- FDA staff is questioning whether Vertex's new Kalydeco (ivacaftar)/lumacaftor combination, known as Orkambi, is worth its expected exorbitant price.
- Kalydeco is approved in the U.S. for 10 cystic fibrosis (CF) transmembrane conductase inhibitor (CF-TR) mutations.
- Orkambi is intended to treat CF patients 12 years and older with another genetic mutation, F508del.
At the heart of the controversy surrounding Kalydeco, and now Orkambi, is the price tag. Kalydeco alone is $307,000.
While it's not clear how Orkambi will be priced, it will undoubtedly be more expensive. While Kalydeco is designed to address genetic deficiencies in roughly 2,000 of the 30,000 people with CF in the U.S., Orkambi is designed to address genetic deficiencies in roughly 8,500 people with CF, according to Reuters.
But the FDA review committee, which will meet on Tuesday to debate whether or not to recommend the new combo's approval, is wondering just how great of an effect Orkambi will have versus Kalydeco on its own since Vertex's trial design tested the med against placebo. Most analysts still expect an approval by July 5. But whether or not payers will cover the new drug is another question.