Orladeyo™ (berotralstat; BioCryst) is now available for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients aged 12 years and older. The oral, once-daily treatment will be shipped to patients with a prescription by specialty pharmacy Optime Care, Inc.
In patients with HAE due to C1-inhibitor deficiency/dysfunction, uncontrolled increases in plasma kallikrein activity result in angioedema attacks. Berotralstat works by decreasing plasma kallikrein activity to control excess bradykinin generation, thereby reducing the swelling and pain associated with HAE.
The approval of Orladeyo was based on data from the phase 3 APeX-2 trial, which showed that treatment with the plasma kallikrein inhibitor significantly reduced the rate of HAE attacks when compared with placebo. Orladeyo-treated patients also reported meaningful improvements in both quality of life and significant reductions in their monthly use of standard of care on-demand medications.
As for safety, the most common adverse reactions (incidence of greater than or equal to 10%) reported with Orladeyo were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. Additionally, an increase in QT prolongation can occur at dosages higher than the recommended 150mg once daily dosage; additional doses or doses higher than 150mg once daily are not recommended.
Orladeyo is supplied as 110mg and 150mg capsules of berotralstat in 28-count blister cards. Dosage adjustment is recommended in patients with hepatic impairment, those with persistent gastrointestinal reactions, and with concomitant use of P-gp or BCRP inhibitors (eg, cyclosporine).
To help patients gain access to treatment, BioCryst has launched EMPOWER Patient Services. “Our goal is to provide a best-in-class partnership that enables an individualized approach for physicians and their patients,” said Charlie Gayer, chief commercial officer of BioCryst. “From the transition to Orladeyo, coordination of deliveries, to ongoing patient support, EMPOWER puts the HAE patient at the center.”
Additional information about EMPOWER Patient Services, as well as a start form, can be found here.
BioCryst announces U.S. availability of ORLADEYO™ (berotralstat) for the treatment of hereditary angioedema. [press release]. December 16, 2020.