Takeda’s Takhzyro (lanadelumab) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to prevent recurrent attacks of hereditary angioedema (HAE) in patients aged two to less than 12 years.
The regulator’s decision expands Takhzyro’s previous paediatric indication in patients aged 12 years and older and makes the drug the first long-term prophylactic treatment of HAE available in the UK for children aged two to under six years.
Estimated to affect one in every 50,000 people, HAE is a rare genetic disorder characterised by recurring oedema (swelling) attacks in various parts of the body, including the abdomen, face, hands and throat. The swelling can be debilitating and painful, and attacks that obstruct the airways can cause asphyxiation and are potentially life-threatening.
“HAE, like so many other rare diseases, is highly complex, and patients, their families and caregivers often undergo years of strain trying to understand their disease, get a definitive diagnosis and gain access to the medicines they need,” Takeda said in a statement.
Takhzyro, which can be administered subcutaneously by patients or their caregivers following appropriate training, is a fully human monoclonal antibody that helps to prevent the swelling and related symptoms of HAE by binding to and blocking the action of an enzyme in the blood called kallikrein.
The MHRA’s latest approval of the therapy is supported by results from the late-stage SPRING study in combination with extrapolation of data from the pivotal adult and adolescent HELP study DX-2930-037 and by data supporting the new 150mg pre-filled syringe formulation.
Commenting on the decision, Angela Metcalfe, chief executive officer of HAE UK, said: “The announcement that children under 12 now have access to this treatment option will be looked back on as a huge milestone for the HAE community.
“It is vitally important that those with HAE who are most vulnerable – no matter how small in number – are looked after.”
The approval comes less than a month after Takeda’s HyQvia was approved by the MHRA as a maintenance therapy for the rare neurological disorder chronic inflammatory demyelinating polyneuropathy (CIDP).
HyQvia (immune globulin infusion 10% [human] with recombinant human hyaluronidase) can now be used in patients of all ages with CIDP following stabilisation with intravenous immunoglobulin therapy.