UK – Takeda’s HAE drug Takhzyro backed by NICE

New treatment option for rare, hereditary disease. Takeda’s drug for the rare disease hereditary angioedema (HAE) should be made available routinely on the NHS in England and Wales, says NICE.

The cost-effectiveness agency has issued its final appraisal document for Takhzyro (lanadelumab) as an injectable treatment option for preventing recurrent attacks in type I/II HAE patients aged 12 and over, provided they are having two or more clinically significant attacks per week despite the use of oral prevention drugs.

According to the guidance document, Takhzyro should be used at the lowest-dose possible, and can only be prescribed subject to a confidential pricing and access deal between Takeda and the NHS.

HAE is a genetic disorder that can cause unpredictable attacks of debilitating and painful swelling in various parts of the body which can last for a number of days. Attacks that obstruct the airways can cause suffocation and are potentially life-threatening.

First-line preventive treatment is with oral drugs like danazol and oxandrolone, and with C1 esterase inhibitors like Takeda’s intravenous Cinryze and CSL Behring’s injectable Berinert that are also used to treat swelling once it has become established. Cinryze is approved for prevention, and while Berinert isn’t, NICE says the drug is sometime prescribed off-label for this purpose…