BioCryst Announces Approval of ORLADEYO® (berotralstat) by the Brazilian Health Regulatory Agency

Rhea-AI Summary

BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) has received approval from the Brazilian Health Regulatory Agency for ORLADEYO® (berotralstat) for the prophylaxis of hereditary angioedema attacks in adults and pediatric patients. This approval marks a significant milestone for BioCryst as they expand their market presence in Latin America.

Pharmaceutical Market Analyst

The approval of ORLADEYO by ANVISA represents a strategic expansion for BioCryst Pharmaceuticals in the Latin American market. Brazil, being the largest market in the region, is poised to significantly contribute to the company's international revenue streams. The partnership with Pint Pharma GmbH, which lays the groundwork for marketing and distribution, mitigates the logistical complexities often associated with penetrating new markets. It's essential to monitor the uptake of ORLADEYO post-launch, as competitive dynamics and pricing strategies will play a critical role in its market performance. The drug's unique value proposition as an oral, once-daily treatment may offer convenience over existing therapies, potentially leading to market share gains in the prophylaxis of hereditary angioedema (HAE).

Health Economics Expert

This development could be a game-changer for HAE patients in Brazil, who now have access to an innovative prophylactic treatment that could improve their quality of life. From a health economics perspective, the impact of ORLADEYO's approval could extend beyond immediate patient benefit to potential cost savings for the Brazilian healthcare system. By reducing the frequency of HAE attacks, there may be fewer hospitalizations and a decrease in the use of acute treatments, which tend to be more expensive. However, the drug's pricing strategy will need to be balanced with the country's healthcare budget constraints and reimbursement policies to ensure broad accessibility.

International Business Strategy Consultant

BioCryst's collaboration with Pint Pharma is an exemplary case of leveraging local expertise to navigate foreign regulatory landscapes and market complexities. This strategy can often accelerate market entry and enhance the scalability of operations. For investors, the critical aspect to consider is the execution of this partnership and the agility with which ORLADEYO is commercialized in the pan-Latin American region. As the product is rolled out, investors should pay close attention to BioCryst's ability to adapt to local market conditions, which can include diverse regulatory environments, patient access programs and competitive responses from other HAE treatments.

RESEARCH TRIANGLE PARK, N.C., April 17, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Brazilian Health Regulatory Agency (ANVISA) has granted approval for oral, once-daily ORLADEYO^® (berotralstat) for the prophylaxis of hereditary angioedema (HAE) attacks in adults and pediatric patients 12 years of age or older.

“We are excited to announce that ORLADEYO is now approved in the region’s largest market, following the positive regulatory decisions we received in Chile and Argentina last year. This is an important moment for patients living with HAE in Brazil, as they will soon be able to access our oral, once-daily prophylactic therapy to help manage their condition. We look forward to working with our partner, Pint Pharma, to make ORLADEYO available in Brazil and across LATAM,” said Charlie Gayer, chief commercial officer of BioCryst.

BioCryst has an exclusive collaboration with Pint Pharma GmbH to register and promote ORLADEYO in the pan-Latin America region. Under the terms of the agreement, Pint is responsible for obtaining and maintaining all marketing authorizations and for commercializing ORLADEYO in the region.

About ORLADEYO^® (berotralstat)
ORLADEYO^® (berotralstat) is the first and only 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.

U.S. Indication and Important Safety Information

INDICATION
ORLADEYO^® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years 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

An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.

The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C).

Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.

ORLADEYO at a 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 titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering 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.

To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information.

About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO^® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding BioCryst’s plans and expectations for ORLADEYO. 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 results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve market acceptance; the FDA, ANVISA 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 actual results to differ materially from those contained in BioCryst’s forward-looking statements.

BCRXW

Contact:
John Bluth
+1 919 859 7910
jbluth@biocryst.com

What is the latest approval announced by BioCryst Pharmaceuticals, Inc. related to the Brazilian Health Regulatory Agency?

BioCryst Pharmaceuticals, Inc. has received approval from the Brazilian Health Regulatory Agency for ORLADEYO® (berotralstat) for the prophylaxis of hereditary angioedema attacks in adults and pediatric patients.

Who is BioCryst's partner for registering and promoting ORLADEYO in the pan-Latin America region?

BioCryst has an exclusive collaboration with Pint Pharma GmbH to register and promote ORLADEYO in the pan-Latin America region.

What is the role of Pint Pharma GmbH in the collaboration with BioCryst?

Under the agreement, Pint Pharma GmbH is responsible for obtaining and maintaining all marketing authorizations and commercializing ORLADEYO in the region.