FDA Updates for Week of July 11, 2022

In COVID-19 news, the FDA grants EUA for Novavax’s COVID-19 vaccine. The agency approved a novel diagnostic dye and another indication for Xalkori, but delayed a decision on a therapy for esophageal cancer. FDA has also accepted a BLA for advanced HER2 breast cancer therapy. Finally, Can-Fite BioPharma plans submission for psoriasis therapy.

FDA grants EUA for Novavax COVID-19 vaccine.

American adults have another COVID-19 vaccine option after the FDA granted emergency use authorization (EUA) for Novavax’s COVID-19 vaccine. In early June, FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 21 to 0, with one abstention, to recommend the FDA grant EUA for the vaccine.

At the June 7, 2022, VRBPAC meeting, Novavax “heard the overwhelming support for our vaccine from physicians, healthcare organizations, and consumers who are eagerly anticipating a protein-based vaccine option,” Stanley C. Erck, president and CEO of Novavax, said in a statement.

Anticipating the EUA, the Department of Health and Human Services (HHS) and the Department of Defense already secured an initial 3.2 million doses of the vaccine, Novavax said in a statementearlier this week.

FDA approves novel diagnostic dye for endoscopy.

The FDA has approved the French company Provepharm’s new drug application for Bludigo (indigotindisulfonate). Bludigo is an indigo carmine injectable, a blue dye that is used as a diagnostic during bladder endoscopy for the assessment of the ureters in adults following surgical procedures.

Provepharm filed its application for approval with the FDA in September 2021 and with this new approval, its U.S. affiliate, Provepharm Inc., will market the product.

FDA approves Xalkori for rare type of tumor.

The FDA has approved Pfizer’s Xalkori (crizotinib) for an additional indication: to treat adult and pediatric patients 1 year of age and older with unresectable, recurrent, or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors (IMT). Myofibroblastic tumors, masses of immune cells, are rare.

The approval was based on safety and efficacy from two trials that included 14 pediatric patients from trial ADVL0912 and seven adult patients from trial A8081013. For the pediatric patients, 12 of the 14 patients experienced an objective response, For the adult patients, five patients had objective responses.

Xalkori, first approved in 2011, is also approved to treat ALK- or ROS1-positive non-small cell lung cancer and ALK-positive large cell lymphoma in children and young adults.

FDA delays decision on BeiGene’s BLA for tislelizumab.

The FDA has deferred action on the biologics license application (BLA) for tislelizumab as a second-line treatment for patients with unresectable or metastatic esophageal squamous cell carcinoma. The FDA has been unable to conduct required inspections in China due to COVID-19 related travel restrictions. The application remains under review, and the FDA did not provide a new action date.

In January 2021, BeiGene and Novartis announced a collaboration granting Novartis rights to co-develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.

Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors. In September 2021, the FDA accepted the BLA for tislelizumab in second-line esophageal squamous cell carcinoma.

FDA accepts BLA for advanced HER2 breast cancer therapy.

The FDA has accepted Byondis’ biologics license application (BLA) for [vic-]trastuzumab duocarmazine (SYD985) in patients with HER2-positive unresectable locally advanced or metastatic breast cancer. The agency has assigned a Prescription Drug User Fee Act (PDUFA) action date of May 12, 2023.

The application is supported by data from the pivotal phase 3 TULIP clinical trial comparing SYD985 with physician’s choice treatment in patients with pre-treated HER2-positive metastatic breast cancer. The study enrolled 437 women with a median of four prior treatments.

SYD985 is a next-generation antibody-drug conjugate (ADC). It adds Byondis’ duocarmazine linker-drug technology ByonZine to trastuzumab. The FDA granted fast track designation to SYD985 in January 2018 based on promising phase 1 data involving heavily pretreated last-line patients with HER2-positive metastatic breast cancer.

Novel psoriasis therapy to be submitted to FDA.

Can-Fite BioPharma has announced plans to submit applications to the FDA and the European Medicines Agency (EMA) for its lead drug candidate piclidenoson in the treatment of moderate-to-severe psoriasis. Piclidenoson is a novel, first-in-class, A3 adenosine receptor agonist small molecule that inhibits the inflammatory cytokines interleukin 17 and 23.

The company plans to submit the final efficacy and safety results from the phase 3 COMFORT study, which assessed the efficacy and safety of piclidenoson in more than 400 adults with moderate-to-severe plaque psoriasis. The study met its primary endpoint with statistically significant improvement over placebo in psoriasis patients.