Biologics

Pfizer BLA for Hemophilia Accepted for Review

FDA has accepted for review a Pfizer BLA for its anti-tissue factor pathway inhibitor (anti-TFPI) candidate marstacimab for treating hemophilia A or hemophilia B without inhibitors to Factor VIII or Factor IX. The agency has set a user fee review action target date in the fourth quarter of 2024. If approved, marstacimab is expected to become the “first once-weekly subcutaneous treatment for people living with hemophilia B and the first treatment administered as a flat dose for people living with hemophilia A or B,” the company says.

“For more than five decades, the most common treatment approach for hemophilia A and B has been factor replacement therapy, which replaces missing clotting factors to facilitate proper blood coagulation,” Pfizer says. “Marstacimab is a novel, investigational treatment for hemophilia that is designed to restore hemostasis by inhibiting TFPI.”

The submission is based on data from the Phase 3 BASIS trial (NCT03938792) that were recently presented at the American Society of Hematology annual meeting. The data showed that marstacimab reduced annualized bleeding rate by 35% and 92% compared to routine prophylaxis and on-demand treatment in patients with hemophilia A and B without inhibitors, respectively, according to the company.

Marstacimab is a monoclonal immunoglobulin G isotype, subclass 1 (IgG1) that targets the Kunitz 2 domain of TFPI, a natural anticoagulation protein that functions to prevent the formation of blood clots, Pfizer says.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome