FDA Updates for the Week of June 6, 2022

In COVID-19 news, an FDA advisory committee recommends EUA for Novavax’s vaccine. The FDA has also granted approvals to Dupixent for children as young as 6 month with atopic dermatitis and to the biosimilar Riabni for rheumatoid arthritis. An advisory committee has also recommended approval for two gene therapies, one for a rare neurogenerative disease and another for a blood disease.

FDA approves the biosimilar Riabni for rheumatoid arthritis.

The FDA has approved Amgen’s Riabni (rituximab-arrx), a biosimilar to Biogen’s Rituxan, in combination with methotrexate for adults with moderate-to-severe active rheumatoid arthritis. The FDA in December 2020 approved Riabni for the treatment of adult patients with non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, granulomatosis with polyangiitis (also called Wegener's granulomatosis) and microscopic polyangiitis.

Riabni is now approved to treat all available Rituxan indications.

FDA approves Dupixent for young children with atopic dermatitis.

The FDA has approved Dupixent (dupilumab) for children aged 6 months to 5 years with moderate-to-severe atopic dermatitis. It is approved for young children whose disease is not adequately controlled with topical prescription therapies. This approval makes Dupixent the first biologic medicine approved to treat moderate-to-severe atopic dermatitis from infancy through adulthood.

Dupixent is a human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. It is being developed jointly by Sanofi and Regeneron.

Dupixent is approved for use in certain patients with atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis and eosinophilic esophagitis in different age populations. Its most recent approval in May 2022 was to treat patients with eosinophilic esophagitis, an inflammatory condition that causes the esophagus to narrow and makes it difficult to eat.

FDA committee recommends EUA for Novavax’s COVID-19 vaccine.

FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 21 to 0, with one abstention, to recommend the agency grant Emergency Use Authorization (EUA) for Novavax’s COVID-19 vaccine (NVX-CoV2373) for individuals aged 18 years and over.

The VRBPAC considered data from Novavax’s phase 3 clinical trial, PREVENT-19, which enrolled around 30,000 participants 18 years and older in the United States and Mexico and was published in the New England Journal of Medicine.

In the trial, the Novavax COVID-19 vaccine demonstrated 90.4% efficacy. Additionally, serious and severe adverse events were low in number and balanced between vaccine and placebo groups.

FDA advisory committee endorses gene therapy for neurogenerative disease.

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted in support of approval of Bluebird Bio’s elivaldogene autotemcel (eli-cel). Committee members unanimously voted yes on the question of whether the benefits of the gene therapy outweigh the risks for treating early active cerebral adrenoleukodystrophy (CALD).

CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes behavioral, cognitive, and neurological deficits. Nearly half of patients who do not receive treatment die within five years of symptom onset.

Allogeneic hematopoietic stem cell transplant (allo-HSCT) is currently the only effective treatment option but is associated with serious potential complications, and patients may not have a matched donor.

The biologic license application for eli-cel is currently under priority review by the FDA with a PDUFA goal date set for Sept. 16, 2022.

FDA advisory committee supports approval of gene therapy for blood disease.

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted in support of approval of Bluebird Bio’s betibeglogene autotemcel (beti-cel) for the treatment of people with beta-thalassemia who require regular red blood cell transfusions. The committee vote was unanimous with 13 yes votes and 0 no votes on the question of whether the benefits of beti-cel outweigh the risks.

Beta-thalassemia is a severe genetic blood disease caused by mutations in the beta-globin gene and is characterized by significantly reduced or absent adult hemoglobin production. This can result in severe anemia and lifelong dependence on red blood cell transfusions.

Patients who require regular transfusions to maintain hemoglobin levels typically undergo a four to seven-hour process every two to three weeks. While transfusions temporarily relieve symptoms associated with severe anemia, including fatigue, weakness, and shortness of breath, they can lead to iron overload and serious complications, including shortened survival.

The biologic license application for beti-cel is currently under priority review by the FDA with a PDUFA goal date set for Aug. 19, 2022.