Vertex Pharmaceuticals Drug To Treat Cystic Fibrosis Approved By FDA

The Food and Drug Administration have cleared a new drug named Orkambi, which is used for treating the most common from of cystic fibrosis. Vertex Pharmaceuticals Inc has developed this combination drug.

Vertex, after successful clinical trials, has developed Orkambi as a ‘newer version’ of Kalydeco. Kalydeco came in the market in 2012 and was the first drug to treat cystic fibrosis; scientists combined the components of Kalydeco with a new element called lumacaftor to produce Orkambi.

Dr. Robert Giusti of New York University who directs the center’s cystic fibrosis program, calls it an amazing innovation because this medicine will potentially make it possible to reverse the damage that the disease causes i.e. about 1 to 2% deterioration each year in the lungs of the patient.

Cystic fibrosis is a genetic condition in which the lungs and digestive system become clogged with thick sticky mucus, the symptoms are evident from childhood and include persistent cough, recurring chest and lung infections and poor weight gain. Screening Tests are usually carried out at an early age, even before symptoms appear, to determine whether the child is suffering from the disease.

FDA has approved the drug for more than 8,500 people aged 12 and above in the U.S.

However, Giusti is hopeful that FDA will eventually expand the reach of the drug and give its approval for patients as young as 6 –year olds.

With the FDA approval, the availability of targeted treatments for the specific defects, that cause cystic fibrosis, increases greatly, stated Dr. John Jenkins, director of the agency’s Office of New Drugs, Center for Drug Evaluation and Research.

Unfortunately the new Vertex drug comes with a hefty price tag of $300,000 a year per patient. 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 former drug Kalydeco had shown a 10% improvement.


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