At a Glance
Therapeutic Area:
Cystic Fibrosis
Approved Applications:
Orkambi is an FDA-approved combination therapy for the treatment of cystic fibrosis (CF).
Orkambi™ is a combination of two categories of molecules, FDA-approved ivacaftor (known by its brand-name Kalydeco) and experimental therapy lumacaftor (known also as VX-809), both of which help in correcting the mutated genes in patients with cystic fibrosis – a novel therapeutic approach designed to treat the root cause of cystic fibrosis instead of treating the severity of symptoms. Both ivacaftor and lumacaftor work by correcting the misfolded CFTR protein, which is the root cause of the F508del mutation. Orkambi has been approved for people ages 12 and older who have two copies of the F508del mutation -- the most common CF mutation. The F508del mutation causes a series of problems that prevent CFTR from achieving the correct shape and reaching the cell surface. The combination drug treats these problems with a two-step approach: Lumacaftor helps move the defective CFTR protein to its proper place at the cell surface. Ivacaftor increases the activity of the protein once it is there, supporting the flow of salt and fluids, which helps thin the thick mucus that builds up in the lungs and other organs.
Orkambi™ is a combination of two categories of molecules, FDA-approved ivacaftor (known by its brand-name Kalydeco) and experimental therapy lumacaftor (known also as VX-809), both of which help in correcting the mutated genes in patients with cystic fibrosis – a novel therapeutic approach designed to treat the root cause of cystic fibrosis instead of treating the severity of symptoms. Both ivacaftor and lumacaftor work by correcting the misfolded CFTR protein, which is the root cause of the F508del mutation. Orkambi has been approved for people ages 12 and older who have two copies of the F508del mutation -- the most common CF mutation. The F508del mutation causes a series of problems that prevent CFTR from achieving the correct shape and reaching the cell surface. The combination drug treats these problems with a two-step approach: Lumacaftor helps move the defective CFTR protein to its proper place at the cell surface. Ivacaftor increases the activity of the protein once it is there, supporting the flow of salt and fluids, which helps thin the thick mucus that builds up in the lungs and other organs.
Applications In Development:
1) Kalydeco + tezacaftor and 2) Kalydeco + tezacaftor + 2nd. generation corrector (VX-440, VX-152, VX-659 or VX-445)
Acquisition Date:
November 2014
