FDA expansion for Symdeko in cystic fibrosis

pharmatimes | June 24, 2019

The US Food and Drug Administration (FDA) has extended the indication for Vertex Pharmaceuticals Symdeko (tezacaftor/ivacaftor) for the treatment of pediatric patients ages six years and older with cystic fibrosis who have certain genetic mutations. The approval comes after the FDA last year approved Symdeko to treat patients ages 12 and older who had the same specific genetic mutations. The initial FDA approval was based on three phase III, double blind, placebo-controlled trials, which demonstrated improvements in lung function and other key measures of the disease, including a reduction in exacerbations, and the extension data was extrapolated from patients age 12 years and older, with additional support from data in patients age six to 12 years. “Decades ago, patients with cystic fibrosis were generally expected to live until 10 years of age, with few surviving into their teenage years. Since then, wide-ranging research on the disease resulted in more treatments for this debilitating disease that have extended life expectancy and improved quality of life for patients, but there is still no cure,” said Banu Karimi-Shah, acting deputy director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research. “Based on their individual genetic makeup, individuals may respond differently to certain drugs, so it is important to provide a variety of options. Today’s approval of Symdeko for children as young as six years old provides an important treatment option for younger patients, and also provides more context on the safety and dosing specific to this population. The FDA remains committed to advancing novel treatment options for areas of unmet patient need, particularly in diseases impacting children.”

Spotlight

Globally, pharmaceutical regulators are becoming attuned to cold chain issues as biologics, vaccines, and other
temperature‐sensitive products are commercialized for a global clientele. Many regulators now recommend including
temperature monitoring technology in every cold chain shipment.

Spotlight

Globally, pharmaceutical regulators are becoming attuned to cold chain issues as biologics, vaccines, and other
temperature‐sensitive products are commercialized for a global clientele. Many regulators now recommend including
temperature monitoring technology in every cold chain shipment.

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