Kalydeco to be landmark in cystic fibrosis treatment
pharmafile | February 1, 2012 | News story | | Vertex, cystic fibrosis, telaprevir
Vertex’s first-in-class treatment for cystic fibrosis Kalydeco has been approved by the FDA.
The approval is a landmark in the treatment of the disease, as the drug is the first which can alter the course of the progressive and ultimately fatal genetic disorder.
Kalydeco (ivacaftor) is only licenced to treat a small sub-set of CF patients, however – those who have the G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.
Just 4% of CF patients are believed to have this mutation, but the approval has nevertheless been hailed as a breakthrough in treating the disease, and also a milestone in drug approval.
Kalydeco is a pill taken two times a day with fat-containing food, and helps the protein made by the CFTR gene function better and as a result, improves lung function and other aspects of CF such as increasing weight gain.
“Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis,” said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research. “This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease.”
The medicine has been granted approval after just three months of appraisal, making it one of the fastest FDA approvals ever.
“Kalydeco is an excellent example of the promise of personalised medicine – targeted drugs that treat patients with a specific genetic makeup,” said FDA Commissioner Margaret Hamburg “The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development.”
Jeffrey Leiden, Vertex’s incoming president and chief executive said Kalydeco was the result of more than 13 years of research into how to target the underlying causes of the disease. The drug was discovered in collaboration with Cystic Fibrosis Foundation Therapeutics, the non-profit drug discovery and development arm of the Cystic Fibrosis Foundation.
“Kalydeco represents a major advance in the treatment of cystic fibrosis for people with a specific type of this disease. But our work isn’t done. With the ongoing support of doctors, patients and the Cystic Fibrosis Foundation, we’re making progress toward our ultimate goal of developing additional medicines to help many more people with cystic fibrosis.”
Two 48-week, placebo-controlled clinical studies involving 213 patients, one in patients ages 12 years and older and another in patients ages 6-11 years, were used to evaluate the safety and efficacy of Kalydeco in CF patients with the G551D mutation. In both studies, treatment with Kalydeco resulted in significant and sustained improvement in lung function.
Kalydeco is effective only in patients with CF who have the G551D mutation. It is not effective in CF patients with two copies of the F508 mutation in the CFTR gene, which is the most common mutation that results in CF. If a patient’s mutation status is not known, an FDA-cleared CF mutation test should be used to determine whether the G551D mutation is present.
A high cost treatment
Unsurprisingly, the orphan drug status of Kalydeco means its price is very high – its annual cost in the US will be $294,000, making it one of the most expensive drugs in the world.
Analysts expect Kalydeco sales to exceed $1 billion worldwide with its current indication, or even $3-4 billion if the drug proves effective for the larger cystic fibrosis population.
The company anticipates a decision in Europe in the third quarter of 2012, while its price is also expected to significantly lower in European markets.
The approval will help transform the fortunes of Vertex, which is already riding high on the rapid uptake of its first approved drug, hepatitis C treatment Incivek/Incivo (telaprevir). Sales of the hepatitis C treatment are rocketing, and is expected to break through the $1billion sales mark this year, which would make it one of the fastest growing drugs of all time.
Andrew McConaghie
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