Baxalta Gets FDA Nod for Rare Disease DrugBy
Baxalta Incorporated, the biopharmaceutical company spun off from Baxter earlier this year has received approval from the US Food and Drug Administration (FDA for Vonvendi (von Willebrand factor [recombinant]), a h for on-demand treatment and control of bleeding episodes in adults (age 18 and older) diagnosed with von Willebrand disease.
Vonvendi is a recombinant protein treatment that includes a physiologic distribution of proteins called multimers, including ultra-large multimers (ULMs), with large multimers being the most active form of the protein supporting clot formation. The treatment is also the first in the United States that contains only trace amounts of Factor VIII (FVIII), offering the flexibility to administer FVIII only when needed. This attribute allows for tailored treatment for patients who may not require additional FVIII.