Okrambi for Cystic Fibrosis

Orkambi is a combination therapy (ivacaftor/Kalydeco and lumacaftor) approved by the U.S. Food and Drug Administration (FDA) for treatment of cystic fibrosis (CF). The oral drug helps 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 just the severity of symptoms.
Developed by the Massachusetts-based pharmaceutical company Vertex, Orkambi is approved for use in cystic fibrosis patients age 12 and older with two copies of the F508del mutation in their CFTR gene. The mutation is the leading cause of CF. FDA approval for the therapy was announced on July 2, 2015. CF affects around 30,000 people in the U.S. — well below the Food and Drug Administration's 200,000-patient-or-less benchmark that determines whether or not a disease is considered rare. Orkambi is a mixture of Vertex's other marketed product, Kalydeco, and lumacaftor, a drug that helps correct the misfolded proteins that are characteristic of CF.

The wholesale acquisition cost for the cystic fibrosis (CF) treatment now clocks in at $20,919 for four, 28-tablet boxes, according to an investor note from Cowen & Co. analyst Phil Nadeau. Patients ages 12 and up are supposed to take Orkambi twice daily, giving it an annual bill of $251,000 before rebates.