NHS Scotland approves new therapies for hereditary angioedema, polycythaemia vera and thrombocytopenia

  • Scottish Medicines Consortium
  • 9 Dec 2019

  • curated by Pavankumar Kamat
  • UK Medical News
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The Scottish Medicines Consortium (SMC) recently published advice pertaining to the acceptance of three new therapies for use by NHS Scotland.

Lanadelumab (Takhzyro) has been accepted for routinely preventing recurrent attacks of hereditary angioedema (HAE) in patients aged ≥12 years. The use of lanadelumab has been restricted only to patients with type I or II HAE, who would otherwise be considered for long-term prophylaxis therapy with C1-esterase inhibitor. Evidence showed that lanadelumab decreased the rate of angioedema attacks compared with placebo.

Ruxolitinib phosphate (Jakavi) has been accepted for the treatment of polycythaemia vera in adults who are refractory or intolerant to hydroxyurea. Evidence from two phase 3 studies showed superiority of ruxolitinib over best available therapy in the indicated population, with or without splenomegaly.

Lusutrombopag (Mulpleo) has been accepted for the treatment of severe thrombocytopenia in adults with chronic liver disease undergoing invasive procedures. Evidence from two phase 3 studies showed lusutrombopag was superior to placebo in decreasing the requirement for platelet transfusions in the indicated population.

The SMC advice considers the benefits of a 'Patient Access Scheme' that improves the cost-effectiveness of lanadelumab and ruxolitinib.