INESSS Recommends Public Reimbursement for BioCryst’s ORLADEYO® (berotralstat) for the Prevention of Attacks for Hereditary Angioedema Patients in Québec
INTENDED FOR CANADIAN AUDIENCES ONLY
RESEARCH TRIANGLE PARK, N.C., Sept. 18, 2023 (GLOBE NEWSWIRE) -- (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’ .
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.
INDICATION
PrORLADEYO® (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 USE
The 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 INFORMATION
The 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 <12 years of age have not been established.
There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production. There are no data on the influence of ORLADEYO use on human fertility.
The frequent side effects include: abdominal discomfort; vomiting; diarrhea; back pain; headache; heartburn; gas; rash; liver function test elevations (shown in blood tests).
You can report any suspected side effects associated with the use of health products to Health Canada by:
- Visiting the Web page on Adverse Reaction Reporting () for information on how to report online, by mail or by fax; or:
- Calling toll-free at 1-866-234-2345
Please see full .
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily PrORLADEYO® (berotralstat) is approved in the United States and many global markets. BioCryst has active programs to develop oral medicines for multiple targets across the complement system, including BCX10013, an oral Factor D inhibitor in clinical development. RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing. For more information, please visit the company’s website at .
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst’s business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst’s and its partners’ development, regulatory processes and supply chains, negatively impact BioCryst’s ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst periodically files with the Securities and Exchange Commission; BioCryst’s ability to successfully implement its commercialization plans for, and to commercialize, ORLADEYO, which could take longer or be more expensive than planned; the commercial viability of ORLADEYO, including its ability to achieve market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management's expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s forward-looking statements.
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