Vertex Pharmaceuticals (NASDAQ:VRTX) won US approval for its combination treatment for the most common form of cystic fibrosis, giving the drugmaker what analysts have projected will be a new blockbuster. By combining Kalydeco with lumacaftor, Boston-based Vertex is seeking to treat an additional 8,500 patients.
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The doctor added that he expects the FDA will expand its approval for Orkambi to patients with CF who are six years or older to increase the amount of people who would benefit from the new treatment. Further label expansions and the release of ‘661 for F508del heterozygous patients provide mid-term optionally, and we remain bullish on the corrector hypothesis that should lead to a more efficacious triple-combo for a wider group of cystic fibrosis (CF) patients…
Read the original article on Reuters. Jeffrey Leiden (Vertex’s chairman, president and CEO.) “The approval of ORKAMBI represents a fundamental change in the treatment of the most common form of CF, marking significant progress for us and for the entire CF community”, Vertex’ CEO said. Also taken into account was the result of the clinical trails in which Orkambi reported a 2.5 to 3% improvement in lung function while the ex- drug Kalydeco had shown a 10% improvement.
The efficacy of Orkambi was demonstrated in two phase III studies in which lung function improved by about three percentage points over placebo. About 70,000 people worldwide suffer from the disease, though only about half of those are thought to have the mutation treated by Orkambi.
The medication, Orkambi, is actually two drugs but it is pricey.
Orkambi and Kalydeco aim at fixing the underlying condition caused by the misbehaving gene, unlike current drugs which treat related symptoms.
Cystic fibrosis is a life-threatening genetic disease that causes serious lung infections and premature death. Kalydeco was developed for patients who have a problem with a protein on their cell walls, which doesn’t properly balance the flow of water and salt from the cell. “More than 15 years ago, our scientists set out to discover and develop medicines to treat the underlying cause of cystic fibrosis”. Patients taking the new Vertex’ pill were experiencing shortness of breath, upper respiratory tract infection, nausea, diarrhea, and rash.
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Ivacaftor/lumacaftor is the first drug to target the root cause of the disease in such people, about 45% of all CF patients, the FDA said. Almost every CF drug available today was made possible because of CF Foundation support. A decision by the European Medicines Agency (EMA) is anticipated by the end of 2015.
