In the wake of positive news about new combination therapies involving the Vertex drug Kalydeco (ivacaftor) for people with cystic fibrosis (CF), the drug recently received a positive recommendation for approval by the European Committee for Medicinal Products for Human Use (CHMP) for patients aged six years and older with one of eight non-G551D gating mutations. The therapeutic was recently proven to be effective in the treatment of a broader CF patient group in the United States, and the expansion to Europe may mean treatment for hundreds of people with specific non-gating mutations.
This opinion from CHMP is not a final decision, but instead will now be taken into consideration by the authorities responsible for the medication’s approval for the European Union, at the European Commission. After an eventual approval, each country will decide individually whether they want to provide it to patients or not.
“It is becoming increasingly important for people with CF to know what their genotype is because, more and more, access to new treatments may be dependent on the precise nature of the mutation. The Trust believes it is not only important but a fundamental right for everyone with cystic fibrosis to know their genotype wherever possible,” explained the director of research at the Cystic Fibrosis Trust, Dr. Janet Allen, as she emphasized the need for personalized medicine.
Kalydeco, the first drug in the world to target the root cause of CF, was first licensed for people with at least one copy of the G551d mutation, which means around four percent of the patients with CF in the UK. All around Europe, about 250 people with G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D mutations could benefit from the approval. The first step into Europe occurred earlier at the 37th European Cystic Fibrosis Society Conference, held June 11-14 in Sweden, where Vertex presented the data from multiple clinical trials assessing the effectiveness of Kalydeco.
The company has also signed a letter of intent with the pan-Canadian Pricing Alliance to enable public reimbursement of Kalydeco for the treatment of eligible Canadians with more than six years old with the G551D gene mutation.