Loading...
Loading...
Baxter International Inc.
today announced that the company has
submitted a biologics license application (BLA) to the United States (U.S.)
Food and Drug Administration (FDA) for the approval of BAX111, the first
highly-purified recombinant von Willebrand Factor (rVWF) in clinical
development as a treatment for patients with von Willebrand disease, the
most common type of inherited bleeding disorder.(1)
"If approved, BAX111 will be the first recombinant replacement treatment for
von Willebrand disease, offering an important new option that may provide
greater flexibility in treating patients with this challenging disease,"
said John Orloff, vice president and global head of research and development
at Baxter BioScience. "Filing for U.S. approval for this treatment helps us
further advance our pursuit of new treatment options and improved quality of
care for people with a range of bleeding disorders around the world."
The filing was based on the completion of a Phase III, multi-center,
open-label clinical trial assessing the safety, efficacy and
pharmacokinetics of BAX111. The study met its primary efficacy endpoint
defined by the number of patients who achieved treatment success for control
of bleeding episodes. All patients treated in the full analysis set (n=22)
experienced a 100% treatment success rating based on a 4-point efficacy
rating scale, comparing prospectively estimated number of infusions needed
to treat the bleeding episodes to the actual number of infusions
administered. The median number of infusions required to treat bleeding
events in the trial was 1.0 and the majority of events (81.1%) were resolved
with a single infusion.
A total of 125 adverse events (AE's) following 318 infusions occurred in
25/37 subjects. Eight AEs were considered causally related to BAX111: six
non-serious related AEs (tachycardia, infusion site paresthesia,
electrocardiography (ECG) T-wave inversion, dysgeusia, generalized pruritis
and hot flush) occurred in four patients, and two related SAEs (chest
discomfort and increased heart rate) occurred in one patient.
Baxter expects to publish additional data from the trial in the coming
months. Both the European Commission and the U.S. Food and Drug
Administration have granted orphan-drug designation for BAX111.
Loading...
Loading...
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Posted In: News
Benzinga simplifies the market for smarter investing
Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.
Join Now: Free!
Already a member?Sign in