The Therapeutic Goods Administration (TGA) of Australia has approved Vertex Pharmaceuticals, Kalydeco (ivacaftor) for 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. Kalydeco is the first medicine to treat the underlying cause of the disease in these patients.

Cystic fibrosis is a rare genetic disease caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. In people with the G551D mutation, Kalydeco helps the defective CFTR protein function more normally. An estimated 250 people, or 8 percent of those with CF in Australia, have the G551D mutation. Kalydeco is the first Vertex medicine approved in Australia.

"The approval of Kalydeco in Australia is an important milestone for the cystic fibrosis community," said Simon Bedson, senior vice president of International Commercial Operations for Vertex. "The rapid review and approval by the TGA is a major step in making Kalydeco available for eligible Australians."

The approval of Kalydeco was based on data from two global phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with Kalydeco experienced significant and sustained improvements in lung function and weight gain compared to those who received placebo. In one study, adults and adolescents who took Kalydeco were also significantly less likely to experience pulmonary exacerbations, which are periods of worsening respiratory signs and symptoms that require increased treatment with antibiotics and hospital visits.

The most common serious adverse events reported in phase 3 studies included abdominal pain, increased liver enzymes and low blood sugar, which occurred in less than 1 percent of patients. Adverse events commonly observed in those taking Kalydeco included headache, upper respiratory tract infection (common cold), stomach pain and diarrhoea. Fewer people in the Kalydeco treatment groups discontinued treatment due to adverse events than in the placebo group. The majority of adverse events associated with Kalydeco were mild to moderate.

Kalydeco was discovered by Vertex as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation in the US.

Australian approval and reimbursement of a new medicine is a multi-step process. Once a new medicine receives approval from the TGA, it is assessed for effectiveness and cost-effectiveness by the Pharmaceutical Benefits Advisory Committee (PBAC) for listing on the Pharmaceutical Benefits Scheme (PBS). Additional information regarding the reimbursement of Kalydeco in Australia is expected to be available later in 2013 following the publicly announced planned review of Kalydeco at the upcoming PBAC meeting, July 10 to 12, 2013, and subsequent discussions.

Kalydeco (ivacaftor) is the first medicine to treat the underlying cause of CF in people with the G551D mutation in the CFTR gene. Known as a CFTR potentiator, Kalydeco is an oral medicine that aims to help the CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways. Kalydeco (150mg, q12h) was approved by the US Food and Drug Administration, the European Medicines Agency and Health Canada in 2012 for use in people with CF ages 6 and older who have at least one copy of the G551D mutation in the CFTR gene.

Vertex retains worldwide rights to develop and commercialise Kalydeco.

Vertex is a global biotechnology company that aims to discover, develop and commercialise innovative new medicines so people with serious diseases can lead better lives.