Orladeyo Approved to Prevent HAE Attacks

December 3, 2020 – The U.S. FDA has approved OrladeyoTM (berotralstat), manufactured by BioCryst Pharmaceuticals, to provide prophylaxis (prevention) of hereditary angioedema (HAE) attacks in patients who are at least 12 years of age. Orladeyo is the first orally administered non-steroidal therapy to receive FDA approval for this indication.

HAE is a rare genetic condition that causes painful episodes of swelling (HAE attacks) that can be life- threatening, depending on where in the body they occur. The condition prevents the body from either producing enough C1 esterase inhibitor (C1-INH) or producing C1-INH that works properly. This causes a protein known as plasma kallikrein to become overactive, which can then trigger inflammation leading to an HAE attack. Orladeyo works by decreasing the activity of plasma kallikrein to prevent attacks.

In a clinical trial, Orladeyo significantly reduced the occurrence of attacks at 24 weeks of treatment, with the reduction sustained through 48 weeks of treatment. Attacks were reduced from a mean of 2.9 attacks per month at baseline to a mean of one attack per month after 48 weeks of therapy. Patients also reported meaningful improvements in quality of life and overall satisfaction with their treatment during the study, and were able to reduce their use of on-demand treatment for HAE.

Recommended dosing with Orladeyo is one 150mg capsule taken by mouth each day with food. Dosage adjustment may be required in patients who have hepatic impairment, experience gastrointestinal reactions, or take certain medications concomitantly with Orladeyo.

BioCyrst has launched the product at a wholesale acquisition cost (WAC) of $37,308 per 28 capsules.