FDA Action Update, July 2023: Approvals, Clearances, and Requests

The FDA was busy in July 2023, making a number of decisions on potential new therapeutic agents including issuing a complete response letter, granting traditional approval, and clearing a clinical trial, among other actions.

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.

FDA Issues Complete Response Letter to Amneal for Parkinson Agent IPX203

Early in the month, on July 3, the FDA issued a complete response letter (CRL) to Amneal Pharmaceuticals for IPX203, an oral formulation of carbidopa/levodopa (CD/LD) extended-release capsules designed for the treatment of Parkinson disease. The reasons behind the decision were not based on efficacy or manufacturing for the agent, but rather established safety for an ingredient of the therapy.¹

In its release, the agency noted that the pharmcokinectic data for the safety of one ingredient, levodopa, was sufficient; however, it felt it needed additional data for the second ingredient, carbidopa. Amneal plans to work closely with the FDA to address the comments and align on the best path forward.

"We are committed to advancing IPX203 for Parkinson’s disease, which has been developed to provide a longer duration of therapeutic benefit than existing formulations with fewer doses,” Chirag and Chintu Patel, co-chief executive officers at Amneal, said in a statement.¹ “We plan to work closely with the FDA to address the agency’s feedback and we remain confident in bringing this new treatment to Parkinson’s patients as soon as possible.”

FDA Grants Traditional Approval to Lecanemab as Therapy for Early-Stage Alzheimer Disease

On June 9, the FDA granted traditional approval to Eisai’s Alzheimer disease (AD) medication lecanemab (Leqembi), a major step in enabling broader access and coverage of the therapy to the aging population. It originally received FDA approval under the accelerated approval pathway in January.²

The decision was expected after a recent meeting between the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee in which the panel voted unanimously in favor of the agent. The committee was asked to vote on whether the results of the phase 3 Clarity AD trial (NCT03887455), the confirmatory study, verified the clinical benefit of lecanemab for the treatment of AD.³ Lecanemab is only the second early AD treatment that has received FDA approval in the past 20 years following the contentious approval of aducanumab (Aduhelm; Eisai/Biogen) in 2021, which remains under conditional approval.

"Today’s action is the first verification that a drug targeting the underlying disease process of Alzheimer’s disease has shown clinical benefit in this devastating disease," Teresa Buracchio, acting director of the Office of Neuroscience, Center for Drug Evaluation and Research, FDA, said in a statement.² "This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer’s disease."

FDA Approves Image Guided Programming Software for Vercise Deep Brain Stimulation Systems

A few days after, on July 11, the FDA approved Boston Scientific's Vercise Neural Navigator 5 Software, a software program that is used to set and adjust stimulation parameters for the Vercise Deep Brain Stimulation (DBS) Systems, a previously approved therapeutic approach for patients with Parkinson disease (PD) and essential tremor.⁴

The newly approved software, with STIMVIEW XT Technology, is the latest addition to the fully integrated portfolio of image guided programming solutions for Boston Scientific DBS Systems. With the Vercise Neural Navigator, clinicians are able to view an enhanced user interface that displays patient data in a simplified format and allows for access to advanced settings for increased therapy delivery.

"The ability to see the precise placement of DBS Systems enables us to target therapy to meet individual needs,” Mustafa Saad Siddiqui, MD, professor of neurology and neurosurgery at Wake Forest School of Medicine and medical director of the DBS program at Atrium Health Wake Forest Baptist, said in a statement.⁴ "The new features in the Vercise Neural Navigator 5 are expected to help further reduce the time needed to adjust stimulation and minimize potential side effects, allowing us to optimize treatment benefits for each patient."

FDA Clears NS-050/NCNP-03 for Phase 1/2 Trial of Duchenne Muscular Dystrophy

A couple of days after, on July 15, the FDA cleared the start of a phase 1/2 study assessing NS-050/NCNP-03, NS Pharma’s investigational candidate for patients with Duchenne muscular dystrophy (DMD) amenable to exon 50 skipping therapy.⁵ Clinical trial enrollment for the trial in the United States is planned to start in the second half of 2023 and will be by NS Pharma's parent company, Nippon Shinyaku.

In the planned trial, patients with DMD will be assessed on dystrophin production, muscle strength, mobility, and functional exercise capacity. Once the trial is ready to begin the enrollment of participants, NS Pharma noted that additional information will then be provided. In addition to NS-050/NCNP-03, Nippon Shinyaku has 3 investigational exon skipping candidates in different stages of preclinical development for DMD.

"This is the second trial clearance from the FDA that NS Pharma has received this year and marks the third candidate from our R&D pipeline to begin clinical trials in Duchenne," Tsugio Tanaka, MSc, president at NS Pharma, said in a statement.⁵ "Our rapid development plans reflect the urgent needs of the Duchenne community and our commitment to extending the impact of our exon skipping technology."

Imperative’s Complete Stroke Solution Expands With FDA-Cleared Large Distal Platform

On July 25, the FDA granted 510(k) clearance to Imperative Care’s Zoom 88 Large Distal Platform (LDP) Support, a catheter-based approach to improving reperfusion in poststroke patients, further expanding the company’s complete stroke system.⁶

Zoom 88 Support is a part of the company’s Zoom Stroke Solution, which includes the existing Zoom 88 LDP and the Zoom RDL Access Platform, as well as Zoom Aspiration Catheters and Zoom POD and Pump. The newly added feature is designed to maintain the capability of 0.88” intracranial access with added stability, giving clinicians even more control over their thrombectomy procedures.

"Zoom 88 Support was purposefully designed to meet the needs of more patients and deliver on the feedback we’ve received from physicians,” Ariel Sutton, general manager of Imperative Care’s Stroke business, said in a statement.⁶ "Zoom 88 Support provides the same benefits of intracranial access found with Zoom 88 LDP, with the ability to treat more proximal or tandem occlusions. With the introduction of Zoom 88 Support, we continue to grow our patient-first portfolio to bring the benefits of large-bore intracranial access to more people."

REFERENCES
1. Amneal Receives U.S. FDA Complete Response Letter for IPX203. News release. Amneal Pharmaceuticals. July 3, 2023. Accessed August 9, 2023. https://investors.amneal.com/news/press-releases/press-release-details/2023/Amneal-Receives-U.S.-FDA-Complete-Response-Letter-for-IPX203/default.aspx
2. FDA Converts Novel Alzheimer’s Disease Treatment to Traditional Approval. FDA. News release. July 6, 2023. Accessed August 9, 2023. https://www.fda.gov/news-events/press-announcements/fda-converts-novel-alzheimers-disease-treatment-traditional-approval
3. FDA Peripheral and Central Nervous System Drugs Advisory Committee Meeting. June 9, 2023. Accessed August 9, 2023. https://www.fda.gov/advisory-committees/advisory-committee-calendar/updated-information-june-9-2023-meeting-peripheral-and-central-nervous-system-drugs-advisory
4. Boston Scientific receives FDA approval for the next innovation in image guided programming software for deep brain stimulation. News release. July 11, 2023. August 9, 2023. https://news.bostonscientific.com/Boston-Scientific-Receives-FDA-ApprovNext-Innovation-Image-Guided-Programming-Software-Deep-Brain-Stimulation#assets_all
5. NS Pharma Announces FDA Clearance to Initiate a Phase I/II Study for NS-050/NCNP-03, an Exon 50 Skipping Candidate for the Treatment of Duchenne Muscular Dystrophy. News Release. NS Pharma. Published June 15, 2023. Accessed August 9, 2023. https://www.prnewswire.com/news-releases/ns-pharma-announces-fda-clearance-to-initiate-a-phase-iii-study-for-ns-050ncnp-03-an-exon-50-skipping-candidate-for-the-treatment-of-duchenne-muscular-dystrophy-301851575.html
6. Imperative Care announces FDA clearance of Zoom 88 Support, designed to bring the benefits of intracranial access to more stroke patients. News release. July 25, 2023. Accessed August 9, 2023. https://www.biospace.com/article/releases/imperative-care-announces-fda-clearance-of-zoom-88-support-designed-to-bring-the-benefits-of-intracranial-access-to-more-stroke-patients/