Massachusetts-based Vertex Pharmaceuticals, Inc. announced yesterday that they have filed for a New Drug Application (NDA) with the US Food and Drug Administration, and a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for their pipeline combination lumacaftor–ivacaftor drug, indicated for the treatment of cystic fibrosis in patients carrying the F508del CFTR gene alteration.
Cystic fibrosis (CF) affects about 75,000 people combined in the US, Europe, and Australia. While the median age of survival has greatly increased, there is still no known cure for this rare hereditary condition, and the average age of death among CF patients is still in the mid-20s.
“The combination of lumacaftor and ivacaftor is the first potential treatment designed to target the underlying cause of cystic fibrosis in people with two copies of the F508del mutation, which is the most common form of the disease,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer, Vertex. “Today’s submissions represent important progress toward our ongoing efforts to develop new medicines for the vast majority of people with cystic fibrosis, and we look forward to working closely with regulatory agencies to bring this treatment to eligible patients as quickly as possible.”
These recent submissions follow promising progress in both American and European territories when the proprietary combination was granted Breakthrough Therapy Designation by the FDA in 2012, and granted Accelerated Assessment of the MAA by the European Committee for Medicinal Products for Human Use (CHMP). If Vertex’s requests are granted, it would receive Priority Review, which reduces the FDA’s review time to as soon as 8 months; and in Europe would shorten review time from 210 to 150 days. As a final hurdle towards approval in Europe, the European Commission will be making their decision based on the CHMP’s review. This step takes about 3 months, and if approved, will initiate the company’s country-by-country reimbursement approval process. Both submissions are for a “fully co-formulated combination treatment dosed as two tablets every 12 hours (four tablets daily).”
To support the two submissions, Vertex included promising findings from a pair of recent international Phase 3 clinical studies: TRAFFIC and TRANSPORT, along with preliminary data from a subsequent rollover trial in F508del gene carriers ages 12 years and older who have received standard CF medication. The two former Phase 3 studies exhibited improved lung function and reduced exacerbations after 6 months of treatment with the combination drug, while interim data from the rollover study showed the improvements were maintained for another 6 months. As for safety, the drug was observed to be well-tolerated, and caused minor adverse effects, such as infective pulmonary exacerbation, headache, cough, and increased sputum production.
Vertex is aware of the seriousness of CF, and is planning to conduct a Phase 3b study with a limited sample population of people with severe lung affectations within Q1 2015, with a subsequent expanded access program in Q2. The company is also working on making the drug available in countries outside of the US and Europe, and hopefully expand research programs by Q2 2015. To know more about Vertex and the lumacaftor-ivacaftor drug, dial U.S.: 1-877-634-8789 or e-mail [email protected].