FDA Approves Kalydeco (ivacaftor) for People Ages 2 and Older With Cystic Fibrosis Who Have Certain Residual Function Mutations
BOSTON--(BUSINESS WIRE) Aug 1, 2017 -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for use in more than 600 people with cystic fibrosis (CF) ages 2 and older who have one of five residual function mutations that result in a splicing defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This approval was based on Phase 3 clinical data for Kalydeco in these mutations and follows the FDA's approval of Kalydeco in May 2017 for 23 other residual function mutations, which was based on analyses of in vitro data. Both approvals are supported by more than five years of real-world clinical experience that demonstrate Kalydeco's established safety and efficacy profile. Based on today's approval, Vertex increased its guidance for 2017 Kalydeco product revenues to a range of $770 million to $800 million. Vertex's guidance range for total CF product revenues in 2017 is now $1.87 billion to $2.1 billion, including ORKAMBI guidance of $1.1 billion - $1.3 billion.
"In the five years since Kalydeco became the first approved medicine to treat the underlying cause of cystic fibrosis, we have been relentless in our efforts to bring this important medicine to all who may benefit," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. "We will continue to pursue this goal until all people with CF have a medicine that treats their form of this serious and life-shortening disease."
CF is caused by defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt (sodium and chloride) and water into or out of cells in a number of organs, including the lungs. The five mutations covered under today's approval (2789+5G— > A, 3272-26A— > G, 3849+10kbC— > T, 711+3A— > G, and E831X) cause CF and result in a moderate loss of chloride transport. People who have these mutations generally experience progressive lung function decline and other complications of the disease. All five of these mutations were evaluated as part of the previously disclosed Phase 3 EXPAND study in which the Kalydeco monotherapy arm met its primary efficacy endpoint and was generally well tolerated.
Kalydeco is now approved in the U.S. to treat people with CF ages 2 and older who have one of 38 ivacaftor-responsive mutations in the CFTR gene.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median predicted age of survival for a person born today with CF is 41 years, but currently the median age of death is 29 years.
About Kalydeco (ivacaftor)
Kalydeco (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, Kalydeco is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. Kalydeco is available as 150 mg tablets for adults and pediatric patients age 6 years and older, and is taken with fat-containing food. It is also available as 50 mg and 75 mg granules in pediatric patients ages 2 to less than 6 years and is administered with soft-food or liquid with fat-containing food.
People with CF who have specific mutations in the CFTR gene are currently benefiting from Kalydeco in 27 different countries across North America, Europe and Australia.
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.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for seven years in a row. For additional information and the latest updates from the company, please visit www.vrtx.com.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the statements in the second paragraph of the press release and statements regarding guidance for 2017 KALYDECO and ORKAMBI net product revenues. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the company's expectations regarding its 2017 KALYDECO and ORKAMBI net product revenues may be incorrect (including because one or more of the company's assumptions underlying its expectations may not be realized), that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
Source: Vertex Pharmaceuticals Incorporated
Posted: August 2017
- FDA Approves Kalydeco (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With CF as Early as Four Months of Age - September 25, 2020
- FDA Approves Kalydeco (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants with CF as Early as Six Months of Age - April 30, 2019
- FDA Approves Kalydeco (ivacaftor) for Cystic Fibrosis in Children Ages 12 to <24 Months with Certain Mutations in the CFTR Gene - August 15, 2018
- FDA Expands Approved Use of Kalydeco to Treat Additional Mutations of Cystic Fibrosis - May 17, 2017
- FDA Approves Kalydeco (ivacaftor) for Cystic Fibrosis in Children Ages 2 to 5 with CFTR Gene Mutations - March 18, 2015
- FDA Approves Kalydeco (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation - December 29, 2014
- FDA Approves Kalydeco to Treat Rare Form of Cystic Fibrosis - January 31, 2012
- FDA Grants Priority Review for Kalydeco (ivacaftor), the First Potential Medicine to Target the Underlying Cause of Cystic Fibrosis - December 15, 2011
- Vertex Submits Application for Priority Review and Approval of Kalydeco (VX-770, ivacaftor) in the U.S. as First Potential Medicine to Target the Underlying Cause of Cystic Fibrosis - October 26, 2011
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