Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) has announced that it has received approval from the EU for KALYDECO® granules label extension. The extension includes treatment of infants having cystic fibrosis aged 4 months and older who weigh over 5Kg.
Expanded label for KALYDECO®
The treatment will be in infants having R117H mutation or one of the gating mutation sin cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. The label extension is based on results from a cohort in the 24-week third phase open-label safety study (ARRIVAL). The study consisted of 6 children with CF aged four-month to less than 6 months with eligible gating mutation.
President and CEO of Vertex Reshma Kewalramani said that KALYDECO, the company’s first CFTR modulator, was approved 8 years ago to treat certain CF patients aged six years and above. Reshma said that with the approval of infants as young as 4 months will be eligible for early treatment, which is vital in managing CF. Further, the approval is a testament to the company’s commitment to researching and developing treatment options for CF patients.
KALYDECO available in Germany
Eligible patients in Germany will now access KALYDECO®, and it will also be available in countries that have signed long-term reimbursement agreements with Vertex. This includes Denmark, the UK, and the Republic of Ireland. The company will work closely with authorities in all the countries across Europe to gain access to an eligible patient. Already KALYDECO® has approval in Europe for CF patients aged 6 months and older and weighing 5 kg and above.
CF is a life-threatening and rare genetic disease that affects around 75,000 people globally. It is a progressive multi-system disease affecting the liver, lungs, GI tract, pancreases, sweat glands, and reproductive tract. The main cause is a defective or missing CFTR protein due to mutations in the CFTR gene. Children have to inherit two defective CFTR genes to have CF, and most people have at least one F508del mutation.