Benzinga | Feb 25, 2021 05:31PM EST

BioCryst Receives Positive CHMP Opinion for ORLADEYO, an Oral, Once-daily Therapy to Prevent Attacks in Patients with Hereditary Angioedema

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that 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(tm) (berotralstat) for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12years 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," said Jon Stonehouse, president and chief executive officer of BioCryst. "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.