Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that 10 abstracts from its cystic fibrosis (CF) research and development program will be presented at the 26th Annual North American Cystic Fibrosis Conference (NACFC) in Orlando, Fla., October 11 to 13, 2012. Previously announced data from a Phase 2 study of VX-809 combined with ivacaftor in people with the most common mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR) gene, F508del, will be presented for the first time. Additional data from Vertex’s work to discover and develop medicines that target the underlying cause of CF will also be presented, including data on KALYDECO™ (ivacaftor) in people with CF who have the G551D mutation. The accepted abstracts are now available on the NACFC website at: https://www.nacfconference.org/. Vertex Abstracts (Oral presentations will also be presented as posters) 1. “Hyperpolarized Gas MRI of Ivacaftor Therapy in Persons with Cystic Fibrosis and the G551D-CFTR Mutation.” Poster #196. An oral presentation is scheduled for October 11, 2012, 10:55 a.m. EDT. 2. “The Investigational CFTR Corrector, VX-809 (Lumacaftor) Co-Administered with the Oral Potentiator Ivacaftor Improved CFTR and Lung Function in F508DEL Homozygous Patients: Phase II Study Results.” Poster #260. An oral presentation is scheduled for October 11, 2012, 11:40 a.m. EDT. 3. “Identification and Characterization of CFTR Corrector VRT-534 (C-18).” Poster #30. 4. “Ivacaftor Potentiates Multiple Mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Forms.” Poster #31. 5. “Long-Term Safety and Efficacy of Ivacaftor in Persons with Cystic Fibrosis who have the G551D-CFTR Mutation.” Poster #211. 6. “Patient-Reported Outcomes in Phase 3 Trials of Ivacaftor in Subjects with CF who have the G551D-CFTR Mutation.” Poster #212. 7. “Nutritional Status Measures Among Persons with CF Carrying the G551D-CFTR Mutation who Received Ivacaftor or Placebo in Phase 3 Clinical Trials.” Poster #214. 8. “Lung Clearance Index to Evaluate the Effect of Ivacaftor on Lung Function in Subjects with CF who have the G551D-CFTR Mutation and Mild Lung Disease.” Poster #249. 9. “Exposure-Response Relationship for FEV 1 and Sweat Chloride in Patients with Cystic Fibrosis Treated with Ivacaftor, a CFTR Potentiator.” Poster #235. 10. “Clinical Pharmacology Profile of Ivacaftor, a CFTR Potentiator.” Poster #236. About KALYDECO KALYDECO™ (ivacaftor) is the first treatment to target the underlying cause of CF in people with the G551D mutation in the CFTR gene. Known as a CFTR potentiator, KALYDECO is an oral medicine that aims to help the CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways. KALYDECO (150mg, q12h) was first approved by the U.S. Food and Drug Administration in January 2012 and by the European Medicines Agency in July 2012, for use in people with CF ages 6 and older who have at least one copy of the G551D mutation in the CFTR gene. Vertex retains worldwide rights to develop and commercialize KALYDECO. KALYDECO is under Priority Review by the Therapeutic Product Directorate (TPD) of Health Canada, and an application for review has been submitted to the Therapeutic Goods Administration (TGA) of Australia.
Indication and Important Safety Information for KALYDECO (ivacaftor)KALYDECO (150mg tablets) is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene. KALYDECO is not for use in people with CF due to other mutations in the CFTR gene. It is not effective in CF patients with two copies of the F508del mutation (F508del/F508del) in the CFTR gene. High liver enzymes (transaminases, ALT and AST) have been reported in patients receiving KALYDECO. It is recommended that ALT and AST be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal. Following resolution of transaminase elevations, consider the benefits and risks of resuming KALYDECO dosing. Moderate transaminase elevations are common in subjects with CF. Overall, the incidence and clinical features of transaminase elevations in clinical trials was similar between subjects in the KALYDECO and placebo treatment groups. In the subset of patients with a medical history of elevated transaminases, increased ALT or AST have been reported more frequently in patients receiving KALYDECO compared to placebo. Use of KALYDECO with medicines that are strong CYP3A inducers such as the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John's Wort substantially decreases exposure of KALYDECO which may diminish effectiveness. Therefore, co-administration is not recommended. The dose of KALYDECO must be adjusted when concomitantly used with potent and moderate CYP3A inhibitors. KALYDECO can cause serious adverse reactions including abdominal pain and high liver enzymes in the blood. The most common side effects associated with KALYDECO include headache; upper respiratory tract infection (the common cold), including sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; and dizziness. These are not all the possible side effects of KALYDECO. A list of the adverse reactions can be found in the full product labeling for each country where KALYDECO is approved. Patients should tell their healthcare providers about any side effect that bothers them or doesn't go away.
Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, rheumatoid arthritis and other life-threatening diseases.Founded more than 20 years ago in Cambridge, Mass., we now have ongoing worldwide research programs and sites in the U.S., U.K. and Canada. Today, Vertex has more than 2,000 employees around the world, and for three years in a row, Science magazine has named Vertex one of its Top Employers in the life sciences. VRTX – GEN