FDA Action Update, December 2022: Approvals, Submissions, and Requests


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.

The FDA took a handful of actions in December 2022, including greenlighting several new clinical tools, a Type A Meeting request, and a trial recruitment pause, among others.

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 and holds.

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

Roche Gets FDA Clearance for CSF Beta-Amyloid and Phospho-Tau Alzheimer Disease Assays

On December 8, Roche announced that the company’s Alzheimer disease (AD) cerebrospinal fluid Elecsys assays—including beta-amyloid1-42 CSF II, known as Abeta42; and phospho-Tau181P, known as pTau181—have been approved by the FDA for use with the cobas fully automated immunoassay analyzers.1

The biomarkers are hallmarks of AD pathology, and the assays utilize their ratio (pTau181/Abeta42), which is in line with a negative beta‑amyloid PET scan if the result is less than or equal to the cutoff (negative) and with a positive beta‑amyloid PET scan if the result is above the ratio cutoff (positive). Roche noted in the announcement that its AD assays “achieve 90% concordance” with amyloid PET scans.

“Globally, up to 75% of people living with Alzheimer’s disease have not been diagnosed, and those who have often report a long and complicated process," Thomas Schinecker, PhD, CEO of Roche Diagnostics, said in a statement. "The Elecsys AD CSF assays have the potential to guide more people with suspected Alzheimer’s disease toward a diagnosis than ever before. As we are starting to see exciting results for new potential Alzheimer’s treatments, reliable tests that have been clinically validated will be critical in ensuring the right patients are identified and able to benefit from them.”

Roche Gets FDA Clearance for CSF Beta-Amyloid and Phospho-Tau Alzheimer Disease Assays

BrainStorm Submits Type A Meeting Request Regarding NurOwn ALS Treatment

About a month after the agency sent a Refusal to File letter, on December 12, BrainStorm Cell Therapeutics announced it had submitted a Type A meeting request to discuss the contents of the letter, which was in response to the biologics license application (BLA) of NurOwn, the company’s investigational amyotrophic lateral sclerosis (ALS) therapy.2

The Type A meeting is expected to occur within 30 days of the FDA’s receipt of the meeting request. In that meeting, BrainStorm intends to discuss a path to an FDA advisory committee meeting.

"Participating in a Type A meeting will be an important next step towards enabling NurOwn's advancement through the regulatory process," Chaim Lebovits, chief executive officer, BrainStorm, said in a statement. "The extensive briefing package submitted with our request contains a comprehensive strategy to fully address the CMC matters raised in the refusal to file letter. We anticipate achieving quick alignment with the FDA on the CMC strategy and expect that its execution will be straightforward. We, therefore, anticipate a Type A meeting focused primarily on discussing how we can secure an Advisory Committee Meeting, which we believe will be a critical step on NurOwn's path towards approval as an ALS therapy."

BrainStorm Submits Type A Meeting Request Regarding NurOwn ALS Treatment

FDA Pauses IND Submission for Duchenne Therapy ENTR-601-44

Later in the month, on December 19, Entrada Therapeutics announced that the FDA placed a clinical hold on its investigational new drug application for its investigational Duchenne muscular dystrophy (DMD) treatment, ENTR-601-44, an exon 44 skipping oligonucleotide developed with Entrada’s Endosomal Escape Vehicle (EEV) platform.3

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. "The clinical hold on our ENTR-601-44 program is disappointing and we will work to address the FDA’s concerns regarding the IND. There are no approved Duchenne therapies for people with exon 44 skippable mutations and we are eager to resolve this hold and continue down the treatment development pathway,” said Dipal Doshi, president and CEO of Entrada Therapeutics, said in a statement.

FDA Pauses IND Submission for Duchenne Therapy ENTR-601-44

FDA Approves Insightec’s Exablate Neuro to Treat Second Side of Essential Tremor

On December 20, the FDA 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 (ET).4

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 in 2018. The new approval allows appropriate patients to have their second side treated at least 9 months after treatment of the first side.

"This FDA approval is a very important milestone for us and demonstrates our unwavering commitment to expanding the treatment options for people living with essential tremor. It's very common for patients who've benefited from tremor reduction from the first side treatment to ask about having the second side treated. This approval paves the way for them to do that," Maurice R. Ferre, MD, CEO and chairman of the Board of Directors, Insightec, said in a statement.

FDA Approves Insightec’s Exablate Neuro to Treat Second Side of Essential Tremor

Anti-CD20 Therapy Ublituximab Gains FDA Approval for Relapsing Multiple Sclerosis

Finally, on December 28, the FDA approved TG Therapeutics’ investigational glycoengineered monoclonal antibody ublituximab (Briumvi) for the treatment of relapsing forms of multiple sclerosis (MS). The therapy is administered in a 1-hour infusion, twice yearly following the starting dose, and is anticipated to become commercially available in the first quarter of 2023.5

Ublituximab, an agent designed to target a unique epitope on CD20-expressing B-cells, was approved based on results from the phase 3 ULTIMATE 1 and 2 trials (NCT03277261; NCT03277248). These trials, which featured 1094 patients with relapsing MS across 10 countries, showed the superior efficacy and safety of ublituximab in comparison with teriflunomide (Aubagio; Sanofi), a relatively newer agent that received FDA approval in 2009. Both trials were conducted under a special protocol assessment established with the FDA.

"Today’s FDA approval marks an exciting day for everyone touched by MS and everyone that has worked on the development of Briumvi. We believe in the importance of treatment alternatives for patients and believe the profile of Briumvi offers unique attributes to patients and physicians alike. We have built a strong commercial team with deep knowledge of the MS landscape and look forward to launching in Q1 2023,” Michael S. Weiss, chairman and CEO of TG Therapeutics, said in a statement. “We want to thank the patients and their families, the clinical investigators and their teams, and our advisors for their support and participation in our trials, and for helping us get to this point. We remain committed to the patients we serve and providing seamless access to Briumvi once launched.”

Anti-CD20 Therapy Ublituximab Gains FDA Approval for Relapsing Multiple Sclerosis
1. Roche Alzheimer’s disease Cerebrospinal Fluid (CSF) assays receive FDA clearance, supporting more accurate and timely diagnosis. News release. Roche. December 8, 2022. Accessed January 3, 2023. https://diagnostics.roche.com/us/en/news-listing/2022/roche-alzheimers-disease-cerebrospinal-fluid-assays-receive-fda-clearance.html
2. BrainStorm Cell Therapeutics submits Type A meeting request to US Food and Drug Adminstration. News release. BrainStorm Cell Therapeutics. December 12, 2022. Accessed January 3, 2023. https://www.prnewswire.com/il/news-releases/brainstorm-cell-therapeutics-submits-type-a-meeting-request-to-us-food-and-drug-administration-301700042.html
3. Entrada Therapeutics announces clinical hold on IND application for ENTR-601-44 in Duchenne muscular dystrophy. News release. December 19, 2022. Accessed January 3, 2023. https://www.biospace.com/article/releases/entrada-therapeutics-announces-clinical-hold-on-ind-application-for-entr-601-44-in-duchenne-muscular-dystrophy/
4. Insightec Recieves FDA Approval to Treat Essential Tremor Patients’ Second Side, Expanding the Total Available Market in the United States. News Release. Insightec. December 20, 2022. Accessed January 3, 2023. https://www.biospace.com/article/releases/insightec-receives-fda-approval-to-treat-essential-tremor-patients-second-side-expanding-total-available-market-in-united-states/?s=74
5. TG Therapeutics Announces FDA Approval of BRIUMVI™ (ublituximab-xiiy). News release. TG Therapeutics. December 28, 2022. Accessed January 3, 2023. https://ir.tgtherapeutics.com/news-releases/news-release-details/tg-therapeutics-announces-fda-approval-briumvitm-ublituximab
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