The U.S. Food and Drug Administration (FDA) has approved oral, once-daily Orladeyo (berotralstat) for prophylaxis to prevent attacks of HAE in adults and pediatric patients 12 years and older.
“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,” says Anthony J. Castaldo, President and CEO of the US Hereditary Angioedema Association (HAEA).
In the pivotal Phase 3 APeX-2 trial, Orladeyo significantly reduced attacks at 24 weeks, and this reduction was sustained through 48 weeks. HAE patients who completed 48 weeks of treatment (150 mg) saw reductions in their HAE attack rates, from a mean of 2.9 attacks per month at baseline to a mean of 1.0 attacks per month after 48 weeks of therapy. In the long-term open label APeX-S trial, patients completing 48 weeks of therapy (150 mg) had a mean attack rate of 0.8 attacks per month.
Orladeyo was safe and well tolerated in both trials. The most frequently reported adverse reactions in patients receiving Orladeyo compared with placebo were gastrointestinal reactions. These reactions generally occurred early after initiation of treatment with Orladeyo, became less frequent with time and typically self-resolved.
“Patients and physicians acknowledge that HAE treatments can add a burden to patients’ lives. As an oral, once-daily option, Orladeyo can provide significant attack reduction and lessen the burden associated with injections and infusions,” says Marc Riedl, M.D., Professor of Medicine and Clinical Director, U.S. Hereditary Angioedema Association Center at the University of California, San Diego, and an investigator in the APeX-2 trial “With this new treatment option, physicians and patients can continue to have collaborative discussions to choose the treatment that meets each patient’s needs, life circumstances and preferences.”
HAE patients note a significant treatment burden associated with existing prophylactic therapy. In addition to reducing HAE attack rate, data from APeX-2 show that patients reported meaningful improvements in both quality of life and overall patient-reported satisfaction, and significant reductions in their monthly use of standard of care on-demand medicine, while taking oral, once-daily Orladeyo (150 mg).
“The FDA approval of Orladeyo fulfills a promise BioCryst made to HAE patients that we were committed to helping them achieve the dream of an oral, once-daily medicine to prevent and reduce the burden of their attacks,” says Jon Stonehouse, President and CEO of BioCryst Pharmaceuticals, Inc. “Thank you to the HAE patients who participated in our clinical trials, to the investigators around the world who conducted these trials, to the HAEA for their patient advocacy and to our employees who never forgot that patients were waiting. We will stay focused on enabling access and providing personalized support to HAE patients and physicians.”
BioCryst is committed to supporting HAE patients taking Orladeyo through a new program designed to streamline access to therapy. Through EMPOWER Patient Services, each HAE patient and their healthcare provider will have a single point of contact for access to Orladeyo. A dedicated care coordinator will support access for each patient with comprehensive financial support tools and reimbursement support.
EMPOWER Patients Services is administered by Optime Care Inc., the exclusive specialty pharmacy provider for Orladeyo. Physicians can begin writing prescriptions for Orladeyo immediately, with direct to patient shipments from Optime Care expected to begin by the end of December 2020.