Tofersen to Begin Expanded Access, FDA Grants De Novo to Chronic Stroke Rehab Therapy, Brain MRI to Predict Post-Partum Relapses


Neurology News Network for the week ending May 1, 2021.

This week Neurology News Network covered the 2-phase compassionate use program for Biogen's SOD1 ALS agent tofersen, the FDA de novo market authorization of the IpsiHand Upper Extremity Rehabilitation System, and data on how MRI can predict post-partum clinical relapses in patients with multiple sclerosis.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

Biogen has announced that its investigational SOD1 amyotrophic lateral sclerosis (ALS) agent, tofersen, which is still being assessed in a phase 3 clinical trial, will be made available to patients through a 2-phase compassionate use program beginning this summer. The first part of the access program is set to begin in mid-July 2021, with eligible patients including rapidly progressing patients—those with an ALS Functional Rating Scale-Revised (ALSFRS) slope decline greater than or equal to 2 points per month. Then, in the fall of this year, the company aims to offer access to the broader SOD1-ALS patient population—estimated to consist of near 2% of the greater ALS population—through an expanded access program, as long as there are no further study requirements, and specifically in countries where the practice is allowed by regulations and for whom Biogen can ensure future access to tofersen, formerly known as BIIB067. All of this will take place prior to application submission to the FDA.

Neurolutions has announced that the FDA has granted de novo market authorization, as well as breakthrough device designation, for its IpsiHand Upper Extremity Rehabilitation System to treat patients with chronic stroke.The IpsiHand system becomes the first FDA approved, device leveraging brain-computer interface technology to assist patients with chronic stroke in rehabilitation. The device utilizes the uninjured, or ipsilateral, side of the brain to improve arm and hand function and is indicated for patients 18 years and older. The FDA assessed the safety and effectiveness of the IpsiHand System device through clinical data submitted by the company, including an unblinded study of 40 patients over a 12-week trial period. All participants demonstrated motor function improvement with the device over the trial. Adverse events reported included minor fatigue and discomfort and temporary skin redness.

Data from a recent study suggest that brain magnetic resonance imaging activity (MRI) during the year before pregnancy can predict post-partum clinical relapses of multiple sclerosis. Researchers found that relapses in the first 3 months post-partum were significantly correlated with an active-MRI pre-pregnancy and expanded disability status scale (EDSS) score pre-pregnancy. The researchers found that mean annualized relapse rate (ARR) in the year pre-pregnancy was 0.34, with 47 women experiencing a relapse. Overall, 90.11% of pregnancies were relapse-free and mean ARR dropped to 0.069 during the second semester, with 3 women experiencing relapse. Mean ARR was 1.11 during the first 3 months post-partum, with 44 pregnancies (25.6%) experiencing 48 relapses, 0.53 in the 3- to 6-month period post-partum with 23 patients (13.4%) experiencing 23 relapses, and 0.4 in the 6- to 9-month period post-partum with 15 patients (8.7%) experiencing 17 relapses. Around half of all pregnancies (52.3%; 90 patients) remained relapse-free by the end of the first year post-partum

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