The U.K.'s National Institute for Health and Care Excellence recommended Takeda Pharmaceutical Company Ltd.'s Takhzyro to treat patients with a rare genetic disorder known as hereditary angioedema.
The drug pricing watchdog said in a final draft guidance that Takhzyro, or lanadelumab, will be made available in the U.K.'s National Health Service as a treatment for patients aged 12 years and above who are also eligible for preventative C1-esterase inhibitor therapy. The agency recommended a starting dose of 300 milligrams to be administered as a subcutaneous injection every two weeks.
Hereditary angioedema, or HAE, is a rare, genetic disorder that results in recurring attacks of swelling in various parts of the body, which can be life-threatening.
The agency's decision was backed by new evidence that showed the company's medicine reduced the average number of monthly HAE attacks by 87% compared to placebo in a phase 3 trial dubbed Help-03.
Takhzyro will be made available to the NHS for a discount. Its list price is £12,420 per 300-microgram vial.
The Japanese drugmaker said the NHS is expected to make Takhzyro available in England within three months following the publication of final guidance and pending any related appeals.
Takhzyro, which is approved in the U.S. and certain other countries for HAE, was inherited by Takeda following its £46 billion acquisition of Shire PLC. The drug is a rival to Dutch biotechnology company Pharming Group NV's Ruconest, which is also approved in the U.S. to treat HAE.
