FDA Updates for Week of Aug. 22, 2022

FDA approves rapid-acting Auvelity for major depression.

The FDA has approved Axsome Therapeutics’ Auvelity (dextromethorphan-bupropion) extended-release tablets to treat major depressive disorder (MDD) in adults. Axsome anticipates Auvelity (formerly called AXS-05) to be commercially available in the United States in the fourth quarter of 2022.

Auvelity is the first and only rapid-acting oral medicine approved for the treatment of MDD with labeling of statistically significant antidepressant efficacy compared with placebo starting at one week. The rapid antidepressant effects were sustained at all subsequent timepoints.

The approval was based on two trials, including the GEMINI placebo-controlled study, and confirmatory evidence and the ASCEND study comparing Auvelity to bupropion sustained-release tablets. Data from the GEMINI study were published in May 2022 in The Journal of Clinical Psychiatry and data from the ASCEND study were published in May 2022 in The American Journal of Psychiatry.

FDA expands Imbruvica for young children with GVHD.

The FDA has approved Imbruvica (ibrutinib) to treat children one year and older with chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy. This is the first pediatric indication for Imbruvica and the 12th approval for the therapy.

The recommended dose for patients 12 years and older is 420 mg taken orally once daily. In patients one year old to less than 12 years of age, the recommended dose is 240 mg/m2 taken orally once daily (up to a dose of 420 mg). This FDA approval also includes an oral suspension formulation of Imbruvica.

Imbruvica is jointly developed and commercialized by Janssen Biotech and Pharmacyclics, an AbbVie company. Since 2017, Imbruvica has been approved as a single-agent therapy for adult patients with cGVHD who have experienced failure of prior systemic therapy, becoming the first FDA-approved treatment for this indication.

FDA expands use of Omnipod 5 for younger children.

FDA has approved Insulet’s Omnipod 5 automated insulin delivery system for children aged two years and older with type 1 diabetes. Omnipod 5, the first tubeless automated insulin delivery (AID) system in the United States, was approved in January 2022 for use in individuals aged six and older.

Omnipod 5 is a system that integrates with the Dexcom G6 CGM system and a compatible smartphone to automatically adjust insulin. The system consists of the tubeless Pod enhanced with SmartAdjust technology, the Omnipod 5 mobile app with its integrated SmartBolus Calculator, and the Dexcom G6 CGM.

In a recent publication in Diabetes Care, a study found that use of Omnipod 5 in patients 2 years of age to 5.9 years of age experienced improved glycemic measures and reduced hypoglycemia during the study phase compared with baseline.

Omnipod 5 is now available through retail pharmacies, as well as specialty and mail-order pharmacies to anyone with a prescription and coverage.

FDA grants EUA for Novavax COVID-19 vaccine for adolescents.

The FDA has expanded the emergency use authorization (EUA) of Novavax’s protein-based COVID-19 vaccine to provide a two-dose primary series to prevent COVID-19 caused in adolescents aged 12 through 17.

The EUA decision was based on data from the ongoing pediatric expansion of the phase 3 PREVENT-19 trial of 2,247 adolescents aged 12 through 17 years across 75 sites in the United States. The efficacy analysis was supported by assessment of antibody titers that were shown to be higher in adolescents than in young adults.

Safety data from the pediatric expansion showed the vaccine to be generally well-tolerated. Serious and severe adverse reactions were low in number and balanced between vaccine and placebo groups, and not considered related to the vaccine.

FDA accepts NDA for GSK’s myelofibrosis drug.

The FDA accepted GSK’s new drug application (NDA) for momelotinib, a potential new medicine that may address the significant unmet medical needs of patients with myelofibrosis who have anemia. The agency has assigned a Prescription Drug User Fee Act action date of June 16, 2023, GSK said in a statement.

Myelofibrosis is a cancer of the bone marrow and can lead to anemia. Momelotinib has the ability to inhibit three key signaling pathways: Janus kinase (JAK) 1, and JAK2 and activin A receptor, type I (ACVR1). Inhibition of JAK1 and JAK2 may improve symptoms and enlarged spleen, GSK said, and direct inhibition of ACVR1 leads to a decrease in circulating hepcidin, which is elevated in myelofibrosis and contributes to anemia.

Momelotinib was most recently developed by Sierra Oncology, which GSK acquired in July 2022. The NDA was submitted in June 2022 by Sierra.

The NDA is based on the results from key phase 3 trials, including the pivotal MOMENTUM trial, which met all primary and key secondary endpoints, including total symptom score, transfusion independence rate and splenic response rate.

Moderna completes application for updated COVID-19 booster.

Moderna has completed its submission to the FDA for emergency use authorization for its omicron-targeting bivalent COVID-19 booster vaccine. The application is for a 50 µg booster dose for adults 18 years of age and older.

The update booster contains both the original strain of SARS-CoV-2, as well as the BA.4/BA.5 subvariants of the omicron strain. Moderna's application is based on preclinical data, as well as clinical trial data from a phase 2/3 studying the updated booster vaccine that targets the omicron BA.1 subvariant. In the study, the updated booster met all primary endpoints, including superior neutralizing antibody response against omicron (BA.1) when compared with a 50 µg booster dose in previously uninfected participants, as well as potent neutralizing antibody responses against the omicron subvariants BA.4 and BA.5 compared with the currently authorized booster.

Pending authorization, Moderna will be ready to ship the updated bivalent booster in September 2022.

Pfizer submits EUA for updated COVID-19 booster.

Pfizer and BioNTech have submitted an emergency use authorization (EUA) request to the FDA for a booster dose of their COVID-19 vaccine that has been adapted specifically for the omicron BA.4/BA.5 variants for those 12 years and older. If authorized the update vaccine will be available to ship immediately.

The application follows guidance from the FDA to include clinical data from the companies’ bivalent omicron BA.1-adapted vaccine and preclinical and manufacturing data from the companies’ bivalent omicron BA.4/BA.5-adapted vaccine to address the continued evolution of COVID-19 virus.

A conditional marketing authorization application has also been initiated with the European Medicines Agency for the omicron BA.4/BA.5-adapted bivalent vaccine and is expected to be completed in the coming days.