FDA Breakthrough Therapy Designation: Vertex Pharmaceuticals Gets #31
On January 29th, Vertex Pharmaceuticals announces that the coveted FDA Breakthrough Therapy Designation (BTD) is granted to VX-661 in combination with Kalydeco (Ivacaftor) for the treatment of Cystic Fibrosis (CF) patients, who have two copies of the F508del mutation. The announcement is made as part of Vertex Pharmaceuticals reporting on its full-year 2013 financial results, 4th quarter 2013 financial results, and financial guidance for 2014.
This is the 3rd FDA BTD that Vertex Pharmaceuticals receives for Kalydeco. Kalydeco receives 2 BTDs at the beginning of 2013, as a monotherapy and in combination with Lumacaftor (VX-809). Both VX-809 and VX-661 are small molecule CFTR correctors, while Kalydeco is a small molecule potentiator of CFTR.
Vertex Pharmaceuticals has submitted a protocol to the FDA for a 12-week Phase II Study for the evaluation of the combination of Kalydeco + VX-661 for pharmacokinetics, safety, and efficacy. The Phase II Study is expected to start in the first half of 2014 for patients with two copies of the F508del mutation.
Vertex Pharmaceuticals will report results from two Phase III trials of the combination of Kalydeco + VX-809 in the middle of 2014. These trial results are very important to the future of the Kalydeco franchise, because it is for treatment of patients with the most common form of CF, the F508del homozygous population, which is about 40% of the total CF population.
Three Kalydeco Breakthrough Therapy Designations
|Kalydeco + Lumacaftor (VX-809)||Cystic Fibrosis|
|Kalydeco + VX-661||Cystic Fibrosis|
Vertex Pharmaceuticals’ financial results for Kalydeco as reported on January 29th are as follows:
• 4th Quarter 2012 Kalydeco net product revenue is $ 58.5 million
• 4th Quarter 2013 Kalydeco net product revenue is $109.5 million
• Full-Year 2012 Kalydeco net product revenue is $171.6 million
• Full-Year 2013 Kalydeco net product revenue is $371.3 million.
Thus, Vertex Pharmaceuticals’ net product revenue for Kalydeco, more than doubled from 2012 to 2013, for both the 4th Quarter and the Full-Year. The increase in revenue is due mostly to the “rapid uptake of Kalydeco by eligible patients in Europe in 2013.” Almost all patients eligible to take Kalydeco (G551D mutation) in the United States and Europe have started use of the drug. Kalydeco is also approved for the G551D mutation in Australia and Canada, but reimbursement talks are still continuing. The future success of the Kalydeco franchise is dependent on the expansion of its label. For 2014, Vertex Pharmaceuticals expects Kalydeco total revenue of $470 – $500 million.
Please Note: FDA Official Logo from FDA website.