EU Approves Tofersen for SOD1 ALS, SynAIRgy Study Aims to Change OSA Treatment, FDA Approves New Dosing for Amifampridine

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

Slightly more than a year after tofersen (Qalsody; Biogen) was approved in the US, Biogen has announced its EU approval for the treatment of SOD1-mutated amyotrophic lateral sclerosis (ALS). Approved under the accelerated approval pathway, it remains the first and only marketed therapy to treat this genetic form of the disease, which impacts approximately 2% of the patient population. The latest approval comes under exceptional circumstances, meaning that there is a positive benefit/risk profile; however, considering the rarity of the disease, it is unlikely that comprehensive data can be obtained under normal conditions of use. Tofersen, an antisense oligonucleotide, was approved based on data from the phase 3 VALOR trial, the same double-blind, placebo-controlled trial that served as supportive evidence for its FDA approval.

At the 2024 SLEEP Annual Meeting, held June 1-5, in Houston, Texas, investigators presented an outline of the phase 3 SynAIRgy trial (NCT05813275), a randomized, controlled study evaluating the efficacy and safety of AD109 (Apnimed), a combination agent, as a potential treatment for obstructive sleep apnea (OSA). AD109, a first-in-class once-daily medication, aims to be the first oral pharmacologic therapy that targets key neurological pathways in OSA that activate upper airway dilator muscles to maintain an open airway during sleep. The study, which is currently ongoing, includes approximately 640 participants with OSA who are randomly assigned 1:1 to either AD109 or placebo for a 26-week treatment period. Those in the trial are adults who either decline or unable to tolerate continuous positive airway pressure (CPAP) treatment, otherwise considered the gold standard for OSA. In addition, eligible patients have an apnea-hypopnea index(AHI)-4 (4%) desaturation definition for hypopneas) greater than 5 and a body mass index less than 42 kg/m2 for women and less than 40 kg/m2 for men.

According to an announcement, the FDA has approved Catalyst Pharmaceuticals’ supplemental new drug application (sNDA) for a higher maximum daily dose of its potassium channel blocker amifampridine (Firdapse) from 80 mg to 100 mg for the treatment of adults and pediatric patients weighing more than 45 kg with Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune neuromuscular disorder characterized primarily by muscle weakness of the limbs. This decision provides an additional indicated dosage option for patients with LEMS who might benefit from a daily dosage greater than 80 mg of amifampridine. In November 2018, the FDA approved the drug for patients with LEMS aged 17 and older and approved the expanded use of the treatment to include patients as young as age 6 in 2022.

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