FDA Grants Priority Review to Luspatercept for First-line Treatment of Anemia in Lower-risk MDS
The FDA has granted priority review to a supplemental biologics license application (sBLA) seeking to expand the current indication of luspatercept-aamt (Reblozyl) to include treatment of anemia in patients with very low- to intermediate-risk MDS who have not previously received erythropoiesis-stimulating agents (ESAs) and may require red blood cell (RBC) transfusions. Furthermore, the European Medicines Agency (EMA) has validated luspatercept’s type II variation application in the same indication.
Data from the phase III COMMANDS trial (NCT03682536) showed that luspatercept demonstrated a statistically significant and clinically meaningful improvement vs epoetin alfa in RBC transfusion independence of 12 weeks or more with a concurrent hemoglobin increase of at least 1.5 g/dL in patients with very low-, low-, or intermediate-risk MDS requiring RBC transfusions. COMMANDS’ detailed findings will be presented at a forthcoming medical convention. Under the Prescription Drug User Fee Act, the FDA has set a target action date of August 28, 2023. After validating the application, the EMA will begin its centralized review process.
“Initial treatment options for [patients with] very low- to intermediate-risk MDS, including ESAs, can alleviate anemia in some patients, but others will either not respond or become resistant to therapy, and additional therapy options have remained urgently needed,” said Noah Berkowitz, MD, Ph.D., senior vice president, Haematology Development, Bristol Myers Squibb. “According to the findings of the COMMANDS study, [luspatercept] significantly improved transfusion independence and hemoglobin levels when compared to the ESA therapy, epoetin alfa.” [Luspatercept] is an essential treatment option for anemia in patients with transfusion-dependent, lower-risk MDS who have failed ESA, and we look forward to working with the FDA and EMA to expand its potential usage as a first-line therapy in eligible patients.”
The FDA approved luspatercept in November 2019 for the treatment of anemia in adult patients with -thalassemia who require regular RBC transfusions. Furthermore, the FDA approved the agent in April 2020 for the treatment of anemia in adult patients with very low- to intermediate-risk MDS with ring sideroblasts or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis requiring 2 or more RBC units over 8 weeks.
FDA Approves Risperidone Extended-Release Injectable Suspension for Schizophrenia
Teva Pharmaceuticals, a subsidiary of Teva Pharmaceutical Industries Ltd. in the United States, and MedinCell announced that the FDA has authorized UZEDY (risperidone) extended-release injectable solution for the treatment of schizophrenia in adults. UZEDY is the first subcutaneous, long-acting risperidone formulation to use SteadyTeq, a MedinCell-developed copolymer technology that regulates risperidone release. A single dose produces therapeutic blood concentrations in 6-24 hours.
“UZEDY embodies Teva’s commitment to bringing innovative advances to patients and to providing people living with schizophrenia with an important new treatment option that was designed to address certain treatment challenges and may decrease the risk of relapse,” said Teva President and CEO Richard Francis. “The approval of UZEDY is the result of a collaborative effort by Teva and MedinCell to bring this important treatment to market.” This achievement demonstrates our strong biopharmaceutical pipeline of innovative medicines, which aims to help more people living with mental health disorders and neurological diseases in the coming years.”
Approximately 80% of patients with schizophrenia experience multiple relapses during the first five years of treatment, most commonly due to poor adherence to oral antipsychotic treatment. Each relapse poses a biological risk of function loss, treatment resistance, and alterations in brain morphology.
“The approval of the first product formulated with our technology is a watershed moment for MedinCell and the many patients who will benefit,” stated MedinCell CEO Christophe Douat. “We are committed to assisting patients by providing innovative therapy options.” It has been a lovely experience with Teva, an ideal partner for realizing UZEDY’s full potential. The commercialization of our technology signals the beginning of an exciting new era for MedinCell, and we are tremendously proud to share this very unique event with all of our workers and stockholders.”
Depending on the dosage level, the wholesale acquisition cost of UZEDY ranges from $1,232 to $3,080 per month. Individual patient costs are expected to be lower than WAC because WAC does not account for any further rebates or discounts that may apply. Teva is dedicated to assisting patients who have been prescribed UZEDY in obtaining their medication and offers patient support specialists to aid with access and reimbursement, prescription pull-through, and patient assistance. Out-of-pocket cost savings may vary depending on the patient’s insurance provider and eligibility for the co-pay assistance program.
FDA Grants Accelerated Approval for QALSODY™ (tofersen) for SOD1-ALS
Biogen Inc. announced that the FDA has approved QALSODY (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in people with a mutation in the superoxide dismutase 1 (SOD1) gene. This indication was granted accelerated approval based on a decrease in plasma neurofilament light chain (NfL) levels reported in patients treated with QALSODY. Continued approval for this indication may be predicated on the clinical benefit being demonstrated in the confirmatory trial(s). The confirmatory trial will be the ongoing Phase III ATLAS trial of tofersen in patients with presymptomatic SOD1-ALS.
“For more than a decade, Biogen has been steadfast in our commitment to pursuing ALS treatments, and I want to thank the scientists as well as the entire ALS community who have all worked tirelessly to bring this first-of-its-kind treatment to people with SOD1-ALS,” said Biogen President and Chief Executive Officer Christopher A. Viehbacher. “Today also marks a watershed moment in ALS research because we have reached consensus for the first time that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS.” We anticipate that this significant scientific achievement will hasten the development of novel ALS drugs.”
Serious neurologic complications, including myelitis and/or radiculitis; papilledema and raised intracranial pressure; and aseptic meningitis, were related to QALSODY. If symptoms of myelitis, radiculitis, papilledema, raised intracranial pressure, or aseptic meningitis appear, a diagnostic workup and treatment should be initiated in accordance with the standard of care. QALSODY may be interrupted or stopped by management. Pain, weariness, arthralgia, elevated cerebrospinal (CSF) white blood cell count, and myalgia were the most common adverse responses that occurred in 10% of QALSODY-treated subjects and more than the placebo arm.
QALSODY was approved based on 12-month integrated data from VALOUR and its OLE comparing earlier tofersen initiation (at the commencement of VALOUR) to delayed tofersen initiation (six months later, in the OLE), which were published in The New England Journal of Medicine. QALSODY will be available for shipping to healthcare professionals in the United States in about a week. Biogen predicts that as institutions and treatment centers learn more about QALSODY, the time to treatment may vary.
Kiromic BioPharma Announces FDA Authorization of IND to Initiate Phase I Clinical Trial Evaluating Deltacel in Non-Small Cell Lung Cancer
Kiromic BioPharma has received approval from the FDA to begin a Phase 1 clinical trial for Deltacel (KB-GDT-01), which is being developed using their DIAMOND® artificial intelligence and data mining platform. The focus of this trial will be to assess the effectiveness of Deltacel for treating non-small cell lung cancer (NSCLC) patients.
Deltacel is an allogeneic Gamma Delta T-cell (GDT) therapy developed by Kiromic BioPharma that does not require viral vectors, resulting in lower manufacturing costs. The company aims to use cell therapy to treat solid malignancies, which make up the majority of cancers, including non-small cell lung cancer (NSCLC). Lung cancer is responsible for a significant number of cancer deaths in the US, surpassing the combined fatalities of colon, breast, and prostate cancers annually. Kiromic’s approach to addressing this unmet need in cancer treatment could prove crucial in reducing the number of deaths caused by lung cancer.
According to Kiromic BioPharma’s CEO, Pietro Bersani, the FDA’s approval to use Deltacel on patients is an important step forward in the clinical development of their GDT therapy candidate. Bersani believes that Deltacel has the potential to be an effective and well-tolerated treatment for patients with non-small cell lung cancer and other solid cancers, and the company is eager to see the impact it may have. Kiromic is currently working on activating clinical trial sites and will provide updates on their progress.
Atsena Therapeutics Receives FDA Clearance of IND Application for ATSN-201
Atsena Therapeutics has announced that the FDA has approved its Investigational New Drug (IND) application for a Phase I/II clinical trial of ATSN-201 in patients with X-linked retinoschisis (XLRS). The treatment utilizes a unique spreading capsid, AAV.SPR was developed by the company to address the obstacles associated with intravitreally delivered AAVs for XLRS treatment.
Shannon Boye, the Founder and Director of Atsena Therapeutics, explained that intravitreally delivered AAVs are not an ideal solution for XLRS treatment, as they do not produce enough gene expression in photoreceptors to provide therapy and can cause inflammation that compromises vision. However, the company’s AAV.SPR has been designed to overcome these limitations and is well-suited for XLRS treatment. AAV.SPR can drive sufficient gene expression in photoreceptors without causing the surgical risks associated with foveal detachment, which is particularly important since XLRS patients have fragile retinas due to the presence of schisis lesions. With this promising gene therapy, Atsena Therapeutics aims to offer a viable treatment option for XLRS patients who currently have no approved treatment available.
Atsena Therapeutics’ Chief Medical Officer, Kenji Fujita, announced that with the FDA’s approval of the IND application for ATSN-201, the company is preparing to move forward with its first program that utilizes AAV.SPR for the treatment of XLRS in mid-2023. Fujita expressed excitement about the opportunity to evaluate ATSN-201 and address the need for a treatment that can enhance or restore vision in XLRS patients.The Lighthouse Study is a Phase I/II clinical trial that will involve an open-label, dose-escalation approach to evaluating the efficacy of subretinal injection of ATSN-201 in male patients aged 6 to 65 who have been clinically diagnosed with XLRS due to pathogenic or likely pathogenic mutations in RS1.