Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has announced that its Cystic Fibrosis treatment known as ORKAMBI has been approved by the Therapeutic Goods Administration (TGA) of Australia.
ORKAMBI is the first drug used to address the causes of cystic fibrosis especially in patients above the age of 12 with two copies of the F508del mutation. About 1,000 people over 12 years old who suffer from the condition in Australia have two copies of the mutation. The reimbursement process for the drug has already commenced between the Pharmaceutical Benefits Advisory Committee (PBAC) and the TGA.
The pharmaceutical company’s senior VP and general manager Simon Bedson stated that the company was happy that the Australian regulatory body has recognized the clinical benefits to be derived from ORKAMBI. Bedson further added that the approval was a vital step towards making the drug available to Australians who are eligible for the treatment, but they currently lack treatments for the underlying causes of CF.
Vertex carried out 2 phase 3 trials for the drug in 24 weeks, and the research findings from the trials were critical towards the approval of the drug in the country. The TRAFFIC AND TRANSPORT studies in the trials involved more than 1,100 test subjects making it the largest cystic fibrosis study in the history of medicine. The patients who were treated with a cocktail of ivacaftor and lumacaftor had noteworthy improvements in their pulmonary function and their body mass index also improved.
The progress in the patients continued throughout the 48 weeks of the study, and the drug combination was well tolerated throughout the three studies. The most common symptoms of CF include gastrointestinal symptoms, upper respiratory tract infection and shortness of breath among others.
The pharmaceutical company warned that ORKAMBI should be administered with a lot of caution to patients who suffer from advanced liver disease. The firm pointed out that in such a case, the drug should be administered only in cases where the advantages outweigh the risks.