The FDA’s decision was based on promising results from the IB1001-301 pivotal clinical trial, a multinational, randomized, double-blind, placebo-controlled study that included 60 pediatric and adult participants diagnosed with NPC. The trial successfully met its primary efficacy endpoint, demonstrating significant improvements in neurological signs and symptoms within just 12 weeks of treatment. Results were published in the February 1, 2024, issue of the New England Journal of Medicine.
Laurie Turner, Family Services Manager at the National Niemann-Pick Disease Foundation, praised the approval, stating, “The FDA approval of AQNEURSA marks a significant breakthrough for those living with Niemann-Pick disease type C. For too long, our community has been without an approved therapy for the treatment of NPC.” She expressed gratitude for the dedication to innovative research that led to this important development, highlighting the hope it brings to families navigating the challenges of NPC.
FDA Greenlights First New Schizophrenia Drug in Over Three Decades
Bristol Myers Squibb has announced the FDA’s approval of COBENFY (xanomeline and trospium chloride), a groundbreaking oral medication for treating schizophrenia in adults. This marks the introduction of the first new class of treatment for schizophrenia in decades, focusing on selectively targeting M1 and M4 receptors in the brain while avoiding D2 receptor blockade. This novel approach has the potential to transform the treatment landscape for those affected by this complex mental illness.
Chris Boerner, PhD, board chair and chief executive officer at Bristol Myers Squibb, emphasized the significance of this approval, stating, “Today’s landmark approval of our first-in-class treatment for schizophrenia marks an important milestone for the community, where after more than 30 years, there is now an entirely new pharmacological approach for schizophrenia — one that has the potential to change the treatment paradigm.” The company’s dedication to neuropsychiatry reflects a commitment to improving outcomes for individuals living with serious mental health conditions.
Schizophrenia affects approximately 2.8 million people in the United States, often leading to persistent and debilitating symptoms that are difficult to manage. Gordon Lavigne, CEO of the Schizophrenia & Psychosis Action Alliance, remarked, “For people living with schizophrenia, it’s often difficult to find a treatment that works for them. Having a variety of treatment options gives patients and healthcare providers the tools to help manage this serious condition.” He highlighted that today’s approval offers new hope for those seeking effective treatments to rebuild their lives.
The FDA’s approval is supported by the EMERGENT clinical program, which included multiple trials demonstrating statistically significant improvements in schizophrenia symptoms compared to placebo. COBENFY showed reductions in symptoms as measured by the Positive and Negative Syndrome Scale (PANSS), establishing a robust safety profile without the typical warnings associated with atypical antipsychotics. Alongside the approval, Bristol Myers Squibb announced the launch of COBENFY Cares™, a program designed to support patients beginning their treatment, with enrollment expected to start in late October.
FDA Approves Selpercatinib for RET-Mutated MTC in Pediatric and Adult Patients
The FDA has approved selpercatinib (RETEVMO, Eli Lilly and Company) for the treatment of advanced or metastatic medullary thyroid cancer with an RET mutation in adult and pediatric patients aged 2 years and older who require systemic therapy. This approval follows the positive results from the clinical trial LIBRETTO-531 (NCT04211337), which demonstrated the drug’s efficacy in this patient population.
Previously, selpercatinib received accelerated approval in 2020 for patients aged 12 and older. In May 2024, the FDA extended this approval to include pediatric patients as young as 2 years. Selpercatinib is an oral medication that inhibits the activity of abnormal RET proteins and fusion proteins associated with RET. The recommended dosing for pediatric patients under 12 years is based on body surface area, while for those aged 12 and older, the dose is determined by weight.
The LIBRETTO-531 trial was a randomized, multicenter, open-label phase III study involving adults and adolescents with advanced or metastatic RET-mutant MTC. Patients were assigned to receive either 160 mg of selpercatinib orally twice daily or standard treatments, including cabozantinib or vandetanib. The primary efficacy endpoint was progression-free survival (PFS), with secondary endpoints assessing tolerability and additional efficacy outcomes. Results showed that the median PFS for patients receiving selpercatinib was not reached, while it was 16.8 months for the control group, indicating a significant advantage for the selpercatinib arm.
The trial findings also highlighted the tolerability of selpercatinib, with patients reporting fewer severe side effects compared to those on cabozantinib or vandetanib. Common adverse events included hypertension, edema, dry mouth, fatigue, and diarrhea. Notably, dose reductions due to adverse events occurred in 38.9% of patients taking selpercatinib, compared to 77.3% in the control group, and treatment discontinuation rates were significantly lower for selpercatinib at approximately 4.7% versus 26.8% for the control.
FDA Grants Landmark Approval to DUPIXENT as First Biologic for COPD
Regeneron Pharmaceuticals, Inc. and Sanofi have announced the FDA approval of DUPIXENT (dupilumab) as an add-on maintenance treatment for adults with inadequately controlled chronic obstructive pulmonary disease (COPD) exhibiting an eosinophilic phenotype. This marks a significant milestone as DUPIXENT becomes the first biologic medicine approved in the U.S. specifically for these patients, addressing a long-standing need for effective therapies in this population.
Patients with inadequately controlled COPD often face debilitating symptoms, such as breathlessness, coughing, and wheezing, which significantly hinder their quality of life. Dr. Jean Wright, CEO of The COPD Foundation, expressed the critical need for new therapeutic options to help these individuals manage their condition. The approval of DUPIXENT is expected to provide relief for many, allowing them to engage more comfortably in daily activities that most people take for granted.
The FDA’s decision was based on data from two pivotal Phase III trials, BOREAS and NOTUS, which demonstrated the efficacy and safety of DUPIXENT compared to placebo in adults on maximal standard-of-care inhaled therapy. In these studies, patients receiving DUPIXENT showed a 30% and 34% reduction in the annualized rate of moderate or severe COPD exacerbations, alongside notable improvements in lung function as measured by post-bronchodilator FEV1 at 12 and 52 weeks. Additionally, a significant proportion of patients reported enhancements in their quality of life as assessed by the St. George’s Respiratory Questionnaire.
Overall safety results from the trials were consistent with the established profile of DUPIXENT, with common adverse events including viral infections, headaches, and nasopharyngitis. Paul Hudson, CEO at Sanofi, highlighted DUPIXENT’s transformative potential, stating that this approval solidifies its role as the first and only add-on biologic treatment for inadequately controlled COPD, offering hope for improved respiratory health and reduced exacerbations. The FDA evaluated DUPIXENT under Priority Review, underscoring its significance as a novel therapeutic option for this serious condition, with ongoing reviews from regulatory authorities worldwide.
Pfizer Pulls OXBRYTA for Sickle Cell Disease From Global Markets
Pfizer Inc. has announced the voluntary withdrawal of all lots of OXBRYTA (voxelotor), a treatment for sickle cell disease, in all approved markets. The decision comes as a result of new clinical data indicating that the overall benefits of OXBRYTA no longer outweigh the associated risks for the approved patient population. This data suggests concerning trends related to vaso-occlusive crises and fatal events, necessitating further investigation.
In addition to pulling OXBRYTA from the market, Pfizer is discontinuing all active voxelotor clinical trials and expanded access programs worldwide. The company’s commitment to patient safety is paramount, as emphasized by Aida Habtezion, Pfizer’s Chief Medical Officer. She stated that the decision was made with the well-being of SCD patients in mind, who face significant challenges and limited treatment options. Pfizer urges affected patients to consult their healthcare providers for alternative treatments while the company continues to analyze the emerging safety data.
Pfizer has promptly notified regulatory authorities regarding this decision and is actively communicating with patients, physicians, and other healthcare professionals about the situation. The company has assured stakeholders that it will keep them updated on the developments concerning OXBRYTA.
Despite the withdrawal and cessation of clinical studies, Pfizer does not anticipate any adverse effects on its financial outlook for the year 2024. The company remains dedicated to exploring safe and effective treatments for SCD and ensuring the safety of the patients it serves.
