Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for KALYDECOTM (ivacaftor) and granted the company's request for six-month Priority Review. KALYDECO targets the defective protein that causes cystic fibrosis (CF) in a subset of people with the disease. If approved, KALYDECO will be the first treatment to target the underlying cause of CF.
The FDA grants Priority Review to medicines that offer major advances in treatment or provide a treatment where no adequate therapy exists. A target review date of April 18, 2012 is set under the Prescription Drug User Fee Act (PDUFA) for the FDA's approval decision, which is four months earlier than the standard review time of 10 months.
In addition, Vertex announced today that its marketing authorization application (MAA) for KALYDECO has been validated by the European Medicines Agency (EMA). Validation indicates that the application is complete and starts the regulatory review process by the Committee for Medicinal Products for Human Use (CHMP). Earlier this year, the EMA accepted Vertex's request for accelerated assessment, which is granted to new medicines of major public health interest and shortens the EMA's review time.
"If approved, KALYDECO will be the first treatment to target the underlying cause of CF," said Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex. "The commitments by the FDA and the EMA to expedite their reviews of our applications underscore the significant potential of KALYDECO to help people living with cystic fibrosis."
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