- An FDA panel's route to reaching a positive decision regarding Vertex's Orkambi(lumacaftor/ivacaftor) was not exactly smooth sailing.
- Orkambi combines Kalydeco (ivacaftor), which is is intended to treat several cystic fibrosis (CF) transmembrane conductase inhibitor (CF-TR) mutations, with ivacaftor, which treats the F508del genetic mutation.
- After a period in which panel members discussed concerns about efficacy, the FDA's Pulmonary-Allergy Advisory Committee voted 12 to 1 to support approval of Orkambi. It also voted 13 to 1 that the product is safe.
Earlier this week, BioPharma Dive reported on FDA concerns that Orkambi may not be much more effective than Kalydeco alone. Although the panel endorsed Orkambi for approval, at one point only three members of the panel were endorsing Orkambi for efficacy, while four members said that the data did not show efficacy, and six others said that efficacy could not be determined with the available data.
There are roughly 30,000 people in the U.S., of which roughly 8,500 could benefit from treatment with Orkambi. While pricing is still unknown, Kalydeco's $300,000-plus price tag alone is daunting. Usually when an FDA panel recommends approval, the drug in question is approved. However, payers will undoubtedly push back as the price of Vertex's CF treatment options increase alongside its potential treatment population.