Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) Receives European Commission KALYDECO Approval For Treatment Of CF In Children

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) has announced that the company has been granted approval by the European Commission for the KALYDECO (ivacaftor) label extension to include treatment of adolescents and children with cystic fibrosis (CF).

Label extension of KALYDECO includes six-month infants

The extension includes children of at least six months weighing more than 5kg, having the R117H mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR). This is the most obvious residual function gene alteration that underlies cystic fibrosis

Reshma Kewalramani, the President and CEO of Vertex, indicated that KALYDECO became the first and only approved treatment eight years ago to treat the cause of CF in patients with certain mutations. He added that since receiving the approval, the company has been working towards ensuring that as many patients with cystic fibrosis benefit from the treatment. Reshma added that with the approval of the label extension, now it implies that more than 500 young people across Europe who have been waiting for a treatment alternative will easily access KALYDECO.

KALYDECO available immediately to patients in Germany

Following the approval, for now, KALYDECO will be available immediately to more needy patients in Germany. The company will then extend availability to other countries where it had previously secured long-term reimbursement agreements. The company will work closely with the relevant authorities and regulators to quickly secure access to eligible patients for treatment with KALYDECO.

Currently, KALYDECO is approved across Europe for the treatment of cystic fibrosis patients aged 18 years and above having the R117H mutation. Equally, it is being used for the treatment of children aged six months and older who weight around 5 kg and above that have either of the following CFTR gene mutation. The mutations include G178R, G551D, G1244E, G1349D, G551S, S1255P, S1251N, S549R, and S549.

Cystic fibrosis is a life-shortening hereditary disease that affects lungs, GI tract, liver, sweat glands, sinuses, reproductive tract, and pancreas affecting around 75,000 people globally. The disease is caused by missing or defective CFTR protein, which results in the CFTR gene mutation.