The U.S. Food and Drug Administration (FDA) has approved Orladeyo (berotralstat), a once-daily, pill as a prophylaxis treatment for hereditary angioedema (HAE) attacks.
More specifically, the drug is indicated to treat adults and pediatric patients 12 years and older with HAE.
Hereditary angioedema (HAE) is a rare genetic disease that results in immunologic attacks that can be life threatening. HAE is due to reduced levels of C1-inhibitor, a protein involved in a number of physiological processes in plasma, most notably in the complement system. C1-inhibitor also binds and inhibits plasma kallikrein and factor XIa, thereby affecting bradykinin production. It is believed that disruptions of these processes lead to fluid leaking from the blood to connective tissue in people with HAE that leads to HAE attacks.
Current treatments are designed to prevent HAE attacks or reduce their severity, and there are now eight FDA approved treatments for HAE. Four focused on the C1-inhibitor (Bertinert, Cinryze, Heagarda, Ruconest) and four that alter the bradykinin/kallikrein system (Firazyr, Kalbitor, Takhzyro, Orladeyo). Orladeyo (berotralstat) is the only treatment available as a capsule.
The approval was largely based on the Phase 3 APeX-2 trial study showing that after 48 weeks of treatment with berotralstat (150 mg capsule taken daily), HAE patients saw a reduction in mean HAE attacks from 2.9 to 1.0 attacks/month. The medication was generally safe and well tolerated with the most common adverse events being gastrointestinal (e.g., abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease).
To learn more about latest research in HAE, watch our exclusive CME program with Timothy Craig, DO, one of the investigators in the Apex-2 trial.