Vertex Pharmaceuticals: FDA Advisory Committee Recommends Kalydeco For R117H CF Mutation
On October 21st, the FDA’s Pulmonary Allergy Drugs Advisory Committee (PADAC) recommends by a vote of 13 to 2, the approval of orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis (CF) patients ages 6 and older who have the R117H mutation in the CF Transmembrane Regulatory (CFTR) gene. In the US, approximately 500 patients ages 6 and older have the R117H mutation.
It is the FDA Advisory Committee that provides the FDA with “independent scientific and medical advice on safety, effectiveness, and appropriate use of potential new medicines.” The FDA often follows the advice of the FDA Advisory Committee. The FDA is expected to make a decision on the approval by December 30, 2014 (PDUFA Date).
Kalydeco receives approval from the FDA in January 2012 for CF patients, ages 6 and older who have at least one copy of the G551D mutation. In February 2014, the FDA approves for people with CF, ages 6 and older, the following 8 mutations in the CFTR gene: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. Thus in the US, Kalydeco is currently approved for 9 mutations.
According to an online Wall Street Journal article, the FDA approval of Kalydeco for the R117H mutation, can result in adding “as much as $160 million in additional revenue by 2017” (the wholesale price of Kalydeco is $311,000/patient).
In Canada, Kalydeco is currently approved for the same US 9 mutations and the additional mutation G970R.
What is being closely watched by patients, investors, and the pharmaceutical industry, is the drug combination of Kalydeco + Lumacaftor, that Vertex Pharmaceuticals is currently testing in patients with the F508del mutation. There are approximately 28,000 people globally that have this mutation.