The U.S. Food and Drug Administration (FDA) has approved KALYDECOTM (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), a rare, genetic disease. KALYDECO is approved for people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
KALYDECO was granted approval in approximately three months, making it one of the fastest FDA approvals ever and marking the second approval of a new medicine from Vertex in less than a year.
Vertex has established a financial assistance and patient support program to help get KALYDECO to eligible patients for whom it is prescribed. KALYDECO was discovered as part of collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.
Vertex is ready to support the introduction of KALYDECO and will begin shipping it to pharmacies in the United States this week.
Conference call for investors and media today: January 31, 2012, at 12:15 p.m. ET. The firm intends to offer more information on KALYDECO availability, price and the financial assistance and patient support program.
"More than 13 years ago we set out to change the lives of people with cystic fibrosis by developing new medicines that address the underlying cause of this rare and devastating disease," said Jeffrey Leiden, M.D., Ph.D., Vertex's incoming President and Chief Executive Officer. "KALYDECO represents a major advance in the treatment of cystic fibrosis for people with a specific type of this disease. But our work isn't done. With the ongoing support of doctors, patients and the Cystic Fibrosis Foundation, we're making progress toward our ultimate goal of developing additional medicines to help many more people with cystic fibrosis."
Prohost Comments: Since its establishment, Vertex has proven its mastery in targeted drug design and development. Accuracy is its advantage and determination to upgrade itself to fulfill a probable vow of developing drugs that act at the root-cause of the intractable debilitating diseases known until now they have yet to find treatments. We are confident that this firm will continue developing drugs that would benefit all patients with cystic fibrosis, which we know it is, indeed, developing them. We also believe that Vertex’ sales of HCV drug Incivek will not be left behind at anytime through the competition, as the firm is in the middle of a combination drugs that include Incivek and other drugs, all are oral with no alfa interferon. Such a firm will not go down in a world that appreciate far-reaching achievers. Vertex will continue to achieve and, we speculate that it will soon have a place among the Top-tier biotech companies and drug companies.
This is great news for science, for biotechnology and for the hope in treating many life-threatening and chronic debilitating diseases at the root-cause of their origin.