Biotechnology company Vertex Pharmaceuticals, Inc. recently announced that the European Union Committee for Medicinal Products for Human Use (CHMP) has released a positive opinion and recommendation for Marketing Authorization of breakthrough cystic fibrosis treatment ORKAMBI (lumacaftor/ivacaftor). The therapy is the first CFTR potentiator and CFTR corrector combination indicated for the treatment of the underlying cause of cystic fibrosis in patients 12 years and older, with two copies of the F508del mutation in their CFTR gene. Estimates show about 12,000 people in Europe have two copies of this mutation.
The same positive opinion and recommendation was also issued for the company’s request that KALYDECO (ivacaftor) be expanded to treat children between 2 to 5 years old, in addition to the present 6 years and up indication. Kalydeco 50mg and 75mg now feature a weight-based oral granule formulation that can be incorporated in soft food and liquid, which the CHMP believes would also prove beneficial in CF patients aged 6 to 11 years old weighing less than 25 kg. The CHMP added a positive Opinion recommending Kalydeco be used to treat CF patients aged 18 and older with the R117H gene mutation, which estimates show is present in about 250 adults in Europe.
“Today’s recommendations bring us closer to being able to help many more people with cystic fibrosis who currently do not have a medicine to treat the underlying cause of this chronic progressive disease,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “However, we know that there are many more people with CF who are still waiting for treatment, and we remain committed to continuing to work toward our goal of creating new medicines for the vast majority of people with cystic fibrosis.”
These CHMP Opinions and recommendations are crucial in the pursuit of approval from the European Commission. Generally, the Commission takes the CHMP’s reports into account and issues final Marketing Authorization within the European Union within 3 months. Upon approval, Vertex will have the green light to launch country-by-country reimbursement approval processes for each expanded indication.