BioCryst Pharmaceuticals Inc. filed SEC Form 8-K: Other Events, Financial Statements and Exhibits

On May 14, 2025, BioCryst Pharmaceuticals, Inc. (the “Company”) announced that the U.S. Food and Drug Administration (“FDA”) has accepted its new drug application for the use of once-daily ORLADEYO® (berotralstat) oral granules in pediatric patients with hereditary angioedema (“HAE”) aged 2 to 11 years. The FDA also granted priority review of the application, with a Prescription Drug User Fee Act (“PDUFA”) target action date of September 12, 2025.

The Company also announced that it has filed its line extension application for the use of ORLADEYO oral granules in patients with HAE aged 2 to 11 years with the European Medicines Agency. Additional regulatory filings are planned in other global territories, including Japan and Canada.

On May 14, 2025, the Company issued a press release announcing the events described in this Item 8.01. A copy of the press release is attached as Exhibit 99.1 hereto and is incorporated herein by reference.

Forward-Looking Statements

This Current Report on Form 8-K contains forward-looking statements, including statements regarding future results, performance or achievements and statements relating to ORLADEYO performance. 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 Company’s ability to successfully implement or maintain its commercialization plans for ORLADEYO; the Company’s ability to successfully progress its development plans as described herein, including meeting the expected timelines; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials, and interim results of a clinical trial do not necessarily predict final results; the commercial viability of ORLADEYO for use in pediatric patients with HAE aged 2 to 11 years, including its ability to achieve sustained market acceptance; and the FDA, European Medicines Agency or other applicable regulatory agency may not approve ORLADEYO for use in pediatric patients with HAE aged 2 to 11 years within the timeframe expected, or at all, may ultimately determine that there are deficiencies in the development program or execution thereof, may require additional information or studies, may disagree with our safety and efficacy conclusions, may impose certain restrictions, warnings, or other requirements, may impose a clinical hold with respect to ORLADEYO, or may withhold, delay, or withdraw market approval for ORLADEYO. Please refer to the documents the Company files periodically with the Securities and Exchange Commission, specifically the Company’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 the Company’s forward-looking statements.

(d) Exhibits.