US regulators have approved Baxalta’s Vonvendi, giving patients with Von Willebrand Disease access to the first recombinant treatment for their condition.
Vonvendi can now be used for on-demand treatment and control of bleeding episodes in adults with von Willebrand disease, after clinical trials showed 100 percent treatment success with a median of only two infusions to treat severe bleeds.
VWD is the most common inherited bleeding disorder worldwide, affecting up to one in 100 people, and its rarest form one in 1,000,000. The genetic disorder causes alterations or deficits in von Willebrand factor, resulting in impaired clotting, and can manifest through a variety of bleeding events such as mucosal bleeds or menorrhagia.
Patients often live with the disease for years without a proper diagnosis; even with a confirmed diagnosis, there are limited treatment options available, the firm noted.
Vonvendi is an innovative recombinant protein treatment that includes a physiologic distribution of proteins called multimers, which support clot formation. It is also the first in the US containing only trace amounts of Factor VIII (FVIII), offering the flexibility to administer FVIII only when needed.
“With its unique feature of ultra-large multimers and the clinical profile seen in our pivotal study, Vonvendi is an important new option with the potential to redefine treatment for adults with von Willebrand disease,” said Brian Goff, president of Haematology at Baxalta.
Bendeka for CLL, NHL
Elsewhere, the regulator also green-lighted Teva and Eagle’s Bendeka, a liquid, low-volume and short-time 10-minute infusion formulation of the cancer drug bendamustine.
Bendeka is approved for the treatment of patients with chronic lymphocytic leukemia or indolent B-cell non-Hodgkin lymphoma (NHL) that has progressed during or within six months of treatment with rituximab or a rituximab-containing regimen.