Vertex Pharma Received FDA approval For Its Cystic Fibrosis Drug

July 2, 2015 U.S. Food and Drug Administration approved Orkambi which is a combination of Lumacaftor 200 mg/Ivacaftor 125 mg and it is the first drug for cystic fibrosis directed at treating the cause of the disease in people who have two copies of a specific mutation.

On November 05, 2014 Vertex Pharma submitted (NDA) 206038 under section 505(b) of the Federal Food, Drug, and Cosmetic Act (FDCA) for Orkambi (Lumacaftor/Ivacaftor) oral tablet at 200mg/125mg for the treatment of cystic fibrosis (CF) in patients age 12 years and older who are homozygous for the F508del mutation in the CFTR gene.

Cystic Fibrosis :
Cystic fibrosis (CF) is a genetic disorder that affects mostly the lungs but also the pancreas, liver, kidneys and intestine. Long-term issues include difficulty breathing and coughing up sputum as a result of frequent lung infections. Other symptoms include sinus infections, poor growth, fatty stool, clubbing of the finger and toes, and infertility in males among others. Different people may have different degrees of symptoms.

Lumacaftor :
Lumacaftor (VX-809) is developed by Vertex Pharmaceuticals, chemically designated as 3-{6-{[1-(2,2-difluoro-1,3-benzodioxol-5-yl)cyclopropanecarbonyl]amino}-3-methylpyridin-2-yl}benzoic acid. Lumacaftor is a white to off-white powder that is practically insoluble in water (0.02 mg/mL). The structure of Lumacaftor is given below:


Ivacaftor :
Ivacaftor (VX-770) was also developed by Vertex Pharmaceuticals. On Jan 31, 2012 Vertex received FDA approval for Ivacaftor which is marketed under the brand name Kalydeco. Ivacaftor is protected in US through US 7,495,103 B2 which is an OB listed patent for Ivacaftor and it is set to expire on May 20, 2027 (including PTA of 695 days). The structure of Ivacaftor is given below:


“We expect 2015 to be a year of rapid and significant growth in revenues based on treating many more people with CF following the potential approval and launch of the combination of Lumacaftor and Ivacaftor and further label and geographic expansion for KALYDECO,” said Ian Smith, Executive Vice President and Chief Financial Officer for Vertex.


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