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 <a xmlns="http://www.w3.org/1999/xhtml" href="http://cts.businesswire.com/ct/CT?id=smartlink&amp;url=http%3A%2F%2Fwww.vrtx.com&amp;esheet=51561303&amp;newsitemid=20170517006271&amp;lan=en-US&amp;anchor=Vertex+Pharmaceuticals+Incorporated&amp;index=1&amp;md5=03a9e947af695326db2fabaa2dba2c2b" rel="nofollow" rel="nofollow">Vertex 
 Pharmaceuticals Incorporated</a> VRTX today announced that the 
 U.S. Food and Drug Administration (FDA) has approved KALYDECO® 
 (ivacaftor) for use in people with cystic fibrosis (CF) ages 2 and older 
 who have one of 23 residual function mutations in the cystic fibrosis 
 transmembrane conductance regulator (CFTR) gene. This precision 
 medicine decision is based on analyses of in vitro data and is 
 supported by more than five years of real-world clinical data that 
 demonstrate KALYDECO's strong safety and efficacy profile. More than 900 
 people ages 2 and older in the U.S. have one of these mutations. Based 
 on this approval, Vertex today increased its guidance for 2017 product 
 revenues of KALYDECO to a range of $740 million to $770 million.
 
 
 In addition to the mutations added to the label today, Vertex is 
 continuing discussions with the FDA concerning the approval for 
 additional people who have mutations responsive to KALYDECO, including 
 one of five "splice" mutations. These five mutations were evaluated as 
 part of the previously disclosed <a xmlns="http://www.w3.org/1999/xhtml" href="http://cts.businesswire.com/ct/CT?id=smartlink&amp;url=http%3A%2F%2Finvestors.vrtx.com%2Freleasedetail.cfm%3FReleaseID%3D1019156&amp;esheet=51561303&amp;newsitemid=20170517006271&amp;lan=en-US&amp;anchor=Phase+3+EXPAND+study&amp;index=2&amp;md5=395f6028eb9b052d06042607a9fd311d" rel="nofollow" rel="nofollow">Phase 
 3 EXPAND study</a> in which the KALYDECO monotherapy arm met its primary 
 efficacy endpoint while being generally well tolerated. More than 600 
 people ages 2 and older in the U.S. have one of these mutations.
 
 
 "Five years ago, KALYDECO became the first medicine to treat the 
 underlying cause of CF. Since then, we have continued to invest in 
 studies to improve the understanding of how this important medicine may 
 benefit others with this serious and life-shortening disease," said 
 Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical 
 Officer at Vertex. "We are encouraged by the FDA's willingness to 
 explore innovative ways to make highly effective medicines like KALYDECO 
 with a well-established safety profile available to more people who are 
 in urgent need. We will continue to work closely with the FDA to bring 
 KALYDECO to more people with responsive mutations who are still in need 
 as rapidly as possible."
 
 
 "CF treatment has advanced rapidly, but there is need for broader access 
 to these important medicines and development of additional medicines 
 remains urgent," said Patrick Flume, M.D., Director of the Medical 
 University of South Carolina Cystic Fibrosis Center. "The use of in 
 vitro data to support this approval is an important step forward in 
 making medicines like KALYDECO available to more patients, especially 
 those with rare mutations."
 
 
 Based on clinical data from numerous prior studies and a demonstrated in 
 vitro response employing a well-established, analytically validated 
 method, KALYDECO is now approved to treat people with CF ages 2 and 
 older who have one of 33 mutations in the CFTR gene.
 
 
 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 (chloride) 
 and water into and out of the cell in a number of organs, including the 
 lungs. Chloride transport is a marker of the function of the CFTR 
 protein at the cell surface. As with the other mutations for which 
 KALYDECO is approved, mutations covered under today's approval are known 
 to have some CFTR protein at the cell surface and have shown in vitro 
 increases in chloride transport in response to KALYDECO by at least 10 
 percent of normal over baseline. Similar to the R117H mutation 
 for which KALYDECO was previously approved, these additional residual 
 function mutations result in a moderate loss of CFTR chloride transport, 
 and people with these mutations generally have progressive lung function 
 decline and other complications of CF.
 
 
 Pipeline Progress for Residual Function Mutations
 
 
 On March 28, 2017, Vertex announced positive results from a Phase 3 
 study that evaluated tezacaftor/ivacaftor combination treatment in 
 people who have one mutation that results in residual CFTR function and 
 one F508del mutation. The study met its primary endpoint and the 
 combination was generally well tolerated. Vertex plans to submit these 
 data as part of a New Drug Application (NDA) to the U.S. Food and Drug 
 Administration (FDA) and a Marketing Authorization Application (MAA) to 
 the European Medicines Agency (EMA) for the tezacaftor/ivacaftor 
 combination early in the third quarter of 2017.
 
 
 About Cystic Fibrosis and KALYDECO® (ivacaftor)
 
 
 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 the median 
 age of death is 27 years.
 
 
 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. In adults and pediatric patients age 6 
 years and older one 150 mg tablet is taken orally every 12 hours with 
 fat-containing food. Pediatric patients ages 2 to less than 6 years who 
 weigh less than 14 kg take one 50 mg packet of oral granules mixed with 
 1 teaspoon (5 mL) of soft food or liquid administered orally every 12 
 hours with fat-containing food. Pediatric patients ages 2 to less than 6 
 years of age who weigh 14 kg or greater take one 75 mg packet of oral 
 granules with 1 teaspoon (5 mL) of soft food or liquid administered 
 orally every 12 hours 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.
 
 
 KALYDECO® (ivacaftor) INDICATION AND 
 IMPORTANT SAFETY INFORMATION
 
 
 KALYDECO (ivacaftor) is a prescription medicine used for the treatment 
 of cystic fibrosis (CF) in patients age 2 years and older who have at 
 least one mutation in the CF gene that is responsive to KALYDECO. 
 Patients should talk to their doctor to learn if they have an indicated 
 CF gene mutation. It is not known if KALYDECO is safe and effective in 
 children under 2 years of age.
 
 
 Patients should not take KALYDECO if they are taking certain 
 medicines or herbal supplements such as: the antibiotics 
 rifampin or rifabutin; seizure medications such as phenobarbital, 
 carbamazepine, or phenytoin; or St. John's wort.
 
 
 Before taking KALYDECO, patients should tell their doctor if they: 
 have liver or kidney problems; drink grapefruit juice, or eat grapefruit 
 or Seville oranges; are pregnant or plan to become pregnant because it 
 is not known if KALYDECO will harm an unborn baby; and are breastfeeding 
 or planning to breastfeed because is not known if KALYDECO passes into 
 breast milk.
 
 
 KALYDECO may affect the way other medicines work, and other medicines 
 may affect how KALYDECO works. Therefore the dose of KALYDECO may 
 need to be adjusted when taken with certain medications. Patients should 
 especially tell their doctor if they take antifungal medications such as 
 ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; 
 or antibiotics such as telithromycin, clarithromycin, or erythromycin.
 
 
 KALYDECO can cause dizziness in some people who take it. Patients should 
 not drive a car, use machinery, or do anything that needs them to be 
 alert until they know how KALYDECO affects them. Patients should avoid 
 food containing grapefruit or Seville oranges while taking KALYDECO.
 
 
 KALYDECO can cause serious side effects including:
 
 
 High liver enzymes in the blood have been reported in patients 
 receiving KALYDECO. The patient's doctor will do blood tests to 
 check their liver before starting KALYDECO, every 3 months during the 
 first year of taking KALYDECO, and every year while taking KALYDECO. For 
 patients who have had high liver enzymes in the past, the doctor may do 
 blood tests to check the liver more often. Patients should call their 
 doctor right away if they have any of the following symptoms of liver 
 problems: pain or discomfort in the upper right stomach (abdominal) 
 area; yellowing of their skin or the white part of their eyes; loss of 
 appetite; nausea or vomiting; or dark, amber-colored urine.
 
 
 Abnormality of the eye lens (cataract) has been noted in some children 
 and adolescents receiving KALYDECO. The patient's doctor should perform 
 eye examinations prior to and during treatment with KALYDECO to look for 
 cataracts. The most common side effects include headache; upper 
 respiratory tract infection (common cold), which includes sore throat, 
 nasal or sinus congestion, and runny nose; stomach (abdominal) pain; 
 diarrhea; rash; nausea; and dizziness.
 
 
 These are not all the possible side effects of KALYDECO.
 
 
 Please <a xmlns="http://www.w3.org/1999/xhtml" href="http://cts.businesswire.com/ct/CT?id=smartlink&amp;url=http%3A%2F%2Fpi.vrtx.com%2Ffiles%2Fuspi_ivacaftor.pdf&amp;esheet=51561303&amp;newsitemid=20170517006271&amp;lan=en-US&amp;anchor=click+here&amp;index=3&amp;md5=443af1bd953fa63db251e5cf9162df51" rel="nofollow" rel="nofollow">click 
 here</a> to see the full Prescribing Information for KALYDECO.
 
 
 Collaborative History with Cystic Fibrosis Foundation Therapeutics, 
 Inc. (CFFT)
 
 
 Vertex initiated its CF research program in 2000 as part of a 
 collaboration with CFFT, the nonprofit drug discovery and development 
 affiliate of the Cystic Fibrosis Foundation. KALYDECO (ivacaftor) was 
 discovered by Vertex as part of this collaboration.
 
 
 About Vertex
 
 
 Vertex is a global biotechnology company that aims to discover, develop 
 and commercialize innovative medicines so people with serious diseases 
 can lead better lives. In addition to our clinical development programs 
 focused on cystic fibrosis, Vertex has more than a dozen ongoing 
 research programs aimed at other serious and life-threatening diseases.
 
 
 Founded in 1989 in Cambridge, Mass., Vertex today has research and 
 development sites and commercial offices in the United 
 States, Europe, Canada and Australia. For seven years in a row, Science magazine 
 has named Vertex one of its Top Employers in the life sciences. For 
 additional information and the latest updates from the company, please 
 visit <a xmlns="http://www.w3.org/1999/xhtml" href="http://cts.businesswire.com/ct/CT?id=smartlink&amp;url=http%3A%2F%2Fwww.vrtx.com&amp;esheet=51561303&amp;newsitemid=20170517006271&amp;lan=en-US&amp;anchor=www.vrtx.com&amp;index=4&amp;md5=1d8b12c9e82a9b9fe0890e2dbc88b327" rel="nofollow" rel="nofollow">www.vrtx.com</a>.
 
 
 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 third and fourth paragraphs of the 
 press release and statements regarding (i) the potential approval of 
 KALYDECO for patients with "splice" mutations, (ii) Vertex's updated 
 guidance for KALYDECO 2017 net product revenues and (iii) Vertex's plans 
 to submit an NDA and MAA for tezacaftor/ivacaftor. While Vertex believes 
 the forward-looking statements contained in this press release are 
 accurate, these forward-looking statements represent the company&#39;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&#39;s expectations regarding its 2017 KALYDECO net product revenues 
 may be incorrect (including because one or more of the company&#39;s 
 assumptions underlying its expectations may not be realized), that data 
 from the company&#39;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&#39;s annual 
 report and quarterly reports filed with the Securities and Exchange 
 Commission and available through the company&#39;s website at <a xmlns="http://www.w3.org/1999/xhtml" href="http://cts.businesswire.com/ct/CT?id=smartlink&amp;url=http%3A%2F%2Fwww.vrtx.com&amp;esheet=51561303&amp;newsitemid=20170517006271&amp;lan=en-US&amp;anchor=www.vrtx.com&amp;index=5&amp;md5=85f8577f16cb0539e28c57fb68784de9" rel="nofollow" rel="nofollow">www.vrtx.com</a>. 
 Vertex disclaims any obligation to update the information contained in 
 this press release as new information becomes available.
 
 
 (VRTX-GEN)
 
 
 
 
 
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