- If approved, KALYDECO (ivacaftor) will be the first and only medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation, as young as 6 months of age -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agencys (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KALYDECO (ivacaftor), to include the treatment of children and adolescents with cystic fibrosis (CF), ages 6 months and older weighing at least 5 kg who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
"Todays announcement is important for young people with CF, as early intervention and treatment of this devastating and progressive disease is key to keeping patients healthier longer," said Carmen Bozic, M.D., Executive Vice President and Chief Medical Officer at Vertex. "This milestone also brings us one step closer to achieving our ultimate goal of bringing medicines forward to all people with CF."
The European Commission will now review the CHMPs positive opinion, and should they issue a favorable adoption, KALYDECO (ivacaftor) will be the first and only approved medicine in Europe to treat the underlying cause of CF in patients ages 6 months and older with the R117H mutation. In countries where long-term reimbursement agreements have been secured, KALYDECO (ivacaftor), if approved, would be available to eligible patients shortly after Marketing Authorization. In Germany, the medicine would be available at Marketing Authorization. In all other countries, we will work closely with relevant authorities in Europe to secure access for eligible patients quickly.
In Europe, KALYDECO (ivacaftor) is already approved for the treatment of people with CF ages 18 and older with the R117H mutation, and children ages 6 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In 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 age of death is in the early 30s.
About KALYDECO (ivacaftor)
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, ivacaftor 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.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Story continues
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.
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, Dr. Bozics statements in the second paragraph of this press release, and statements regarding our expectations for the approval and availability of KALYDECO in Europe, and our plans for securing access to KALYDECO for eligible patients in Europe. 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 data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, risks related to obtaining and commercializing KALYDECO in Europe, 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 http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
View source version on businesswire.com: https://www.businesswire.com/news/home/20200501005167/en/
Contacts
Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com or +1 617-961-7163
Media: mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: +1 617-341-6992
Read more from the original source:
Vertex Receives European CHMP Positive Opinion for KALYDECO (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages 6 Months...
- Muscular .Dystrophy -Successful-treatment by acupressure , Ayurveda, Yoga [Last Updated On: May 10th, 2011] [Originally Added On: May 10th, 2011]
- Muscular Dystrophy Treatment - Todd Harrison Improving Balance [Last Updated On: May 16th, 2011] [Originally Added On: May 16th, 2011]
- Oculopharyngeal Muscular Dystrophy (OPMD): Exploring Causes and Treatment [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Muscular Dystrophy Treatment - Todd Harrison holding body weight [Last Updated On: May 21st, 2011] [Originally Added On: May 21st, 2011]
- David G VECTTOR Muscular Dystrophy Treatment double arm strength month 6 [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- David Gould Becker Muscular Dystrophy VECTTOR Treatment [Last Updated On: June 2nd, 2011] [Originally Added On: June 2nd, 2011]
- Muscular Dystrophy Halo [Last Updated On: June 3rd, 2011] [Originally Added On: June 3rd, 2011]
- Muscular Dystrophy VECTTOR Treatment Documentary - Todd's 6 Month Journey [Last Updated On: June 7th, 2011] [Originally Added On: June 7th, 2011]
- Muscular Dystrophy Treatment Results - David Gould - New Footage [Last Updated On: June 8th, 2011] [Originally Added On: June 8th, 2011]
- Muscular Dystrophy - Rewriting History with VECTTOR [Last Updated On: June 10th, 2011] [Originally Added On: June 10th, 2011]
- Muscular Dystrophy - David Gould 5 month VECTTOR treatment for BMD [Last Updated On: June 12th, 2011] [Originally Added On: June 12th, 2011]
- Duchenne's Muscular Dystrophy Stem Cell Treatment - Reelabs India [Last Updated On: June 14th, 2011] [Originally Added On: June 14th, 2011]
- Muscular Dystrophy VECTTOR Treatment - Todd Harrison/Luau Presentation [Last Updated On: June 16th, 2011] [Originally Added On: June 16th, 2011]
- Dr. William Rader - Muscular Dystrophy Breakthrough [Last Updated On: June 16th, 2011] [Originally Added On: June 16th, 2011]
- Muscular Dystrophy patient at Xcell-center - Nabeel Mohamed Abdulhusain, 46 years [Last Updated On: June 17th, 2011] [Originally Added On: June 17th, 2011]
- Charlie's Story: Duchenne Muscular Dystrophy Part 4 [Last Updated On: July 18th, 2011] [Originally Added On: July 18th, 2011]
- Muscular Dystrophy VECTTOR Treatment - David Gould/Luau Presentation [Last Updated On: July 19th, 2011] [Originally Added On: July 19th, 2011]
- Testimonial 4 of Muscular Dystrophy after Stem Cell Therapy [Last Updated On: August 5th, 2011] [Originally Added On: August 5th, 2011]
- Drug Combo Dynamic in Muscular Dystrophy [Last Updated On: August 9th, 2011] [Originally Added On: August 9th, 2011]
- Testimonial for Fetal Stem Cell Treatment of Duchenne Muscular Dystrophy [Last Updated On: August 25th, 2011] [Originally Added On: August 25th, 2011]
- Improvement seen in Duchenne Muscular Dystrophy after Stem Cell Therapy [Last Updated On: September 11th, 2011] [Originally Added On: September 11th, 2011]
- Muscular Dystrophy STS/VECTTOR treatment results [Last Updated On: September 24th, 2011] [Originally Added On: September 24th, 2011]
- Muscular Dystrophy VECTTOR Treatment Documentary - Todd's One Year Journey [Last Updated On: October 2nd, 2011] [Originally Added On: October 2nd, 2011]
- Muscular Dystrophy Treatment Day 18 - Todd Harrison's Improvement [Last Updated On: October 2nd, 2011] [Originally Added On: October 2nd, 2011]
- Wang Yisheng - Muscular Dystrophy Adult Stem Cell Patient [Last Updated On: October 9th, 2011] [Originally Added On: October 9th, 2011]
- Becker Muscular Dystrophy Miracle TREATMENT [Last Updated On: October 11th, 2011] [Originally Added On: October 11th, 2011]
- Muscular dystrophy patient_Kleber_Brazil.wmv [Last Updated On: October 12th, 2011] [Originally Added On: October 12th, 2011]
- Testimonial 1 of Muscular Dystrophy after Stem Cell Therapy - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- Becker Muscular Dystrophy STS Treatment - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- Potential Stem Cell treatment of Duchenne Muscular Dystrophy - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- Muscular Dystrophy treated by Dr Rajesh Shah at Life Force - Video [Last Updated On: October 22nd, 2011] [Originally Added On: October 22nd, 2011]
- Defying Muscular Dystrophy - I Made It - Video [Last Updated On: October 23rd, 2011] [Originally Added On: October 23rd, 2011]
- Becker Muscular Dystrophy AMAZING treatment results - Video [Last Updated On: October 27th, 2011] [Originally Added On: October 27th, 2011]
- First targeted treatment success for Duchenne muscular dystrophy - Video [Last Updated On: November 6th, 2011] [Originally Added On: November 6th, 2011]
- 125 Days of VECTTOR Treatment Progress [Last Updated On: November 18th, 2011] [Originally Added On: November 18th, 2011]
- Becker Muscular Dystrophy WALKING ABILITY IMPROVED - Video [Last Updated On: November 25th, 2011] [Originally Added On: November 25th, 2011]
- PT Muscular Dystrophy Treatment Results - Video [Last Updated On: December 5th, 2011] [Originally Added On: December 5th, 2011]
- Duchenne Muscular Dystrophy Treated by Cellmedicine - Video [Last Updated On: January 8th, 2012] [Originally Added On: January 8th, 2012]
- muscular DYSTROPHY treatment IN HOMEOPATH.mp4 - Video [Last Updated On: January 28th, 2012] [Originally Added On: January 28th, 2012]
- Giulio's strategy is to cure dystrophy with stem cell treatment - Video [Last Updated On: January 31st, 2012] [Originally Added On: January 31st, 2012]
- Parent Project Muscular Dystrophy is a Featured Nonprofit Selected by Webkinz(TM) Foundation [Last Updated On: February 1st, 2012] [Originally Added On: February 1st, 2012]
- MDA Awards More Than $12 Million in Grants to Advance Neuromuscular Disease Research [Last Updated On: February 1st, 2012] [Originally Added On: February 1st, 2012]
- "For treatment we will have in the future" - Video [Last Updated On: February 2nd, 2012] [Originally Added On: February 2nd, 2012]
- Renowned Pediatric Cardiology Physician-Scientist Linda Cripe Joins Nationwide Children's Hospital [Last Updated On: February 3rd, 2012] [Originally Added On: February 3rd, 2012]
- JumpStart Invests $250,000 in Milo Biotechnology [Last Updated On: February 14th, 2012] [Originally Added On: February 14th, 2012]
- When nerve meets muscle, biglycan seals the deal [Last Updated On: February 14th, 2012] [Originally Added On: February 14th, 2012]
- Medical clinics offer help for Big Island children [Last Updated On: February 16th, 2012] [Originally Added On: February 16th, 2012]
- Dateline Long Beach: The Aquatic Center brings swim therapy to disabled [Last Updated On: February 19th, 2012] [Originally Added On: February 19th, 2012]
- Parent Project Muscular Dystrophy Awards $500,000 to Tivorsan Pharmaceuticals [Last Updated On: February 21st, 2012] [Originally Added On: February 21st, 2012]
- Cure Duchenne Announces Three New Funded Research Projects to Help Develop Treatments and Find a Cure for Duchenne ... [Last Updated On: February 21st, 2012] [Originally Added On: February 21st, 2012]
- Ligand Licenses DARA Program to Retrophin [Last Updated On: February 21st, 2012] [Originally Added On: February 21st, 2012]
- AVI BioPharma Regains NASDAQ Compliance [Last Updated On: February 23rd, 2012] [Originally Added On: February 23rd, 2012]
- Scientists create potent molecules aimed at treating muscular dystrophy [Last Updated On: February 23rd, 2012] [Originally Added On: February 23rd, 2012]
- AVI BioPharma Announces Fourth Quarter and Full Year 2011 Financial Results and Corporate Update Conference Call [Last Updated On: February 23rd, 2012] [Originally Added On: February 23rd, 2012]
- Pembroke's Christine McSherry is an 'Inspirational Woman' [Last Updated On: February 25th, 2012] [Originally Added On: February 25th, 2012]
- The Rare Clinical Diseases Research Network [Last Updated On: February 25th, 2012] [Originally Added On: February 25th, 2012]
- A Solution for Duchenne Muscular Dystrophy? - Research Summary [Last Updated On: February 28th, 2012] [Originally Added On: February 28th, 2012]
- A Solution for Duchenne Muscular Dystrophy? -- In Depth Doctor's Interview [Last Updated On: February 28th, 2012] [Originally Added On: February 28th, 2012]
- Antisense oligonucleotides make sense in myotonic dystrophy [Last Updated On: February 28th, 2012] [Originally Added On: February 28th, 2012]
- A life of dependence [Last Updated On: February 29th, 2012] [Originally Added On: February 29th, 2012]
- Nationwide Children's Hospital neuromuscular disorder podcasts now available on iTunes [Last Updated On: March 2nd, 2012] [Originally Added On: March 2nd, 2012]
- Next-generation DNA sequencing to improve diagnosis for muscular dystrophy [Last Updated On: March 6th, 2012] [Originally Added On: March 6th, 2012]
- The Dire Limits of Health Care [Last Updated On: March 7th, 2012] [Originally Added On: March 7th, 2012]
- AVI BioPharma Announces Late-Breaker Oral Presentation of Phase IIb DMD Study at 2012 AAN Annual Meeting in April [Last Updated On: March 12th, 2012] [Originally Added On: March 12th, 2012]
- Cataracts affect millions of people around the globe, and for many of us they will be a normal part of our aging ... [Last Updated On: March 14th, 2012] [Originally Added On: March 14th, 2012]
- Your Health: Duchenne's Muscular Dystrophy [Last Updated On: March 16th, 2012] [Originally Added On: March 16th, 2012]
- Newborn Screening for Duchenne Muscular Dystrophy Shows Promise as an International Model [Last Updated On: March 20th, 2012] [Originally Added On: March 20th, 2012]
- Newborn screening for DMD shows promise as an international model [Last Updated On: March 20th, 2012] [Originally Added On: March 20th, 2012]
- Rhenovia launches drug discovery for Huntington's disease [Last Updated On: March 20th, 2012] [Originally Added On: March 20th, 2012]
- Halo Therapeutics Reports Favorable Independent Review of Lead Drug Candidate HT-100 [Last Updated On: March 21st, 2012] [Originally Added On: March 21st, 2012]
- Invasive treatment strategy may increase survival for patients with certain neuromuscular disorder [Last Updated On: March 28th, 2012] [Originally Added On: March 28th, 2012]
- Parent Project Muscular Dystrophy Endorses FAST Act Legislation to Expedite FDA Review of Life-Saving Therapies [Last Updated On: March 29th, 2012] [Originally Added On: March 29th, 2012]
- AVI BioPharma Announces Conference Call and Webcast on Monday, April 2, 2012, to Discuss Top-Line Data Results From ... [Last Updated On: March 31st, 2012] [Originally Added On: March 31st, 2012]
- Drug for rare disease may lift AVI BioPharma shares: Barron's [Last Updated On: April 2nd, 2012] [Originally Added On: April 2nd, 2012]
- AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 ... [Last Updated On: April 2nd, 2012] [Originally Added On: April 2nd, 2012]
- U.S. Stock Futures Little Changed Before Factory Report [Last Updated On: April 2nd, 2012] [Originally Added On: April 2nd, 2012]
- Leading experts on congenital muscular dystrophy convene at University of Nevada, Reno [Last Updated On: April 20th, 2012] [Originally Added On: April 20th, 2012]
- Nature Publishes Work Utilizing N-Gene's Core Technology to Advance the Treatment of Duchenne Muscular Dystrophy [Last Updated On: April 20th, 2012] [Originally Added On: April 20th, 2012]
- Getting the boots filled [Last Updated On: April 29th, 2012] [Originally Added On: April 29th, 2012]
- Local business, civic leaders 'arrested' for MDA fundraiser [Last Updated On: May 3rd, 2012] [Originally Added On: May 3rd, 2012]