The European subsidiary of Japanese pharma company Shionogi has announced the launch of its thrombocytopenia drug Mulpleo in the UK, offering a new treatment option for patients with severe disease.
Shionogi’s Mulpleo (lusutrombopag) is the first licensed treatment available on the NHS in the UK for severe thrombocytopenia – or severely low levels of blood platelets – in adults with chronic liver disease who are undergoing invasive procedures.
The UK’s medicines watchdog NICE and the Scottish Medicines Consortium recently backed the use of Mulpleo in this patient population after determining that the drug was cost-effective. The positive recommendations of the drug were based on the data from two phase 3 clinical trials – L-PLUS 1 and L-PLUS 2.
In the L-PLUS 1 study, 79.2% of patients receiving the Mulpleo treatment required no platelet transfusion prior to the primary invasive procedure, compared to 12.5% of those on the placebo arm. Meanwhile, in the L-PLUS 2 study 64.8% on the Mulpleo arm required no platelet transfusions compared to 12.5% of those receiving placebo.
Across both trials, platelet counts remained above 50,000/µL for a median of 20.9 days in patients receiving Mulpleo treatment, compared with 9.5 days in patients treated with placebo.
Chronic liver disease affects a large number of the population in the UK, with over 600,000 people having a form of serious liver disease and 60,000 having cirrhosis – a condition in which the liver does not function properly due to long-term damage and is characterised by the replacement of normal liver tissue by scar tissue.
“It is great news that Mulpleo is now available on the NHS following approvals from both the SMC and NICE,” said Andrew Holt, consultant physician (GI, HPB & Liver), University Hospitals, Birmingham.
“Until now, there have been no specific therapies available to treat thrombocytopenia, a common blood-related complication of chronic liver disease which can complicate or delay crucial interventions for these patients,” he added.
Earlier this month, NICE also published final draft guidance recommending the use of Dova Pharmaceuticals’ Doptelet (avatrombopag) for thrombocytopenia who are scheduled to undergo an invasive procedure.
Doptelet has a list price of £640 or £960 per five-day treatment course, for the 40,000 to below 50,00 and below 40,000 platelets per microlitre of blood groups respectively.
Mulpleo, on the other hand, has a cost of £800 per seven-day treatment costs, although this may vary in different settings due to ‘negotiated procurement discounts’.