FDA Expands Approval of Vonvendi for von Willebrand Disease, Including Pediatric Use

“This approval demonstrates FDA flexibility in evaluating applications of therapeutics to treat rare diseases,” said FDA Vinay Prasad, M.D., M.P.H., Director of the FDA’s Center for Biologics Evaluation and Research. “When we see the trifecta of plausible mechanism of action, robust pharmacology/biologic science, and supportive clinical study data, we promptly act even if that data is derived from a small sample size study.”

Von Willebrand disease is a bleeding disorder in which a person’s blood doesn't clot properly. People with the disease have low levels of von Willebrand factor (VWF), a protein that helps blood clot, or the protein doesn't perform as it should.

Vonvendi is the only recombinant (non-plasma derived) VWF product approved for VWD in the U.S., and this is the first recombinant VWF product approved for pediatric patients in the U.S. Prior to this approval, only plasma derived VWF products were available to the pediatric population.

“Today’s approval is a testament to the collaborative interactions between CBER review teams and product developers, aimed at making innovative therapies available for use in pediatric populations, at the same time as adults,” said Vijay Kumar, M.D., acting director of the CBER Office of Therapeutic Products.

Efficacy of Vonvendi was demonstrated in multiple clinical studies that showed success in treatment control of bleeding episodes and use in perioperative management in patients with VWD of all ages. It also proved successful for prevention of bleeding episodes in adults with VWD.

The most common adverse reactions observed in greater than, or equal to, 2% of patients in clinical trials with Vonvendi were headache, vomiting, nausea, dizziness and itchy skin (generalized pruritus).

This approval was completed under Priority Review and the product received Orphan Drug Designation. The FDA granted approval of this product to Takeda.