Kalydeco (ivacaftor) is a drug treatment developed and commercialized by the company Vertex for patients who suffer from cystic fibrosis (CF). The medication was approved in the United States by the U.S. Food and Drug Administration (FDA) to be used in patients older than two years and it is also available in Europe, Canada, Australia and New Zealand.
Kalydeco is a potentiator of the CF transmembrane conductance regulatory (CFTR) and was the first drug to address the underlying causes of the disease, instead of only treating its symptoms. The oral medication was first introduced in the country in January, 2012 to treat CF adult patients, and providing a new hope due to its unique mechanism of action. In March, 2015, new research led the FDA to approve the use of the drug also in children with determined mutations of the CF gene.
History of Kalydeco
The efficacy of Kalydeco was evaluated by the Massachusetts-based pharmaceutical company Vertex in two randomized, double-blind, placebo-controlled clinical trials, which were used to justify the FDA’s approval. The studies included 213 individuals who were treated with Kalydeco 150 mg twice daily or placebo every 12 hours, together with their prescribed CF therapies. The two studies revealed Kalydeco’s safety and superiority in improving FEV1, alterations that were persistent through 48 weeks of follow-up.
The company had also conducted a double-blind, placebo-controlled, cross-over trial with 39 adult patients to assess dose ranging, which was also used to submit the drug to the FDA for approval. The agency approved Kalydeco in 2012 to treat patients older than six years based on the data presented by the company, and most recently, the FDA approved a granulated version of the medication for children between two and five years old based on the company’s latest studies.
The unique mechanism of action of Kalydeco revolutionized the market of CF treatment, as it became the first treatment to address the underlying causes of the disease, a genetic defect that causes alterations in the protein responsible for the salt in cells regulation. Due to this mutation, patients develop a thick and sticky mucus in the respiratory, digestive and reproductive systems. However, Kalydeco maintains the CFTR proteins at the cell surface open to facilitate the transport of salt and water through the cells, improving hydration and mucus clearance.
Other Details About Kalydeco
In the U.S. and Europe, Kalydeco is available for patients who suffer from G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R mutations in the CFTR gene. In Canada, in addition to the previous ones, the treatment is also available for patients older than six with the G970R mutation, while in both the U.S. and Canada, patients with the R117H mutation can also be treated with the medication. However, Canadian patients with the last mutation need to be older than 18 years.
In Europe and Canada, where the minimum age to receive Kalydeco is six, as well as in the United States, Kalydeco is commercialized in 150 mg tablets, while in the U.S., there are also 50 mg and 75 mg oral granules for patients age 2 to less than 6 years. The treatment with Kalydeco is not effective in patients with CF and two copies of the F508del mutation (F508del/F508del) in the CFTR gene, and the safety and efficacy of it in babies younger than two years old was never studied, which makes its use not recommended.
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