Orkambi ready to ship WITHIN DAYS!!!! Here's the Vertex press release. Sorry, had to cut and paste from email (I subscribe for updates) since their webpage is crashed:
FDA Approves ORKAMBI(TM) (lumacaftor/ivacaftor) - the First Medicine to Treat the Underlying Cause of Cystic Fibrosis for People Ages 12 and Older with Two Copies of the F508del Mutation
-Approximately 8,500 people in the U.S. are ages 12 and older and have two copies of the F508del mutation, the most common genetic form of the disease-
BOSTON--(BUSINESS WIRE)--
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved ORKAMBI™ (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older with two copies of the
F508del mutation. It is only indicated for these patients, who can be identified with a genetic test.
Cystic fibrosis is a rare, life-threatening genetic disease. People with two copies of the
F508del mutation represent the largest group of people with CF. Of the 30,000 people in the United States with CF, approximately 8,500 ages 12 and older have two copies of the
F508del mutation. ORKAMBI will be available for shipment to specialty pharmacies in the United States within days.
"Today is a remarkable day for science, medicine and the CF community," said Jeffrey Leiden, M.D., Ph.D., Vertex's Chairman, President and Chief Executive Officer. "More than 15 years ago, our scientists set out to discover and develop medicines to treat the underlying cause of cystic fibrosis. Today, the approval of ORKAMBI represents a fundamental change in the treatment of the most common form of CF, marking significant progress for us and for the entire CF community. While we celebrate this important step forward, we also recognize that two out of three patients in the U.S. still do not have a medicine to treat the underlying cause of their disease. We share their urgency and are committed to continuing our significant investment in research and development to discover new medicines for them and to improve upon what we offer patients today."
Vertex will host an investor conference call on Thursday, July 2, at 2:15 p.m. ET. to provide more information on the approval of ORKAMBI.
The approval of ORKAMBI was based on data from two Phase 3 studies (TRAFFIC and TRANSPORT) that enrolled more than 1,100 people with CF ages 12 and older with two copies of the
F508del mutation. Patients treated with ORKAMBI experienced statistically significant improvements in lung function. Patients also experienced reductions in pulmonary exacerbations and improvements in body mass index (BMI). The most common adverse events included shortness of breath and/or chest tightness, upper respiratory tract infection (common cold) and gastrointestinal symptoms (including nausea, diarrhea, or gas).
Vertex continues to invest in CF research and development with the goal of treating the vast majority of people with the disease and enhancing the benefit for those we treat. Multiple Phase 2 and Phase 3 clinical studies are in progress and Vertex has an ongoing research program focused on discovering new CF medicines.
"In 1998, Vertex and the CF Foundation embarked on a scientific challenge that many believed would be impossible - to discover medicines that treat the cause of CF," said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. "Today's approval is a milestone for the CF community. We congratulate Vertex for their success in developing new CF medicines and are pleased with their continuing commitment to help all eligible patients get access to these medicines."
Helping Patients Access ORKAMBI
The people who work at Vertex understand that medicines can only help patients who can get them. The Vertex Guidance & Patient Support (Vertex GPS™) program provides a dedicated team of Vertex employees who help eligible patients who have been prescribed our medicines within their labeled indications understand their insurance benefits and the resources that are available to help them.
Vertex also offers a co-pay assistance program for patients with commercial insurance coverage and a free medicine program for qualifying patients who are uninsured and who meet certain income and other eligibility criteria. More information is available by visiting
www.VertexGPS.com or by calling
1-877-752-5933.
About CF and ORKAMBI
Cystic fibrosis is a rare genetic disease that is caused by defective or missing cystic fibrosis transmembrane conductance regulatory (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. In people with two copies of the
F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little to no CFTR protein at the cell surface. Patients with two copies of the
F508del mutation are easily identified by a simple genetic test.
ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the
F508del CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. ORKAMBI is taken every 12 hours - once in the morning and once in the evening.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR ORKAMBI™ (lumacaftor/ivacaftor) TABLETS
ORKAMBI is a combination of lumacaftor and ivacaftor indicated for the treatment of cystic fibrosis (CF) in patients age 12 years and older who are homozygous for the
F508del mutation in the
CFTR gene. The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the
F508del mutation
.
Worsening of liver function, including hepatic encephalopathy, in patients with advanced liver disease has been reported in some patients with CF while receiving ORKAMBI. ORKAMBI should be used with caution in patients with advanced liver disease and only if the benefits are expected to outweigh the risks. If ORKAMBI is used in these patients, the patients should be closely monitored and the dose reduced.
Serious adverse reactions related to elevated transaminases have been reported in patients with CF receiving ORKAMBI and, in some instances, associated with concomitant elevations in total serum bilirubin. It is recommended that ALT, AST, and bilirubin be assessed prior to initiating ORKAMBI, every 3 months during the first year of treatment, and annually thereafter. For patients with a history of ALT, AST, or bilirubin elevations, more frequent monitoring should be considered. Patients who develop increased ALT, AST, or bilirubin should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST greater than 5x upper limit of normal (ULN) when not associated with elevated bilirubin. Dosing should also be interrupted in patients with ALT or AST elevations greater than 3x ULN when associated with bilirubin elevations greater than 2x ULN. Following resolution of transaminase elevations, consider the benefits and risks of resuming dosing.
Respiratory events (e.g., chest discomfort, shortness of breath, and chest tightness) were observed more commonly in patients during initiation of ORKAMBI compared to those who received placebo. Clinical experience in patients with percent predicted FEV[SUB]
1[/SUB] < 40 is limited, and additional monitoring of these patients is recommended during initiation of therapy.
TO BE CONTINUED