RARE Daily

FDA Expands Vertex’ Kalydeco Approval to Eligible Infants

September 25, 2020

Rare Daily Staff

The U.S. Food and Drug Administration has expanded the approval of Vertex Pharmaceuticals’ Kalydeco to include use in children with cystic fibrosis ages four months to less than six months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Kalydeco based on clinical and/or in vitro assay data.

Kalydeco is already approved in the United States and the European Union for the treatment of cystic fibrosis in patients ages six months and older.

“Since the initial approval of Kalydeco more than eight years ago, we have continued to advance our clinical development program with the goal of treating the underlying cause of cystic fibrosis as early in life as possible,” said Reshma Kewalramani, CEO and president of Vertex. “Today’s approval is a testament to our relentless efforts, alongside the clinical and scientific community, to reach all people with CF who may benefit from our medicines.”

Cystic fibrosis is a rare, progressive, and life-shortening genetic disease affecting approximately 75,000 people worldwide. It is a multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. While there are many different types of CFTR mutations that can cause the disease, most people with CF have at least one F508del mutation.

These mutations lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in several organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

This FDA approval is based on data from a cohort in the 24-week phase 3 open-label safety cohort consisting of 6 children with CF ages four months to less than six months who have one of 10 mutations in the CFTR gene. This cohort demonstrated a safety profile similar to that observed in older children and adults.

“Initiating therapy that treats the underlying cause of cystic fibrosis as early as four months of age may have the potential to modify the course of the disease,” said Margaret Rosenfeld, a physician at Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine.

Photo: Reshma Kewalramani, CEO and president of Vertex


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