FDA Action Update, December 2021: Approvals, Acceptances, Designations


Catch up on any of the neurology news headlines you may have missed over the course of the last month, compiled all into one place by the NeurologyLive® team.

A number of FDA actions took place in December 2021, including a first-in-class approval for generalized myasthenia gravis, a medical device exemption for a cognitive assessment, and the acceptance of applications for a number of therapeutics for neurologic diseases.

With all the treatments that have progressed through the pipeline of clinical development, the NeurologyLive® team has been hard at work covering all the agency movements to make sure you are up to date on the latest news in neurology. To give you a chance to catch up on any of the headlines you may have missed over the course of the last month, we’ve compiled all the updates into one place. The coverage includes the latest FDA approvals, new designations, submissions and resubmissions, and clinical trial initiations.

Click the read more buttons for more detail and information about each update.

FDA Accepts Grants Priority Review to Fenfluramine for Lennox-Gastaut Syndrome

December 2021 opened with an announcement from Zogenix that the FDA had accepted for filing a supplemental new drug application (sNDA) and granted priority review designation to fenfluramine (Fintepla) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS). The agency noted that it plans to complete the review within the required 6-month window, with a prescription user drug fee act (PDUFA) date set for March 25, 2022.1

An approval for LGS would be the second indication for fenfluramine after originally being greenlit by the FDA in June 2020 to treat seizures associated with Dravet syndrome in patients 2 years of age and older.

This application, submitted in September 2021, was backed by data from the phase 3 Study 1601 (NCT03355209), which showed that treatment with fenfluramine at a dose of 0.7 mg/kg/day was superior to placebo in reducing the monthly drop seizure frequency (MDSF; = .0012). Additional long-term safety and effectiveness data in ongoing open-label extension trials were also included in the submission. Gail Farfel, PhD, executive vice president and chief development officer, Zogenix, said in a statement that “this is a critically important milestone for our FINTEPLA development program in LGS and brings us one step closer to a potential new treatment option for this rare and difficult to treat childhood developmental and epileptic encephalopathy.”

FDA Accepts Grants Priority Review to Fenfluramine for Lennox-Gastaut Syndrome

Cognetivity’s Integrated Cognitive Assessment Device Completes FDA Registration

On the same day as the Zogenix news, Cognetivity Neurosciences announced that its Integrated Cognitive Assessment (CognICA) had met the requirements for a Class II Exempt Medical Device, following review by the FDA. As Cognetivity received confirmation on its 510(k) submission the month before, it was then permitted to market the medical device for commercial distribution in the US.2

The ICA device was previously granted regulatory approval in Europe, marketed as a CE-marked medical device. In the UK’s National Health Service, the device has been integrated into both primary and specialist clinical care. Sina Habibi, PhD, CEO, Cognetivity, said in a statement that “this grants us access to the world’s largest healthcare market, where, sadly, there is much more to be done to tackle the massive problem of dementia. Of course, we’re excited about the opportunity to revolutionize the way cognitive impairment is assessed and managed in the US and make a positive impact on the health and wellbeing of millions of Americans.”

Delivered via iPad devices, the CognICA is a computerized cognitive assessment for studies into dementias, taking patients approximately 5 minutes to complete. The system is artificial intelligence-powered, offering benefit in comparison to traditional pen-and-paper tests that require administration by a health care professional. The device has a high sensitivity to early-stage cognitive impairment and also avoids cultural and educational bias and shows an absence of learning effect when administering repeat testing. The device will integrate with existing electronic health records systems, as testing is able to be remotely self-administered at scale.

Cognetivity’s Integrated Cognitive Assessment Device Completes FDA Registration

FDA Accepts Ublituximab Biologics License Application for Relapsing MS

In mid-December, TG Therapeutics announced that a biologics license application (BLA) for ublituximab, its investigational glycoengineering anti-CD20 monoclonal antibody, was accepted by the FDA for the treatment of relapsing forms of multiple sclerosis (RMS).3

The FDA noted that it does not plan to hold an advisory committee meeting to discuss the application and has set a prescription drug user fee goal date of September 28, 2022. The BLA was submitted in September 2021 following positive results from the company’s ULTIMATE 1 and 2 trials (NCT03277261; NCT03277248), identical phase 3 studies that evaluated ublituximab compared with teriflunomide (Aubagio; Sanofi) in patients with RMS. Both trials were conducted under a special protocol assessment established with the FDA.

“We are excited to share that we have received formal communication from the FDA, and the BLA for ublituximab to treat relapsing forms of MS has been accepted and granted a standard review,” Michael S. Weiss, chairman and CEO, TG Therapeutics, said in a statement at the time. “This is a major milestone for us as it is our first US marketing application for an autoimmune indication. We look forward to working with the FDA throughout this review process.”

FDA Accepts Ublituximab Biologics License Application for Relapsing MS

Efgartigimod Approved for Generalized Myasthenia Gravis

A few days later, the FDA announced that it had approved Argenx’s first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn), efgartigimod (Vyvgart), for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody.4

The agency had previously granted the therapy both fast track and orphan drug designations. The biologics license application (BLA) submitted to the agency was supported by data from the phase 3 ADAPT trial (NCT03669588). All told, 167 patients were randomized to an evaluation arm in the ADAPT trial, 84 who were randomized to the efgartigimod group and 83 to the placebo group. Of the full population, 77% (n = 129) were AChR-antibody positive.

Billy Dunn, MD, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, said in a statement that the approval "is an important step in providing a novel therapy option for patients and underscores the agency’s commitment to help make new treatment options available for people living with rare diseases.”

"The approval of efgartigimod by the FDA is going to again transform our management of myasthenia gravis, without question. The ability of this drug to reduce levels of circulating antibody IgG will provide patients with a new option for therapy, a much safer option for therapy, and one that has been demonstrated in clinical trials to be of substantial durability in its duration of effect," clinical trial investigator James F. Howard, MD, Distinguished Professor of Neuromuscular Disease, University of North Carolina School of Medicine, told NeurologyLive® at the time.

Efgartigimod Approved for Generalized Myasthenia Gravis
1. Zogenix announces US FDA acceptance for priority review of supplemental new drug application for fintepla (fenfluramine) for the treatment of seizures associated with Lennox-Gastaut syndrome. News release. Zogenix. December 1, 2021. Accessed January 12, 2022. https://finance.yahoo.com/news/zogenix-announces-u-fda-acceptance-130000785.html
2. Cognetivity Neurosciences granted FDA clearance for commercial distribution of ICA throughout US healthcare market. News release. Cognetivity Neurosciences. October 20, 2021. Accessed January 12, 2022. https://cognetivity.com/cognetivity-neurosciences-granted-fda-clearance-for-commercial-distribution-of-ica-throughout-us-healthcare-market/
3. TG Therapeutics announces FDA acceptance of biologics license application for ublituximab as a treatment for patients with relapsing forms of multiple sclerosis. News release. TG Therapeutics. December 14, 2021. Accessed Januart 12, 2022. https://www.biospace.com/article/releases/tg-therapeutics-announces-fda-acceptance-of-biologics-license-application-for-ublituximab-as-a-treatment-for-patients-with-relapsing-forms-of-multiple-sclerosis/
4. FDA Approves New Treatment for Myasthenia Gravis. News release. FDA. December 17, 2021. Accessed January 12, 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-myasthenia-gravis
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