European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of Kaftrio in combination with Kalydeco for the treatment of CF children 2-5 years old, who have at least one F508del mutation in the CFTR gene.
The European Commission is expected to reach a final decision on the approval of the medicine within two months.
“Kaftrio has demonstrated unprecedented clinical benefit for eligible people living with CF,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex. “Treating the underlying cause of CF as early as possible in life has the potential to slow disease progression, which is why we are pleased the CHMP is supportive of expanding the indication for KAFTRIO to patients as young as 2 years.”
You can find the detailed press release of Vertex Pharmaceuticals here.