CHMP Recommends EU Approval of Shire’s HAE Drug

Rare Daily Staff

The European Commission’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion on Shire’s lanadelumab for the preventive treatment of hereditary angioedema in patients aged 12 years and older.

Hereditary angioedema (HAE) is a rare, genetic disorder that results in recurring attacks of edema in various parts of the body, including the abdomen, face, feet, genitals, hands and throat, that can be debilitating and painful. Laryngeal attacks that obstruct the airways are potentially life-threatening due to the risk of asphyxiation.

Lanadelumab, a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity. Lanadelumab is formulated for subcutaneous administration and has a half-life of approximately two weeks in patients with HAE.⁶ Lanadelumab is intended for self-administration or administration by a caregiver, only after training by a healthcare professional.

If approved, lanadelumab will be a first-of-its-kind, fully human monoclonal antibody available in the European Union that inhibits the activity of plasma kallikrein, an enzyme which is uncontrolled in people with HAE, to help prevent attacks.

“This positive opinion marks an important step towards providing adults and adolescents living with HAE in Europe a first-of-its-kind monoclonal antibody treatment option to help prevent attacks,” said Andreas Busch, executive vice president, head of research and development at Shire. “We are excited about the future potential of lanadelumab in helping to address the needs of those living with this chronic and unpredictable disease.”

The positive opinion is supported by data from the phase 3 HELP study, the largest randomized controlled prevention study conducted to date in HAE, which evaluated the efficacy and safety of subcutaneously administered lanadelumab versus placebo over 26 weeks in 125 patients 12 years of age or older with HAE.

Lanadelumab was previously granted accelerated assessment by the EMA, reducing the number of evaluation days required from 210 to 150. The CHMP’s positive opinion will be reviewed by the European Commission, which has the authority to grant marketing authorization in the European Union.

Lanadelumab won approval for the prevention of HAE attacks in patient 12 years and older in the United States in August and in Canada in September under the brand name Takhzyro.

October 22, 2018
Photo: Andreas Busch, executive vice president, head of research and development at Shire