FDA Approves Essential Tremor Treatment, Alzheimer's Association Files Request to CMS, Duchenne Therapy ENTR-601-44 Placed on Hold


Neurology News Network for the week ending December 31, 2022. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

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

According to an announcement, the FDA has approved an expanded indication of Insightec’s Exablate Neuro platform to include the treatment of both sides of the body in patients with essential tremor.The device, which uses focused ultrasound (FUS) waves, was originally approved for the treatment of medication-refractory ET in 2016, and later received approval for tremor-dominant Parkinson disease (PD) in 2018. The new approval allows appropriate patients to have their second side treated at least 9 months after treatment of the first side. The basis for the new indication came from a 51-patient cohort study that spanned 7 academic medical centers in the US over a 6-month period. There, investigators observed significant reductions in tremor in the second side of patients immediately following treatment with Exablate, which was sustained through at least 6 months of follow-up. Notably, these findings were consistent with the first side treated. In the study, there was a significant improvement in functional disability and the safety profile was similar to the first side, with the majority of adverse events being mild in nature.

According to an announcement from the Alzheimer’s Association, it has filed a formal request to the Centers for Medicare & Medicaid Services (CMS) to remove the requirements for Coverage with Evidence Development (CED) in its national coverage determination (NCD) for FDA-approved antiamyloid therapies for Alzheimer disease (AD).In April 2022, CMS published its final decision memo on its national coverage analysis for the class of monoclonal antibodies as treatments of AD, restricting the coverage of these products to 2 scenarios. The first provided coverage for approved agents that show change in surrogate end points in randomized controlled trials conducted under an investigational new drug application, while the second provided coverage in CMS-approved prospective comparative studies. Study data for CMS-approved prospective comparative studies must also be collected in a registry.

Entrada Therapeutics has announced that the FDA has placed a clinical hold on the investigational new drug application for its investigational Duchenne muscular dystrophy (DMD) treatment, ENTR-601-44.ENTR-601-44 is an exon 44 skipping oligonucleotide developed with Entrada’s Endosomal Escape Vehicle (EEV) platform. It aims to target the underlying genetic cause of DMD to allow muscle cells to produce functional dystrophin, and the company estimates that ENRE-601-44 will benefit roughly 7.5% of the DMD population who are considered exon 44 skipping amenable. The agency noted that within 30 days, it will supply Entrada with a clinical hold letter, and the company stated that it planned to share updates regarding further communication with the FDA.

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