Prefilled Syringe for At-home Use of Takhzyro Favored for Approval in EU

The Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), is recommending the approval of a prefilled syringe to administer Takhzyro (lanadelumab), a preventative treatment for hereditary angioedema (HAE) in patients 12 or older.

The European Commission (EC), which typically accepts CHMP opinions, is expected to announce a final decision within 12 months. But as Takhzyro is approved as an under-the-skin injection treatment across the European Union, this new request is for a “type 2” variation changing only how it is given.

As such, Takeda Pharmaceuticals, which markets the treatment, said in a press release that under this variation’s terms it expects to launch the prefilled syringe formulation in the EU later this year.

Currently, Takhzyro is given as a 300 mg solution (in a vial), administered via an injection every two weeks. While intended for at-home use, training from a healthcare professional is necessary. The therapy, with a similar label, was approved in both Europe and the U.S. in 2018.

The prefilled syringe comes fully assembled, making at-home use easier.

“Our goal is to continuously innovate in all areas of HAE management,” Isabel Kalofonos, the global product strategy lead for HAE at Takeda, said in the release. “This positive opinion marks another important step forward as we aim to enhance the experience of treatment administration for people receiving Takhzyro.”

Hereditary angioedema is a chronic genetic condition characterized by severe and unpredictable swelling attacks underneath the skin. These attacks are caused by excess production of a protein called bradykinin, which is responsible for promoting inflammation.

Takhzyro is a monoclonal antibody designed to attach to and deactivate the protein kallikrein, which regulates the amount of bradykinin that enters the bloodstream. By blocking kallikrein activity, Takhzyro works to limit the ability of bradykinin to cause inflammatory attacks in HAE patients.

The treatment was investigated in a Phase 3 clinical trial (NCT02586805), called HELP, in 125 people with type 1 or type 2 HAE. Patients were treated with either Takhzyro at 150 mg every four weeks, or 300 mg every two or four weeks, or given a placebo.

Researchers found that treatment at 300 mg every two weeks led to an average 87% reduction in attack frequency each month. It also increased the number of patients who were attack-free during the study compared to the placebo (44.4% versus 2.4%).

Takhzyro was seen to be safe and well tolerated. Most commonly reported side effects seen in this trial were injection site pain and rash, headache, and upper respiratory tract infections, which were typically mild in severity.

“We look forward to bringing the pre-filled syringe innovation to the HAE community in Europe, starting later this year, and continue to progress plans to expand to other geographies in future months,” Kalofonos added.

Shire, which is now part of Takeda, developed Takhzyro.