HAE patients get new option as NICE backs Takeda's Takhzyro

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Patients in England and Wales with hereditary angioedema (HAE) should soon have a new treatment option after NICE recommended Takeda’s Takhzyro (lanadelumab) should be funded by the NHS in the rare debilitating genetic disorder.

In final draft guidance NICE recommended funding as a preventative therapy in people aged 12 and older for the subcutaneous injection.

HAE is a rare genetic disorder that can result in unpredictable and potentially life-threatening recurring attacks of debilitating and painful swelling in various parts of the body, which can last for several days.

Only patients eligible for preventive C1-esterase inhibitor treatment will be eligible in line with NHS England’s commissioning policy, and the lowest dosing frequency must be used.

Takeda has also agreed a confidential discount from the list price £12,420 per 300 mg dose in the guidance with NICE, which applies to England and Wales.

The Scottish Medicines Consortium is also reviewing Takhzyro, and is due to publish guidance in the final quarter of this year.

Takhzyro was originally developed by the rare disease specialist Shire before it merged with Takeda at the beginning of this year in a deal worth around $62 billion.

There are already several HAE drugs already on the market – NICE noted in its guidance that CSL Behring’s Berinert and Takeda's Cinryze, an old Shire drug, are well established as treatments in UK clinics, while Pharming’s Ruconest is very rarely used in practice.

Berinert is only licensed to treat acute attacks but is often used in hospitals as a long-term prophylactic, NICE noted.

The final draft guidance is expected to lead to final guidance, due to be published in October.

Unless there are any appeals against NICE’s decision, NHS patients in England should be able to receive Takhzyro within three months of final guidance publication.

Laura Szutowicz, CEO of the patient group HAE UK said: “HAE attacks are unpredictable, painful and debilitating. Today’s 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. We hope the NHS and Healthcare Professionals will provide swift access to patients who could benefit from this new treatment.”