CADTH Recommends Reimbursement for BioCryst’s ORLADEYO® (berotralstat) for the Routine Prevention of Attacks in Hereditary Angioedema Patients in Canada

INTENDED FOR CANADIAN AUDIENCES ONLY

RESEARCH TRIANGLE PARK, N.C., March 08, 2023 (GLOBE NEWSWIRE) --  BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has issued a positive recommendation for ORLADEYO^® (berotralstat) to be reimbursed for the routine prevention of attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older.

“We are still in need of additional options to help people living with HAE better manage their disease, despite the incredible progress we have made to date in addressing the treatment burden. Today’s announcement is a welcome one for the HAE community in Canada, as it should help increase access to this oral prophylactic treatment across our country,” said Daphne Dumbrille, on behalf of the HAE Canada Board of Directors.

“As I have observed in my own practice, it is beneficial to have additional therapeutic options available to patients with HAE because each patient has an individualized treatment experience. In issuing this positive recommendation, CADTH is opening the door to many Canadian HAE patients being able to access ORLADEYO,” said Stephen Betschel, HBSc, MD, FRCPC, FAAAAI, Chair of the Canadian Hereditary Angioedema Network.

“BioCryst is delighted that ORLADEYO has received this positive recommendation from CADTH. I am grateful to our team here at BioCryst and our many partners in the HAE community who underscored the value of ensuring Canadian patients have access to an oral, once-daily therapy for this rare, debilitating disease,” said Anand Janack, vice president and general manager of BioCryst Canada.

Health Canada authorized ORLADEYO in June 2022 for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older. The full CADTH reimbursement recommendation for ORLADEYO is available on CADTH’s website.

About ORLADEYO^® (berotralstat)
ORLADEYO^® (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.

INDICATION¹
ORLADEYO^® (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 USE¹
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 INFORMATION¹
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.

ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose adjustment may be required for P-gp substrates when co-administered with ORLADEYO.

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.

What are the possible side effects from using ORLADEYO?
These are not all the possible side effects you may have when taking ORLADEYO. If you experience any side effects not listed here, tell your healthcare professional.

Side effects you may experience include: abdominal discomfort; vomiting; diarrhea; back pain; headache; heartburn; gas; rash; liver function test elevations (shown in blood tests).

If you have a troublesome symptom or side effect that is not listed here or becomes bad enough to interfere with your daily activities, tell your healthcare professional.

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 (https://www.canada.ca/en/health-canada/services/drugs-health-products/medeffect-canada/adverse-reaction-reporting.html) for information on how to report online, by mail or by fax; or:
• Calling toll-free at 1-866-234-2345

Please see full Product Monograph.

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 ORLADEYO^® (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 www.biocryst.com.

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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