The U.S. Food and Drug Administration has approved Shire‘s Takhzyro (lanadelumab-flyo), a first-of-its-kind antibody injection to prevent attacks of hereditary angioedema (HAE) in patients age 12 and older.
The approval follows a priority review granted by FDA to Takhzyro, which helps accelerate the clinical program of an investigational drug that is expected to have a great impact on the treatment of a certain disease.
HAE is a chronic, rare, genetic disease characterized by recurrent attacks of edema (swelling) in the deeper layers of the skin.
“HAE attacks are painful, debilitating, and potentially life threatening. Takhzyro provides the HAE community with a new option for the prevention of HAE attacks,” Anthony J. Castaldo, president of the U.S. Hereditary Angioedema Association, said in a news release.
“We are grateful for the time and effort put forth by the patients and researchers who participated in the clinical trial program that enabled this important addition to the HAE treatment landscape,” Castaldo added.
Takhzyro is a monoclonal (one type) antibody designed to inhibit plasma kallikrein, a protein that is chronically uncontrolled in people with HAE, leading to attacks of swelling.
Takhzyro’s approval was supported by positive data from Shire’s Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) study (NCT02586805), the largest prevention study conducted to date in HAE and designed to evaluate the safety and effectiveness of the therapy in preventing (prophylaxis) HAE attacks.
The trial showed that Takhzyro (300 mg every two weeks) effectively reduced the number of monthly swelling attacks by an average of 87% compared to placebo, and improved quality-of-life scores in HAE patients when used as a preventive treatment.
Based on these data, the recommended dose of Takhzyro was set to 300 mg every two weeks, although an interval of four weeks can also be considered if the patient is well-controlled (attack-free) for more than six months.
Four clinical trials contributed to the FDA approval of Takhzyro, including the HELP study. Of the patients who completed the HELP study and who received the treatment, 97% enrolled in an ongoing extension study designed to evaluate the long-term safety and efficacy of Takhzyro.
In April this year, the European Medicines Agency (EMA) also announced the acceleration of the review process for Takhzyro in Europe.
And, a marketing authorization application for Shire’s medicine was cleared by the Swiss Agency for Therapeutic Products (Swissmedic), demonstrating the global urgency for the approval of an HAE treatment.
“With the approval of Takhzyro, HAE patients have an innovative treatment that works differently than current options to help prevent attacks,” said Andreas Busch, PhD, executive vice president and head of research and development at Shire.
“Based on an exploratory and post hoc analysis, after six doses of Takhzyro 300 mg every two weeks, 77% or nearly eight of ten patients had zero attacks,” he said.
“This approval reinforces our ongoing commitment to developing novel therapies that have a meaningful impact on patients. Looking to the future, we continue to work towards our goal of a world in which those living with HAE can aim for zero attacks,” Busch added.