FDA Approves Berotralstat for Hereditary Angioedema prophylaxis
The US Food and Drug Administration has approved oral, once-daily ORLADEYO™ (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older on December 3, 2020.
"ORLADEYO offers people with HAE and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents an important and welcome step in making more treatment options available to physicians and patients," said Anthony J. Castaldo, president and chief executive officer of the US Hereditary Angioedema Association (HAEA).
ORLADEYO™ (berotralstat) has become the first drug approved by the U.S. Food and Drug Administration (FDA) indicated for HAE attack prophylaxis. Berotralstat is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) and at a dose of 150 mg, it is a moderate inhibitor of CYP2D6 and CYP3A4. In a pivotal Phase 3 APeX-2 trial, berotralstat significantly reduced HAE attacks at 24 weeks and sustained through 48 weeks. Among patients treated with 150 mg of berotralstat, the researchers observed a significant reduction in HAE attack from baseline (2.9)to 48 weeks(1.0). In a long-term open-label assessment of the same patient population, researchers observed a decrease to just 0.8 attacks monthly. In both the trials, berotralstat found to be safe and well-tolerated. However, the researchers also observed some Gastrointestinal adverse reactions which occurred early after initiation of treatment and became less frequent with time and typically self-resolved.
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