INTENDED FOR CANADIAN AUDIENCES ONLY
RESEARCH TRIANGLE PARK, N.C., Sept. 18, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Institut national d'excellence en santé et services sociaux (INESSS) has issued a positive recommendation for PrORLADEYO® (berotralstat) to be reimbursed for the prevention of hereditary angioedema (HAE) attacks in adults and pediatric patients 12 years of age and older in Quebec.
“While much progress has been made on behalf of the HAE community in Québec, people living with HAE continue to be in need of additional treatment options to help manage their HAE attacks. On behalf of people living with HAE and their caregivers, I appreciate this positive recommendation from INESSS, as it should help ensure this oral prophylactic therapy is available to those who could benefit from it. Additionally, this recommendation is a step forward as it highlights the need for continued, and simple access for people living with HAE, and their caregivers,” said Charles St-Pierre, president of Hereditary Angioedema Québec (AOH Québec).
“As a physician who treats HAE patients, I see first hand how people respond to different HAE treatments, and not every patient’s experience is the same. Today’s announcement is encouraging, as I have seen the benefits of ORLADEYO in helping to reduce the burden of disease in the convenience of an oral, once-daily therapy, and I am pleased INESSS acknowledges the value of this important prophylactic option,” said Dr. Louis Marois MD, PhD, FRCP.
“We are committed to bringing ORLADEYO to as many Canadians living with HAE as possible. This announcement from INESSS, which follows the positive recommendation from CADTH earlier this year, takes us one step closer to making our oral, once-daily therapy available to Canadians who are in need of a new option to help improve control of their HAE attacks,” said Anand Janack, vice president and general manager of BioCryst Canada.
Health Canada authorized ORLADEYO in June 2022 for the routine prevention of recurrent HAE attacks in patients 12 years and older. In February 2023, the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee issued a positive recommendation for ORLADEYO.
The full INESSS reimbursement recommendation for ORLADEYO is available on INESSS’ website.
About PrORLADEYO® (berotralstat)PrORLADEYO® (berotralstat) is an oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
INDICATIONPrORLADEYO® (berotralstat) is indicated for the routine prevention of attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older.
LIMITATIONS OF USEThe safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.
IMPORTANT SAFETY INFORMATIONThe overall safety of ORLADEYO has been evaluated in multiple long-term clinical studies, which included 381 patients with HAE (uncontrolled, open-label and placebo-controlled, blinded studies).
Of the patients treated with ORLADEYO in the placebo-controlled blinded Phase 3 study (Study 302, Part 1), the most common adverse reactions associated with ORLADEYO 150 mg were gastrointestinal reactions, which included abdominal pain in any location (23%), vomiting (15%), and diarrhea (15%). These reactions generally occurred early after initiation of treatment with ORLADEYO, became less frequent with time, and typically self-resolved. No patients in the ORLADEYO 150 mg dose group discontinued treatment due to a gastrointestinal adverse reaction. There were no serious drug-related treatment-emergent adverse events in patients who received ORLADEYO.
Patients with moderate or severe hepatic impairment may develop increased serum berotralstat concentrations. Use of ORLADEYO in these patients should be avoided.
ORLADEYO is a P-glycoprotein (P-gp) and breast cancer resistance protein (BCRP) substrate. ORLADEYO exposure may be increased with concomitant administration of P-gp and BCRP inhibitors, but no dose adjustment is necessary. Close monitoring for adverse events is recommended for concomitant use with P-gp and BCRP inhibitors. P-gp and BCRP inducers (e.g., rifampicin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of berotralstat. The use of P-gp inducers is not recommended with ORLADEYO.
ORLADEYO at a once-daily dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose adjustment of these medications may be required.
The safety and effectiveness of ORLADEYO in pediatric patients