Scholar Rock (NASDAQ: SRRK) receives FDA Complete Response Letter for SMA drug apitegromab

Rhea-AI Filing Summary

Scholar Rock Holding Corporation reported that the U.S. Food and Drug Administration has issued a Complete Response Letter (CRL) for its Biologics License Application for apitegromab, a treatment candidate for spinal muscular atrophy (SMA). The company disclosed that it announced the CRL in a press release dated September 23, 2025, which is furnished as an exhibit. A CRL means the FDA has completed its review of the application and, instead of approving it in its current form, has provided feedback that must be addressed before approval can be granted.

Positive

• None.

Negative

• FDA Complete Response Letter for apitegromab BLA delays potential approval for spinal muscular atrophy and introduces additional regulatory steps before the product can be reconsidered.

Insights

Biotech regulatory and drug development analyst

FDA Complete Response Letter delays potential approval of apitegromab for SMA.

The disclosure that the FDA has issued a Complete Response Letter for Scholar Rock’s Biologics License Application for apitegromab in SMA indicates the application was not approved in its current form. A CRL generally outlines issues that must be resolved before the agency will reconsider approval, which can range from additional data requests to manufacturing or labeling concerns, though specific details are not provided here.

For a company focused on a specific indication like spinal muscular atrophy, regulatory setbacks for a lead asset can be significant because they may affect the timing and certainty of potential product launch. Future communications from the company and any follow-up regulatory interactions will determine how quickly the BLA can be resubmitted after addressing the FDA’s feedback.

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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): September 23, 2025

Scholar Rock Holding Corporation
(Exact Name of Registrant as Specified in Charter)

Delaware	001-38501	82-3750435
(State or Other Jurisdiction of Incorporation)	(Commission File Number)	(I.R.S. Employer Identification Number)
301 Binney Street, 3rd Floor, Cambridge, MA 02142
(Address of Principal Executive Offices) (Zip Code)

(857) 259-3860

(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, par value $0.001 per share	SRRK	The 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 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). 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 7.01. Regulation FD Disclosure.

On September 23, 2025, Scholar Rock Holding Corporation (the “Company”) issued a press release announcing that the U.S. Food and Drug Administration (“FDA”) has issued a Complete Response Letter (“CRL”) regarding the Biologics License Application (“BLA”) for apitegromab for the treatment of spinal muscular atrophy (“SMA”). A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.

The information in Item 7.01 of this Form 8-K, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.

Item 8.01. Other Events.

On September 23, 2025, the Company announced that the FDA has issued a CRL regarding the BLA for apitegromab for the treatment of SMA.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.		Description
99.1		Press Release issued by the Company on September 23, 2025, furnished hereto.
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.

	Scholar Rock Holding Corporation
Date: September 23, 2025	By:	/s/ Junlin Ho
		Junlin Ho
		General Counsel & Corporate Secretary

FAQ

What did Scholar Rock Holding Corporation (SRRK) disclose in this 8-K?

The company disclosed that the U.S. Food and Drug Administration issued a Complete Response Letter for its Biologics License Application for apitegromab for the treatment of spinal muscular atrophy (SMA), as announced in a press release dated September 23, 2025.

What is the status of Scholar Rock’s apitegromab application for SMA?

Scholar Rock reported that the FDA has issued a Complete Response Letter to its BLA for apitegromab for SMA, meaning the application was not approved in its current form and the FDA has provided comments that must be addressed before approval can be granted.

When did Scholar Rock announce the FDA Complete Response Letter for apitegromab?

The company announced the FDA’s Complete Response Letter regarding the BLA for apitegromab on September 23, 2025 and furnished a related press release as Exhibit 99.1.

Where can investors find more details about the FDA’s CRL to Scholar Rock?

Additional details are provided in the company’s press release dated September 23, 2025, which is furnished as Exhibit 99.1 to this 8-K and incorporated by reference for informational purposes.

Does this 8-K affect Scholar Rock’s financial statements directly?

This 8-K is presented under Regulation FD Disclosure and Other Events and focuses on the FDA’s Complete Response Letter for apitegromab; it does not present updated financial statements within the text provided.

What exhibits are included with this Scholar Rock (SRRK) 8-K filing?

The filing includes Exhibit 99.1, a press release issued by the company on September 23, 2025, and Exhibit 104, the cover page interactive data file embedded within the Inline XBRL document.