FDA approves expanded use of Takeda’s VONVENDI for VWD patients

Published 06/09/2025, 01:50
FDA approves expanded use of Takeda’s VONVENDI for VWD patients

OSAKA/CAMBRIDGE - The U.S. Food and Drug Administration has approved Takeda’s (TSE:4502/NYSE:TAK) supplemental Biologics License Application for VONVENDI, expanding its use to include routine prophylaxis in adults with Type 1 and 2 von Willebrand Disease (VWD) and on-demand treatment in pediatric patients. The pharmaceutical giant, with a market capitalization of $48.3 billion and annual revenue of $31.1 billion, has seen its stock rise 11.8% year-to-date, according to InvestingPro data.

The approval makes VONVENDI the only recombinant von Willebrand Factor replacement therapy with approved indications for both adults and children with VWD, according to a company press release.

VWD, which affects more than 3 million people in the U.S., is caused by low levels or dysfunction of von Willebrand factor, a protein essential for blood clotting. This can lead to prolonged bleeding episodes that may disrupt daily activities. InvestingPro analysis indicates Takeda maintains a "Good" overall financial health score, suggesting strong positioning to support this expanded treatment offering. Subscribers can access detailed financial health metrics and 12+ exclusive ProTips about Takeda’s market position.

The expanded approval is based on data from three clinical trials and supportive real-world evidence. In clinical studies, the majority of non-surgical bleeds were treated with a single VONVENDI infusion in both adults (157/192) and children (80/104).

"The FDA’s approval of VONVENDI for routine prophylactic use in adults with all VWD types marks a pivotal milestone in advancing care for this challenging condition," said Jonathan C. Roberts, an investigator in the VONVENDI prophylaxis study.

VONVENDI was previously approved for on-demand and perioperative use in adults with VWD and routine prophylactic use in adults with severe Type 3 VWD receiving on-demand therapy.

Common adverse reactions observed in clinical trials included headache, vomiting, nausea, dizziness, and generalized pruritus. The therapy carries warnings about potential thromboembolic events, hypersensitivity reactions, and the development of neutralizing antibodies.

Takeda has also submitted applications seeking approval to expand VONVENDI’s indications in Japan and the European Union. With analyst consensus suggesting potential upside and the stock currently appearing undervalued according to InvestingPro’s Fair Value model, investors can access comprehensive analysis through InvestingPro’s detailed research reports, which provide deep-dive insights into Takeda’s growth prospects and market position.

In other recent news, Takeda Pharmaceutical Company reported its Q1 2025 earnings, revealing a revenue of 1,100 billion yen. This figure represents a decrease of 8.4% compared to the previous year. Despite the decline in revenue, Takeda has maintained its full-year guidance, expecting revenue to remain broadly flat. Analysts and investors noted that the stock market showed no immediate reaction to the earnings announcement. The company has not announced any mergers or acquisitions recently. There were also no significant analyst upgrades or downgrades reported in relation to Takeda. These developments provide a current snapshot of Takeda’s financial performance and market position.

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