Twice-daily KALYDECO improves lung function in people with cystic fibrosis

Vertex Pharmaceuticals (Australia) Pty Ltd., announced the presentation of data from KALYDECO™ (ivacaftor) studies at the 10th Australasian Cystic Fibrosis Conference in Auckland, New Zealand, August 17 – 20, 2013, which further demonstrate the benefits of the medicine in people with cystic fibrosis (CF) ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The data show that treating the underlying cause of cystic fibrosis in these patients with twice-daily KALYDECO led to significant and sustained improvements in lung function and other measures of disease.

Cystic fibrosis is a rare genetic disease caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. KALYDECO is the first medicine to treat the underlying cause of CF in people with the G551D mutation. In people with this mutation, KALYDECO helps the defective CFTR protein function more normally.

Significant and sustained benefits in children, adolescents and adults receiving twice-daily KALYDECO (ACFC Oral Abstract 05, Monday, 20 August 2013)

In the previously-reported individual global pivotal studies, STRIVE and ENVISION, children, adolescents and adults with CF ages 6 and older who have at least one copy of the G551D mutation and received 150mg of KALYDECO twice daily experienced significant improvements in lung function and other measures of disease. A new analysis was presented that assessed the efficacy and safety of KALYDECO when responses were pooled across the two Phase 3 studies (STRIVE and ENVISION).

Lung function improvements (measured as forced expiratory volume in one second, FEV₁) were seen at week two and were sustained throughout the 48-week studies. Pooled results from the two studies showed a mean absolute improvement in lung function of 11.2 percentage points through week 24 among those treated with KALYDECO compared to placebo. Through week 48, the mean absolute improvement in lung function for those treated with KALYDECO was 10.9 percentage points compared to placebo.

In the pooled group of patients with the G551D mutation, the safety profile through 48 weeks of KALYDECO treatment was consistent with what was observed independently in the STRIVE and ENVISION studies.

KALYDECO efficacy and safety sustained for up to 96 weeks (ACFC Poster Abstract P9)

PERSIST, an ongoing extension study, enrolled people with cystic fibrosis ages 6 and older who have at least one copy of the G551D mutation and who completed treatment in the Phase 3 STRIVE or ENVISION studies (KALYDECO and placebo treatment groups) and met certain other eligibility criteria. Data from this long-term follow-up study showed that the improvements in lung function, respiratory symptoms and weight gain among those who were treated with KALYDECO for 48 weeks in one of these two pivotal studies were durable for up to 96 total weeks of treatment.

In addition, the analysis showed that people who switched to KALYDECO after receiving 48 weeks of treatment with placebo in one of the Phase 3 studies experienced improvements in lung function, respiratory symptoms and weight gain comparable to those seen in people who received KALYDECO from the beginning of the Phase 3 studies.

KALYDECO improved lung function in people with early-stage and severe CF (ACFC Poster Abstract P8)

CF-related lung disease is known to start before it’s detectable by deterioration in FEV₁. Even when FEV₁ is within the normal range, structural damage may have already occurred in the lungs; much of this can be irreversible.

A secondary analysis of data from the STRIVE, ENVISION and PERSIST studies was conducted to better understand the effect of KALYDECO in people whose lung function was within what is considered the normal range (FEV₁ >90% predicted) as well as in people with severe lung dysfunction (FEV₁ <40% predicted). Most of those with baseline FEV₁ of less than 40 percent predicted who received KALYDECO demonstrated a consistent improvement in lung function and other measures of disease. Patients treated with KALYDECO who had baseline FEV₁ greater than 90 percent predicted also generally maintained their lung function over 48 weeks.