FDA Action Update, December 2023: Approval and Clearances

The FDA was busy in December 2023, making a number of decisions on potential new therapeutic agents including granting clearances and granting an approval.

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 details and information about each update.

FDA Clears Vivos CARE Appliance for Severe Obstructive Sleep Apnea

Earlier in the month, on December 4, the FDA granted 510(k) clearance to Vivos Therapeutics for its removable CARE (Complete Airway Repositioning and/or Expansion) oral appliances as a treatment option for patients with severe cases of obstructive sleep apnea (OSA). The newly approved devices, which include the flagship DNA (Daytime-Nighttime Appliance) oral appliance, the mRNA oral appliance, and the mmRNA oral appliance, give patients a new alternative to continuous positive airway pressure (CPAP) or surgical neurostimulation.¹

The latest clearance comes less than a year after the FDA granted Vivos 510(k) clearance for the DNA oral appliance to treat mild to moderate OSA. In addition, it represented the first marketed oral appliance to treat moderate and severe OSA in adults, 18 years of age and older along with PAP and/or myofunctional therapy, as needed.

"This achievement is a pivotal milestone for Vivos, and elevates our proven treatment options right into the mainstream of sleep medicine," Kirk Huntsman, chairman and chief executive officer at Vivos, said in a statement.¹ "It is even more important for the millions of severe OSA patients who are desperate for an effective alternative treatment. Before this, severe OSA patients’ only realistic treatment options were CPAP, neurostimulation implants or other invasive surgeries. Today, they have what we believe is a far more desirable option that is very affordable and doesn’t require surgery or a lifetime of nightly use and intervention."

FDA Clears NeuroOne’s OneRF Ablation System for Neurosurgerical Procedures

A week later, on December 11, NeuroOne announced that the FDA granted 510(k) clearance for its OneRF Ablation System, a stereoelectroencephalogram (sEEG)-guided tool used in neurosurgery to record electrical activity and ablate nervous tissue under temperature-controlled environments. The company is targeting a launch in early 2024.²

OneRF includes a customized radio frequency (RF) generator with real-time temperature monitoring capabilities, allowing for greater control during ablation procedures for patients with epilepsy, Parkinson disease, dystonia, and essential tremor, among others. The combination system, comprised of both diagnostic and therapeutic capabilities, is believed to be a safer and more cost-efficient clinical option for patients, with lower number of invasive procedures and hospital stays.

"When NeuroOne was founded, one of our ambitious goals was to be the first to develop and commercialize thin-film electrodes capable of performing both diagnostic and therapeutic functions and offer a better methodology to treat patients suffering from a variety of neurological conditions,” Dave Rosa, chief executive officer, NeuroOne, said in a statement.² "We believe we have the potential to dramatically change the way these surgeries are performed so that patients may only need to go through surgery once for both diagnostic and therapeutic procedures."

FDA Approves Eplontersen for Polyneuropathy of Hereditary Transthyretin Amyloidosis

Later in the month, on December 21, the FDA approved eplontersen (Ionis, AstraZeneca), a ligand-conjugated antisense oligonucleotide, for the treatment of adult patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Approved under the market name Wainua, the therapy is the only approved treatment for ATTRv-PN that can be self-administered via an autoinjector.³

Data from the interim analysis of the phase 3 NEURO-TTRansform trial (NCT04136184) was the basis for the approval, with results indicating that eplontersen reduced serum transthyretin (TTR) at 35 weeks. Presented the results at the 2022 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting, the least square mean percent reduction of TTR was 81.2% after 35 weeks of treatment with eplontersen, compared with a 14.8% reduction for the placebo group (P <.0001). Also of note, the treatment resulted in reductions in neuropathy impairment and improvements in quality of life compared with placebo.

"This is wonderful news for patients. Another treatment option to combat this devastating disease and give patients an improved quality of life," principal investigator Sami Khella, MD, chief, department of neurology at Penn Presbyterian Medical Center and professor of clinical neurology at the Perelman School of Medicine at the University of Pennsylvania School of Medicine, told NeurologyLive®.

REFERENCES
1. Vivos Therapeutics receives first ever FDA 510(k) clearance for oral device treatment of severe obstructive sleep apnea. News release. Vivos Therapeutics. November 29, 2023. Accessed January 2, 2024. https://vivos.com/vivos-therapeutics-receives-first-ever-fda-510k-clearance-for-oral-device-treatment-of-severe-obstructive-sleep-apnea/
2. NeuroOne receives FDA 510(k) clearance to market its OneRF Ablation System. News release. NeuroOne. December 11,2023. Accessed January 2, 2024. https://www.globenewswire.com/news-release/2023/12/11/2793821/0/en/NeuroOne-Receives-FDA-510-k-Clearance-to-Market-its-OneRF-Ablation-System.html
3. WAINUA™ (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis. News Release. Ionis Pharmaceuticals. Published December 21, 2023. Accessed January 2, 2024. https://www.prnewswire.com/news-releases/wainua-eplontersen-granted-regulatory-approval-in-the-us-for-the-treatment-of-adults-with-polyneuropathy-of-hereditary-transthyretin-mediated-amyloidosis-302021385.html