Vertex Pharmaceuticals reported that the U.S. Food and Drug Administration (FDA) has granted approval for its cystic fibrosis (CF) drug Orkambi to be used to treat children, ages 6 to 11, who have two copies (one inherited from each parent) of the F508del mutation in the CFTR gene.
Having two copies of the F508del mutation is the leading cause of CF. According to the Cystic Fibrosis Foundation and UK CF Registry, approximately half of all people with cystic fibrosis in both the U.S. and the U.K. have two copies of the F508del mutation, identified by a simple genetic test.
Orkambi was previously approved by the FDA for the treatment of people ages 12 and older with two copies of the F508del mutation. This extended approval opens the treatment to some 11,000 more CF patients in the U.S.
If you’ve missed the news updates, the application for Orkambi’s inclusion on our Pharmaceutical Benefits Scheme has already been rejected and the next meeting where they will consider it is November of this year. (Closing date for any further submissions is 5th October).
If you’d like to pledge your support for Orkambi before the next PBS meeting in November, you must do so by 5th October here
More detail from Vertex’s previous investor announcement:
Phase 3 safety study of lumacaftor/ivacaftor in children ages 6 to 11
Final data were presented from an open-label Phase 3 safety study that evaluated lumacaftor/ivacaftor in 58 children with CF ages 6 through 11 who have two copies of the F508del mutation. In the study, all children received a twice-daily fixed-dose combination of lumacaftor (200mg) and ivacaftor (250mg) for 24 weeks. As announced in January 2016, the study met its primary safety endpoint.
Safety data showed that the combination was well tolerated, and the most common adverse events were cough, headache, infective pulmonary exacerbation, nasal congestion, abdominal pain, increased sputum and elevated liver enzymes. Two patients (3.4%) discontinued treatment because of adverse events (rash and abnormal liver function tests). Respiratory events, including dyspnea, respiration abnormal and wheezing, were observed in four patients (6.9%) and were not associated with treatment discontinuation. Serious adverse events were reported in four patients (6.9%), with one patient (1.7%) having elevated abnormal liver function tests.
Additional details from the study are being presented today at ECFS, including results for the study’s secondary and exploratory efficacy endpoints, as part of Workshop 19, Late Breaking Science.
At 24 weeks, there were improvements in multiple secondary endpoints, including a reduction in sweat chloride of -24.8 mmol/L (p < 0.0001), a weight gain of 2.6 kg (p < 0.0001), an improvement in the CFQ-R respiratory domain score of 5.4 points (p=0.0085), an absolute improvement in FEV1 of 2.5 percentage points (p=0.067), and a -0.88 (p=0.0018) improvement in the exploratory endpoint of lung clearance index (LCI2.5).
The U.S. FDA recently granted Vertex’s request for Priority Review of a supplemental New Drug Application (sNDA) for approval of ORKAMBI for children ages 6 through 11 who have two copies of the F508del mutation and set a target review date of September 30, 2016 for a decision on the sNDA. There are approximately 2,400 children ages 6 to 11 who have two copies of the F508del mutation in the U.S.
Enrollment is complete in a six-month Phase 3 efficacy study required to support potential approval of lumacaftor/ivacaftor in the European Union in children ages 6 through 11 who have two copies of the F508del mutation. The study is evaluating lumacaftor/ivacaftor in approximately 200 children and the primary endpoint is the absolute change in lung clearance index. Pending data from the study, Vertex plans to submit a Marketing Authorization Application (MAA) variation in the European Union in the first half of 2017. In Europe, there are approximately 3,400 children ages 6 through 11 who have two copies of the F508del mutation.