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Fourth FDA approval for Vertex’s cystic fibrosis medicines
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Fourth FDA approval for Vertex’s cystic fibrosis medicines

Fourth FDA approval for Vertex’s cystic fibrosis medicines

The FDA has approved Trikafta™ (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population. This product is the the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. With this approval, for the first time, approximately 6,000 people with CF ages 12 years and older who have one F508del mutation and one minimal function mutation (F/MF) have a medicine that targets the underlying cause of their CF. Additionally, approximately 12,000 people with one or two F508del mutations who are currently eligible for one of Vertex’s three other FDA-approved CF medicines are now also eligible for Trikafta™.
The efficacy of Trikafta™ was demonstrated in two trials. The first trial was a 24-week, randomized, double-blind, placebo-controlled trial in 403 patients who had an F508del mutation and a mutation on the second allele that results in either no CFTR protein or a CFTR protein that is not responsive to ivacaftor or tezacaftor/ivacaftor alone. The second trial was a four-week, randomized, double-blind, active-controlled trial in 107 patients who had two identical F508del mutations. In each trial, the primary analysis looked at increases in the percent predicted forced expiratory volume in one second, known as ppFEV1, which is an established marker of cystic fibrosis lung disease progression. Trikafta™ increased the ppFEV1 in both trials. In the first trial, it increased mean ppFEV1 13.8% from baseline compared to placebo. In the second trial, it increased mean ppFEV1 10% from baseline compared to tezacaftor/ivacaftor. In the first trial, treatment with Trikafta also resulted in improvements in sweat chloride, number of pulmonary exacerbations (worsening respiratory symptoms and lung function), and body mass index (weight-to-height ratio) compared to placebo.
Trikafta™ is the fourth Vertex’s CF medicine approved by the FDA. The US agency has reviewed and approved the drug in approximately three months, ahead of the March 19, 2020 review goal date. Vertex has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for this combination regimen. The company is now evaluating elexacaftor/tezacaftor/ivacaftor in people ages 6 through 11 with F/MF and F/F CF mutations in an ongoing Phase 3 study and is committed to evaluating elexacaftor/tezacaftor/ivacaftor in children <6 years of age as part of planned future studies.

Latest reimbursement agreements for Orkambi® and Symkevi®

Furthermore Vertex Pharmaceuticals and NHS England have recently concluded an access agreement  for all currently licensed Vertex CF medicines and any future indications of these medicines. This means that within 30 days patients with CF in England ages 2 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can be prescribed Orkambi® (lumacaftor/ivacaftor) by their doctor and CF patients ages 12 years and older who either have two copies of the F508del mutation or one copy of the F508del mutation and a copy of one of the other 14 licensed mutations can be prescribed Symkevi® (tezacaftor/ivacaftor) in combination with ivacaftor. The agreement also offers expanded access to Kalydeco® (ivacaftor) to include people ages 18 years and older who have the R117H mutation and those patients ages 12 months and older who have one of the nine licensed gating mutations.
In addition to England, reimbursement agreements have also recently been announced. The Spanish Government has approved terms for the national reimbursement of Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor) in combination with Kalydeco® (ivacaftor) for eligible patients in Spain living with cystic fibrosis . From the first of November 2019, these drugs can be prescribed for eligible patients living in Spain, namely children ages 6 to 11 years with CF who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene for Orkambi® and patients ages 12 years and older who either have two copies of the F508del mutation, or one copy of the F508del mutation and a copy of one of the other 14 mutations approved within the license in which the CFTR protein shows residual activity, for Symkevi® in combination with Kalydeco®. In Australia, both medicines will also be listed on the Pharmaceutical Benefits Scheme (PBS) from December 1st.

(October 24th, 2019)


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