BioCryst (NASDAQ: BCRX) gets FDA OK for pediatric ORLADEYO pellets

Rhea-AI Filing Summary

BioCryst Pharmaceuticals reports that the U.S. Food and Drug Administration has approved its new drug application for an oral pellet formulation of once-daily ORLADEYO (berotralstat). The approval covers use of this oral pellet formulation as prophylactic therapy in pediatric patients with hereditary angioedema aged 2 to <12 years.

The company has furnished a press release with additional details about this FDA approval as an exhibit to this report.

Positive

• FDA approval of an oral pellet formulation of once-daily ORLADEYO (berotralstat) for prophylactic therapy in pediatric hereditary angioedema patients aged 2 to <12 years.

Negative

• None.

Insights

Biotech regulatory analyst

FDA approves pediatric oral pellet formulation of ORLADEYO for hereditary angioedema.

BioCryst Pharmaceuticals disclosed that the U.S. Food and Drug Administration approved its new drug application for an oral pellet formulation of once-daily ORLADEYO (berotralstat). The approval is specifically for prophylactic therapy in pediatric patients with hereditary angioedema aged 2 to <12 years, using an oral, once-daily regimen.

This decision formally establishes an FDA-approved oral pellet dosage form tailored to younger children with hereditary angioedema within the stated age range. The company also provided a press release as an exhibit, indicating that the pediatric use and formulation details are important enough to highlight separately for stakeholders.

Regulatory approvals of new formulations and age groups can influence treatment patterns in rare diseases, and this action confirms that ORLADEYO’s oral pellet form meets FDA standards for prophylactic use in pediatric hereditary angioedema patients aged 2 to <12 years.

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

_________________

FORM 8-K

_________________

CURRENT REPORT

Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported):  December 12, 2025

_______________________________

BioCryst Pharmaceuticals, Inc.

(Exact name of registrant as specified in its charter)

_______________________________

Delaware	000-23186	62-1413174
(State or Other Jurisdiction of Incorporation)	(Commission File Number)	(I.R.S. Employer Identification No.)

4505 Emperor Blvd., Suite 200

Durham, North Carolina 27703

(Address of Principal Executive Offices) (Zip Code)

(919) 859-1302

(Registrant's telephone number, including area code)

(Former name or former address, if changed since last report)

_______________________________

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐	Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐	Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐	Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐	Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock	BCRX	Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 8.01. Other Events.

On December 12, 2025, BioCryst Pharmaceuticals, Inc. (the “Company”) issued a press release announcing that the U.S. Food and Drug Administration has approved the Company’s new drug application for the use of an oral pellet formulation of once-daily ORLADEYO^® (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema aged 2 to <12 years.

A copy of the press release is furnished as Exhibit 99.1 hereto and is incorporated herein by reference.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits.

Exhibit No.		Description
99.1		Press Release dated December 12, 2025 entitled “BioCryst Announces FDA Approval of ORLADEYO® (berotralstat) Oral Pellets, First and Only Oral Prophylactic Treatment for Patients with HAE Aged 2 to <12 Years”
104		Cover Page Interactive Data File (embedded within the Inline XBRL document)

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

	BioCryst Pharmaceuticals, Inc.
Date: December 12, 2025	By:	/s/ Alane Barnes
		Alane Barnes
		Chief Legal Officer

FAQ

What FDA decision did BioCryst (BCRX) disclose in this report?

BioCryst disclosed that the U.S. Food and Drug Administration has approved its new drug application for an oral pellet formulation of once-daily ORLADEYO (berotralstat).

For which patients is the new ORLADEYO oral pellet formulation approved?

The oral pellet formulation of once-daily ORLADEYO is approved for prophylactic therapy in pediatric patients with hereditary angioedema aged 2 to <12 years.

What condition does ORLADEYO treat according to this BioCryst filing?

ORLADEYO (berotralstat) in the newly approved oral pellet formulation is indicated for prophylactic therapy in pediatric patients with hereditary angioedema.

Did BioCryst provide additional information about the ORLADEYO approval?

Yes. BioCryst furnished a press release as Exhibit 99.1 that provides additional details about the FDA approval of ORLADEYO oral pellets.

When did the FDA approval of ORLADEYO oral pellets occur for BioCryst?

The report states that on December 12, 2025, BioCryst issued a press release announcing the FDA’s approval of the new drug application for ORLADEYO oral pellets.

What type of formulation did the FDA approve for ORLADEYO in pediatric HAE?

The FDA approved an oral pellet formulation of once-daily ORLADEYO (berotralstat) for prophylactic use in pediatric hereditary angioedema patients.