The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of Orladeyo (berotralstat) for routine prevention of recurrent attacks of HAE in adult and adolescent patients aged 12 years and older.
The European Commission (EC) will review the CHMP recommendation and a final approval decision from the EC on the marketing authorization application (MAA) for Orladeyo is expected in the second quarter. If approved, Orladeyo would be the first oral, once-daily therapy in the European Union to treat patients with HAE by preventing recurrent attacks. The CHMP positive opinion is based on data from the pivotal APeX-2 clinical trial and supporting data from the APeX-S trial. In APeX-2, Orladeyo met its primary endpoint (p<0.001) for ORLADEYO 150 mg compared to placebo. Orladeyo showed a positive safety profile and was generally well-tolerated over 48 weeks in both APeX-2 and APeX-S.
“Following the recent approvals in the U.S. and Japan, we continue to focus on bringing our oral, once-daily treatment to HAE patients around the world,” says Jon Stonehouse, President and CEO of BioCryst Pharmaceuticals, Inc. “The positive CHMP opinion for Orladeyo is an important step closer to delivering a new option to HAE patients across Europe and our commercial team is in place and ready to launch quickly upon final EC approval.”
In December 2020, the U.S. Food & Drug Administration (FDA) approved Orladeyo in the U.S. In January 2021, Orladeyo was approved by the Ministry of Health, Labour and Welfare in Japan. An Early Access to Medicines Scheme (EAMS) for HAE patients has been approved by the Medicines & Healthcare products Regulatory Agency in the United Kingdom.