, 0.54-0.95; P = .02) for incident all-cause dementia.

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