Vertex Pharmaceuticals, a global biotechnology company developing and commercializing innovative therapies for diseases such as cystic fibrosis, recently announced that the European Union Committee for Medicinal Products for Human Use (CHMP) has released a positive opinion and recommends Marketing Authorization for the company’s landmark CF combination therapy ORKAMBI (lumacaftor/ivacaftor). ORKAMBI is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene, a condition that affects about 12,000 patients aged 12 years and older in Europe, and over 30,000 people in the United States.
The CHMP has also released a positive opinion and recommends KALYDECO as a medicine to be used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have one of the following mutations in their CF gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. KALYDECO is also used for the treatment of CF in patients aged 2 years and older who have an R117H mutation in their CF gene.
The new opinion for ivacaftor as treatment for CF children aged 2 to 5 years involved a recommendation to lengthen ivacaftor’s weight/dose for CF children aged 6 to 11 years who weigh less than 25 kg with the use of a novel oral granule formulation.
CHMP has also released a positive opinion recommending ivacaftor as a treatment for CF patients aged 18 years and older who suffer from the R117H mutation, a mutation that affects about 125 children in Europe aged 2 to 5 years who have one of the nine CF genetic mutations. In Europe, nearly 250 adults have the R117H genetic mutation.
“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 in a recent news release. “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.”
The positive opinions from CHMP must now be approved by the European Commission (EC). The EC is the authority that makes the final decision regarding the approval of drugs in the European Union.