Cost regulators for the NHS in England and Wales have called on Norgine to supply more evidence on its liver failure/brain disease drug Xifaxan (rifaximin; also celled Targaxan 550).
The National Institute for Health and Care Excellence (NICE) is currently appraising Xifaxan as a treatment for the serious and potentially life-threatening condition hepatic encephalopathy (HE), which affects about 10,000 patients in the UK.
HE can occur in patients with liver disease from the build up of serum toxic substances produced by gut bacteria that are not properly removed by the damaged organ. These toxins can cause a range of neuropsychiatric disorders, from mild intellectual impairment to personality changes, depressed level of consciousness and even coma.
Xifaxan, which was developed by Alfa Wasserman, was launched in the UK in January this year. Marketing clearance was based on data from a pivotal trial showing a 58% relative risk of breakthrough episodes of overt HE and a 50% relative reduction in the risk of hospitalisations in patients given the drug.
However, when reviewing the evidence, the Appraisal Committee concluded that "there were still questions to be answered about the drug", which is why it has requested more details from Norgine. "We want to ensure that we have sufficient information to make an informed final recommendation," the Institute said.
Norgine will respond
Norgine said it welcomes the Committee’s request for additional analyses "and a further opportunity to demonstrate the value which this innovative treatment can offer patients and their carers living with hepatic encephalopathy in England and Wales".
The drugmaker said it will respond by the end of the consultation period on Wednesday 18 December, and expects a final decision from NICE sometime in 2014.
Peter Martin, Chief Operating Officer at Norgine, said his firm is confident in the clinical efficacy of the drug and its value to the NHS.
"This treatment offers a significant advance as it can alleviate the substantial burden on patients, their families and healthcare systems and improve quality-of-life," he said, noting "tt is particularly critical as there is currently no cure for hepatic encephalopathy apart from liver transplantation".
The drug has been accepted for use by the Scottish Medicines Consortium.