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FDA Action Update, August 2024: Approvals, Designations, and Clearances
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Catch up on any of the neurology news headlines you may have missed over the course of August 2024, compiled all into one place by the NeurologyLive® team.
The FDA was busy in August 2024, making a number of decisions on potential new therapeutic agents including granting approvals, designations, and clearances.
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 Indapta Therapeutics’ IND for Cell Therapy IDP-023 in Progressive Multiple Sclerosis
Early in the month, on August 6, the FDA cleared the investigational new drug (IND) application for Indapta Therapeutics’ g-natural killer (g-NK) cell therapy IDP-023 to be assessed in a phase 1 clinical trial for the treatment of patients living with progressive multiple sclerosis (MS).1
In the trial, investigators from Stanford University and the University of California, San Francisco (UCSF), will examine the biologic effects of IDP-023 using a translational program developed in collaboration by both universities. During the study, the participants with progressive MS will be given IDP-023 in combination with the FDA approved anti-CD20 monoclonal antibody, ocrelizumab. For context, ocrelizumab remains the only approved therapy for the treatment of progressive MS.
“We are very encouraged that the FDA has cleared our phase 1 trial for the use of our g-NK cell therapy, IDP-023, for the treatment of patients with progressive MS. There is a high unmet need for these patients, with ocrelizumab (Ocrevus; Roche) being the only FDA approved therapy for patients with primary progressive MS,” Mark Frohlich, MD, CEO at Indapta, told NeurologyLive®. “G-NK cells in combination with ocrelizumab have the potential to eliminate the autoreactive T and B cells that are responsible for the disease. Given the poor prognosis of these patients, a new therapy with novel mechanisms of action has the potential to be transformative for progressive MS."
FDA Approves Carbidopa/Levodopa ER Capsules Formulation IPX203 for Parkinson Disease
About a day later, on August 7, the FDA approved Amneal Pharmaceuticals’ investigational agent IPX203, an oral formulation of carbidopa/levodopa (CD/LD) extended-release capsules, as a treatment for patients with Parkinson disease (PD), according to an announcement from the company. Marketed as Crexont, the therapy is an oral formulation CD/LD which includes immediate-release (IR) granules as well as extended-release (ER) pellets.2
The treatment is indicated for PD, PD caused by infection or inflammation of the brain, or PD-like symptoms that may result from carbon monoxide or manganese poisoning in adults. It is not recommended in combination with nonselective monoamine oxidase inhibitors, nor with other CD/LD preparations without consultation with a healthcare provider. Amneal noted its expectation is for the oral formulation to become available to patients in the United States in September 2024.
Robert A. Hauser, MD, a professor of neurology at the University of South Florida and the director of the Parkinson's Disease and Movement Disorders Center, said in a statement that "the treatment goals for people living with Parkinson’s disease include achieving a more robust duration of benefit per dose of LD, reducing OFF time, and simplifying dosing regimens. Crexont’s longer Good ON time per day and per dose represents a substantial advancement in managing motor symptoms and maintaining more consistent therapeutic effects, which is very encouraging for both patients and the Parkinson’s community."2
FDA Approves Medtronic’s Deep Brain Stimulation Technology for Asleep Capabilities
A couple of days later, on August 12, the FDA granted approval to Medtronic’s deep brain stimulation (DBS) technology to be used while a patient is under general anesthesia, or asleep, in the context of treating PD or essential tremor.3
Typically, the standard established DBS procedure is based on awake surgery with intraoperative microelectrode recording associated with clinical testing to ensure good positioning of the lead; however, this procedure is sometimes perceived as uncomfortable for patients who are unable to tolerate awake surgery with extensive time off their medication. Ultimately, the FDA’s decision to approve Medtronic’s DBS for asleep purposes may provide greater treatment opportunities to a significant portion of potential candidates for surgery.
"This is a significant advancement in our surgical offering, providing another safe and effective option for patients considering DBS." Amaza Reitmeier, vice president and general manager of Brain Modulation, a portion of the NeuroScience Portfolio at Medtronic, said in a statement.3 "This approval underscores our dedication to continuous innovation to address the needs of patients and healthcare providers."
FDA Fast Tracks Autologous Treg Therapy ABA-101 for Progressive Multiple Sclerosis
About a week later, on August 22, the FDA granted fast track designation to Abata Therapeutics’ autologous regulatory T cell (Treg) therapy ABA-101 for the treatment of patients with progressive MS.4
In animal models, the agent has shown a tolerable safety profile with antigen-dependent Treg functionality, anti-inflammatory cytokine production, suppression of the production of inflammatory cytokines, and therapeutic effect. ABA-101 was specifically designed for patients with progressive MS who have imaging evidence of ongoing inflammatory tissue injury and are HLA-DRB1*15:01 positive-an estimated patient group of about 45,000 in the US today.
"There are no effective treatments for progressive MS, and rapidly advancing new therapies is critical for patients and their families. We are very pleased that the FDA granted us Fast Track designation as it will enable us to expedite our efforts to bring ABA-101 to patients," Samantha Singer, MS, MBA, president and chief executive officer at Abata, said in a statement.4 "We are focused on initiating our Phase 1 study this year in patients and evaluating the potential impact of this important new therapy."
FDA Clears Clearmind Biomedical’s Neuroblade System for Minimally Invasive Neurosurgery
A couple of days later, on August 26, the FDA granted 510(k) clearance to Clearmind Biomedical’s Neuroblade System, which allows for minimally invasive procedures with integrated features such as visualization, illumination, irrigation, suction, coagulation, and powered debridement. The company also reported a successful completion of the first surgery using the Neuroblade in the United States, which was performed by Christopher Kellner, MD, a cerebrovascular neurosurgeon in the department of neurology at Mount Sinai Hospital in New York.5
The Neuroblade System is composed of 3 components including the Neuroblade, the Neuropad, and Clearpath. The Neuroblade is a single-use multifunctional neuroendoscope while the Neuropad is a reusable medical-grade tablet, and Clearpath is a disposable transparent access sheath. This system was developed to enhance the overall efficiency and outcomes of patients who undergo minimally invasive neurosurgical procedures.
"The Neuroblade system offers a valuable new tool for the surgical management of intracerebral hemorrhage. Its minimally invasive design streamlines the operating room setup and integrates essential functions into a single handpiece, potentially improving patient outcomes. Neuroblade system represents a new class of technology that should simplify neurosurgical procedures and provide patients a chance at more favorable outcomes,” Kellner said in a statement.5
FDA Grants Fast Track Designation to PI-2620 Tau-PET Diagnostic Scan for Neurodegenerative Diseases
A couple of days later, on August 28, the FDA granted fast track designation to [18F]PI-2620, a tau positron emission tomography (PET) diagnostic, for several neurodegenerative diseases, including Alzheimer disease (AD), progressive supranuclear palsy (PSP), and corticobasal degeneration (CBD).6 This designation highlights the significant potential of [18F]PI-2620, developed in collaboration with AC Immune SA and Life Molecular Imaging, to improve diagnosis of these 3 neurodegenerative diseases.
Currently, [18F]PI-2620 is being tested in a phase 3 clinical development program for detecting tau pathology in patients with AD and is also being assessed in other neurodegenerative diseases such as PSP and CBD. Prior research has shown that the compound has had robust brain uptake and fast wash-out in non-target regions, a broad imaging window between 30- and 90-minutes post-injection for AD as well as successful reproducibility between test and retest scans. The absence of significant off-target binding allows PI-2620 to identify and quantify early tau deposition in the brain, which is noted by the companies as a hallmark of neurodegenerative diseases.
“Fast Track Designation for PI-2620 is an important reflection of its potential to accurately diagnose Alzheimer’s and other neurodegenerative diseases. Early diagnosis of these conditions will be key for effective treatment before irreversible damage occurs, and is an essential element in our goal of achieving precision prevention,” Andrea Pfeifer, PhD, CEO at AC Immune SA, commented in a statement.6 “The FDA has previously granted Fast Track status to two of our active immunotherapies in phase 2 development, ACI-35.030 and ACI-24.060, which target phospho-Tau and Abeta, respectively. The designation for PI-2620 is a further recognition of AC Immune’s drug discovery and development platform and of how we, together with our partners, continue to drive innovation.”
FDA Grants Priority Review to New Drug Application of Neurofibromatosis Agent Mirdametinib
On the same day, August 28, the FDA accepted and granted priority review to SpringWorks Therapeutics’ new drug application (NDA) for mirdametinib, an investigational MEK inhibitor for the treatment of adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). The agency has scheduled a PDUFA action date of February 28, 2025, and stated that it does not have current plans to hold an advisory committee meeting to discuss the application.7
In the update, the company also announced that the European Medicines Agency validated the Marketing Authorization Application (MAA) for mirdametinib for the treatment of adult and pediatric patients with NF1-PN. Both submissions for the NDA and the MAA included data from the pivotal phase 2b ReNeu trial (NCT03962543), a multicenter, open-label study that featured 114 patients with NF1 PN (58 adult and 56 pediatric) who received mirdametinib for up to 24 months.
"We look forward to working with regulatory authorities on their reviews of our applications for mirdametinib, both in the U.S. and Europe. We are also focused on sharing additional data from the phase 2b ReNeu trial at upcoming medical meetings and expect the data to be published in a peer-reviewed publication later this year. In parallel, we are advancing our preparations in anticipation of a potential launch in early 2025," Jim Cassidy, MD, PhD, chief medical officer at SpringWorks Therapeutics, told NeurologyLive®.
REFERENCES
1. Indapta Therapeutics Announces FDA Clearance of IND for Phase 1 Trial of IDP-023 for Progressive Multiple Sclerosis. News Release. Indapta Therapeutics. Published August 6, 2024. Accessed September 9, 2024. https://www.businesswire.com/news/home/20240806112913/en/Indapta-Therapeutics-Announces-FDA-Clearance-of-IND-for-Phase-1-Trial-of-IDP-023-for-Progressive-Multiple-Sclerosis
2. Amneal Receives U.S. FDA Approval for IPX203 for Treatment of Parkinson’s Disease to Be Launched as CREXONT® (Carbidopa and Levodopa) Extended-Release Capsules. News release. Amneal. August 7, 2024. Accessed September 9, 2024. https://investors.amneal.com/news/press-releases/press-release-details/2024/Amneal-Receives-U.S.-FDA-Approval-for-IPX203-for-Treatment-of-Parkinsons-Disease-to-Be-Launched-as-CREXONT-Carbidopa-and-Levodopa-Extended-Release-Capsules/default.aspx
3. Medtronic receives landmark FDA approval for asleep deep brain stimulation surgery. News release. Medtronic. August 12, 2024. Accessed September 9, 2024. https://news.medtronic.com/2024-08-12-Medtronic-receives-landmark-FDA-approval-for-Asleep-Deep-Brain-Stimulation-surgery
4. Abata Therapeutics receives FDA fast track designation for ABA-101 for the treatment of progressive multiple sclerosis. News release. August 22, 2024. Accessed September 9, 2024. https://www.globenewswire.com/news-release/2024/08/22/2934105/0/en/Abata-Therapeutics-Receives-FDA-Fast-Track-Designation-for-ABA-101-for-the-Treatment-of-Progressive-Multiple-Sclerosis.html
5. Clearmind Biomedical Announces FDA Clearance and First U.S. Surgery of the Neuroblade System for Minimally Invasive Neurosurgery. News Release. Published August 26, 2024. Accessed September 9, 2024. https://prnmedia.prnewswire.com/news-releases/clearmind-biomedical-announces-fda-clearance-and-first-us-surgery-of-the-neuroblade-system-for-minimally-invasive-neurosurgery-302227315.html
6. AC Immune’s PI-2620 Tau-PET Diagnostic in Phase 3 Receives Fast Track Designation in Three Neurodegenerative Conditions. News Release. AC Immune SA. News Release. August 28, 2024. Accessed September 9, 2024. https://ir.acimmune.com/news-releases/news-release-details/ac-immunes-pi-2620-tau-pet-diagnostic-phase-3-receives-fast
7. FDA Grants Priority Review to SpringWorks Therapeutics’ New Drug Application for Mirdametinib for the Treatment of Adults and Children with NF1-PN. News Release. SpringWorks Therapeutics. August 28, 2024. Accessed September 9, 2024. https://ir.springworkstx.com/news-releases/news-release-details/fda-grants-priority-review-springworks-therapeutics-new-drug
