Here is information on the latest US FDA approvals for the week of February 5–9, 2024
Eplontersen for ATTR-CM
Eplontersen (Wainua) has been granted Fast Track designation by the FDA for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults. Eplontersen, a Ligand-Conjugated Antisense (LICA) medicine, inhibits the production of transthyretin (TTR) protein. It is under investigation in the CARDIO-TTRansform study for both hereditary or wild-type ATTR-CM. Eplontersen was approved in the US, in December 2023, for treating the polyneuropathy of ATTR. ATTR-CM is an underdiagnosed and potentially fatal disease characterized by the accumulation of misfolded TTR protein in the cardiac muscle, leading to heart failure and death within three to five years of onset. ATTR-CM includes both genetic and wild-type forms, affecting an estimated 300,000 to 500,000 patients worldwide. Eplontersen is being developed and commercialized under a global agreement between Ionis Pharmaceuticals and AstraZeneca.
https://ir.ionispharma.com/news-releases/news-release-details/eplontersen-granted-us-fda-fast-track-designation-patients
Nivolumab for NSCLC
The FDA has accepted two supplemental biologics applications (sBLAs) for nivolumab (Opdivo)-based regimens for the treatment of resectable stage 2A to 3B non-small cell lung cancer (NSCLC). Nivolumab is a PD-1 immune checkpoint inhibitor first approved in 2014. The sBLA submissions were based on positive outcomes from the CheckMate-77T trial, which evaluated nivolumab in combination with chemotherapy followed by surgery, and adjuvant nivolumab as perioperative treatment for resectable NSCLC. This trial demonstrated improved event-free survival, pathological complete response and major pathological response. Nivolumab is already indicated across various stages of lung cancer and other cancers as both a neoadjuvant and adjuvant agent. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of October 8, 2024, for the current sBLAs. Nivolumab is manufactured by Bristol-Myers Squibb.
https://news.bms.com/news/corporate-financial/2024/Bristol-Myers-Squibb-Announces-Acceptance-of-U.S.-and-EU-Regulatory-Filings-for-Neoadjuvant-Opdivo-nivolumab-and-Chemotherapy-Followed-by-Surgery-and-Adjuvant-Opdivo-in-Resectable-Non-Small-Cell-Lung-Cancer/default.aspx
RSV Vaccine for Adults Aged 50 to 59
The FDA has accepted GSK’s application for priority review for extended use of its respiratory syncytial virus (RSV) vaccine Arexvy in adults ages 50 to 59 at higher risk for complications. Arexvy is already approved in the US for adults aged 60 and over for the prevention of lower respiratory tract disease caused by RSV. Approximately 177,000 adults aged 65 and older are hospitalized annually due to RSV. The sBLA for Arexvy’s expanded indication is supported by results from a phase 3 trial evaluating the immune response in participants aged 50 to 59 with stable chronic diseases, as well as those without chronic conditions, compared to adults aged 60 and older. Arexvy, which was first approved in May 2023, is the inaugural RSV vaccine to receive FDA approval. The CDC’s Advisory Committee on Immunization Practices (ACIP) recommends Arexvy for individuals over the age of 60. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) date of June 7, 2024, for the additional indication in this younger age group.
https://www.gsk.com/en-gb/media/press-releases/arexvy-accepted-under-priority-review-in-us-for-the-prevention-of-rsv-disease-in-adults-aged-50-59-at-increased-risk/
Spinal Cord Stimulation System for NSBP
The FDA has approved an extended indication for Boston Scientific’s WaveWriter Spinal Cord Stimulation (SCS) Systems for the treatment of chronic low back and leg pain in individuals who have not undergone previous back surgery. This condition is often referred to as non-surgical back pain (NSBP). The approval was supported by the results from the SOLIS randomized trial, which provided one-year data showing that the WaveWriter SCS Systems met its primary endpoint of a 50% or greater reduction in pain at the three-month interval. At the one-year mark, 84% of patients using the system experienced substantial pain relief, with a pain reduction of 50% or greater. According to the manufacturer press release, first-line treatment for people with chronic back pain is usually limited to conventional medical management such as physical therapy and medication, which are not effective for many people. Effective chronic pain management may lead to improved quality of life and reduced opioid use.
https://news.bostonscientific.com/FDA-Approves-Boston-Scientific-Spinal-Cord-Stimulator-Systems-Treatment-Non-Surgical-Back-Pain
OK-101 for Neuropathic Corneal Pain
The FDA has approved an Investigational New Drug (IND) application for OK-101, marking the first time the FDA has cleared an IND specifically for the treatment of neuropathic corneal pain (NCP). NCP is recognized as an Orphan disease by the National Organization for Rare Disorders. OK-101, a lipid conjugated chemerin peptide agonist targeting the ChemR23 receptor, has demonstrated potential in reducing inflammation and pain in ocular conditions. The drug’s development includes a novel approach to enhance ocular residence time, showing promising results in a Phase 2 trial for dry eye disease (DED), which shares symptomatology with NCP. The upcoming Phase 2 clinical trial for OK-101 in NCP is set to begin in the second quarter of 2024. The approval was granted to OKYO Pharma Limited.
https://okyopharma.com/okyo-pharma-receives-fda-approval-of-ind-for-ok-101-in-neuropathic-corneal-pain/
Viz ICH Plus for Brain Bleed
The FDA has granted 510(k) clearance to Viz ICH Plus, an algorithm designed to automate the identification, labeling, and quantification of segmentable brain structures on non-contrast computed tomography (NCCT) images. This approval introduces an advanced solution for the accurate and quantifiable measurement of intracerebral hemorrhages, a condition representing up to 15% of all strokes and characterized by high morbidity and mortality rates. Viz ICH Plus, is specifically indicated for the analysis of intracranial hyperdensities, lateral ventricles, and midline shift, offering volume measurements of brain bleeds to support timely and informed treatment decisions and automates the traditionally manual process of measuring brain bleeds. Viz ICH Plus is being developed by Viz.ai.
https://www.viz.ai/news/viz-ai-receives-fda-510k-clearance-for-artificial-intelligence-algorithm-for-the-quantification-of-intracerebral-hemorrhage
Vepdegestrant for MBC
The FDA has granted Fast Track designation to vepdegestrant (ARV-471) for the treatment of adults with estrogen receptor-positive/human epidermal growth factor 2-negative (ER-positive/HER2-negative) locally advanced or metastatic breast cancer who have previously undergone endocrine-based therapy. Vepdegestrant is an oral PROteolysis Targeting Chimera (PROTAC) estrogen receptor (ER) degrader under development by Arvinas and Pfizer. The decision by the FDA is supported by data from the Phase 1/2 VERITAC trial, which has shown significant efficacy in patients with this type of breast cancer. Vepdegestrant is also being evaluated in combination with various other therapies, including palbociclib (Ibrance), in the first-line setting as part of the ongoing Phase 3 VERITAC-3 clinical trial.
https://www.pfizer.com/news/announcements/arvinas-and-pfizers-vepdegestrant-arv-471-receives-fda-fast-track-designation
VerTouch Spinal Puncture Device
The FDA has cleared VerTouch, a novel handheld imaging tool designed to improve the accuracy and consistency of spinal punctures. This device aids healthcare providers in performing spinal punctures with greater precision directly at the bedside. VerTouch leverages a proprietary spinal mapping technology generating a 2D image of lumbar spinal anatomy, helping users visualize the key landmarks of the patient’s spine to inform their decision and guide where to initiate needle placement. The traditional approach to spinal punctures relies on palpating the patient’s vertebrae and inserting the needle without visual guidance, with a rate of first-attempt failures estimated between 40% to 60%. This approval was granted to IntuiTap Medical.
https://www.businesswire.com/news/home/20240208434976/en/