CHMP Grants Positive Opinion to Vertex for KALYDECO® in Treating Cystic Fibrosis in Infants Aged 1 Month and Above

Vertex Pharmaceuticals (Nasdaq: VRTX) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a favorable opinion regarding the expansion of the label for KALYDECO® (ivacaftor) to include the treatment of infants with cystic fibrosis (CF) aged 1 month to less than 4 months. These infants must have specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, including R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.

Fosca De Iorio, Vice President of International Medical Affairs at Vertex, emphasized the significance of early intervention in cystic fibrosis: “Cystic fibrosis is a chronic, progressive disease that is present from birth, which is why it is important to treat the underlying cause of this disease as early as possible to potentially slow disease progression.” If approved, the label expansion for KALYDECO would enable eligible infants with CF to receive targeted treatment from the outset of their lives.

KALYDECO® is already approved in the European Union for the treatment of individuals with CF aged 4 months and older with specific mutations in the CFTR gene.

Cystic fibrosis is a rare genetic disease affecting over 92,000 individuals worldwide. It is characterized by progressive multi-organ complications, impacting various bodily systems including the lungs, liver, pancreas, GI tract, sinuses, sweat glands, and reproductive tract. CF is caused by mutations in the CFTR gene, which result in defective or absent CFTR protein, leading to impaired salt and water flow across cell membranes. This dysfunction causes the buildup of thick mucus in the lungs, leading to chronic infections, lung damage, and decreased life expectancy.

Diagnosis of CF typically involves genetic testing and confirmation through sweat chloride testing. Treatment aims to restore CFTR function, thereby reducing symptoms and improving outcomes.

KALYDECO® (ivacaftor) is an oral medication designed to enhance the function of CFTR proteins at the cell surface, facilitating the transport of salt and water across cell membranes. It is indicated for the treatment of CF in patients aged at least 4 months with specific CFTR gene mutations.

Vertex Pharmaceuticals is a global biotechnology company focused on developing transformative medicines for serious diseases. Its portfolio includes approved therapies for cystic fibrosis, sickle cell disease, and transfusion-dependent beta thalassemia, with ongoing research programs in various other disorders.

Vertex cautions that forward-looking statements in this press release are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Factors include the potential for regulatory approval delays and data from ongoing development programs. Vertex disclaims any obligation to update forward-looking statements as new information becomes available.

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