Takeda’s HAE drug Takhzyro backed by NICE

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.

That makes Takhzyro the first self-injected subcutaneous treatment to be approved for use in attack prevention, says Takeda UK, and so provides a more convenient treatment option for patients.

“HAE attacks are unpredictable, painful and debilitating. [The] recommendation from NICE means that people living with HAE across England and Wales have another medication that gives them the chance to lead a full and active life,” said Laura Szutowicz, chief executive of medical charity HAE UK.

“We hope the NHS and healthcare professionals will provide swift access to patients who could benefit from this new treatment,” she added.

Like Firazyr and Cinryze, Takhzyro was originally developed by Shire, which has been absorbed by Takeda in a $63bn takeover.

Analysts have predicted that Takhzyro could make up to $2bn in peak sales. It’s given as a subcutaneous injection that takes less than a minute to deliver every two weeks, requiring around 75% fewer injections than other products for HAE prevention.