Vertex Pharmaceuticals, a global biotechnology company,  has signed a letter of intent with the pan-Canadian Pricing Alliance (pCPA) to enable the public reimbursement of Kalydeco (ivacaftor) for the treatment of eligible Canadians with cystic fibrosis (CF) ages 6 and older who have the G551D mutation. The letter of intent represents an agreement in principle with the pCPA regarding the public reimbursement of Kalydeco in Canada. However, before patients can get access through public reimbursement, each participating province or territory must decide to reimburse Kalydeco through its individual drug programme. In Canada, there are approximately 100 people ages 6 and older with this specific mutation. Kalydeco is the first medicine to treat the underlying cause of cystic fibrosis for people with the G551D mutation in the CFTR gene.

Cystic fibrosis is a rare genetic disease for which there is no cure. CF is caused by defective or missing CFTR proteins that result from mutations in the CFTR gene. The defective function or absence of CFTR proteins in people with CF results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs. Kalydeco facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein.

"The letter of intent signed with the pan-Canadian Pricing Alliance is an important step toward eligible Canadians receiving Kalydeco through public reimbursement. However, our work is not complete until each province has added Kalydeco to its individual drug programme to ensure people can get access to this medicine," said Stuart Arbuckle, executive vice president and chief commercial officer for Vertex. "We share the urgency of the CF community to bring this process to a successful conclusion, and we will work as quickly as the provinces are able to so that people can receive Kalydeco without delay."

The process to add Kalydeco to drug programmes at the provincial and territorial level is ongoing. The letter of intent does not include the province of Quebec, which does not participate in the pCPA process.

Kalydeco is now available to eligible people with CF in more than 15 countries around the world, including the United States, England, Scotland, Northern Ireland, Wales, the Republic of Ireland, France, Germany, the Netherlands, Austria, Denmark, Sweden, Norway, Greece, Italy and Switzerland. Kalydeco was approved in Australia in July 2013, however public reimbursement discussions are ongoing.

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 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 US Food and Drug Administration in January 2012 for use in people with CF ages 6 and older who have at least one copy of the G551D mutation and in February 2014 for use in people with CF ages 6 and older who have the following additional CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. In Canada, Kalydeco was first approved in November 2012 for use in people with CF ages 6 and older who have at least one copy of the G551D mutation and in June 2014 for use in people with CF ages 6 and older who have the following additional CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D and G970R.

Kalydeco was approved by the European Medicines Agency in July 2012 and by the Therapeutic Goods Administration in Australia in July 2013 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 commercialise Kalydeco.

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. Today, the median predicted age of survival for a person with CF is between 34 and 47 years, but the median age of death remains in the mid-20s.

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 more than 1,900 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR protein at the cell surface. The defective function or absence of CFTR proteins in people with CF results in poor flow of salt and water into and 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.

Vertex initiated its CF research programme in 1998 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. This collaboration was expanded to support the accelerated discovery and development of Vertex's CFTR modulators.