Therapy Areas: Hereditary Disorders
FDA and EMA Accept Vertex Applications for Tezacaftor/Ivacaftor Combination Treatment in People with Cystic Fibrosis
25 August 2017 - - Applications by Boston, Massachusetts-based serious disease therapeutics developer Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) for the use of the tezacaftor/ivacaftor combination treatment in people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation or one F508del mutation and one residual function mutation that is responsive to tezacaftor/ivacaftor have been accepted by the US Food and Drug Administration and the European Medicines Agency (EMA), the company said.
In the United States, the FDA has granted Priority Review of the New Drug Application and set an action date of February 28, 2018.
In the European Union, the EMA has validated the Marketing Authorization Application, which confirms that the submission is complete and initiates the centralized review process of approximately 210 days for the Committee for Medicinal Products for Human Use to give an Opinion.
The submissions are supported by positive results from two global Phase 3 studies.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.
The company has research and development sites and commercial offices in the United States, Europe, Canada, and Australia.