Scholar Rock gets FDA OK for SMA drug ISEMBYLD

Scholar Rock corrects prior comments by confirming FDA-mandated post-marketing pregnancy safety study and fracture reporting for newly approved ISEMBYLD.

Rhea-AI Filing Summary

Scholar Rock Holding Corp (SRRK) reported that the U.S. Food and Drug Administration has approved ISEMBYLD™ (apitegromab-mstn) for the treatment of spinal muscular atrophy in adults and children 2 years of age and older who are receiving a survival motor neuron 2 (SMN2)-targeted treatment. The company clarified remarks made during an investor call, where management had incorrectly stated that the FDA did not require post-marketing safety commitments for bone or reproductive safety. Scholar Rock disclosed that, as part of the approval, the FDA imposed a post-marketing requirement to establish a pregnancy safety study to collect and analyze data on pregnancy complications and birth outcomes in exposed pregnancies, and also requested submission of all serious and non-serious domestic and foreign fracture cases as 15-day “alert reports.” Scholar Rock stated its intention to conduct this study and submit these reports in accordance with the FDA’s request.

Positive

• FDA approval of ISEMBYLD™ for spinal muscular atrophy in adults and children 2+ receiving SMN2-targeted treatment represents a major regulatory milestone for Scholar Rock’s lead therapy.

Negative

• None.

8-K Event Classification

Item 7.01 — Regulation FD Disclosure

1 item

Item 7.01 Regulation FD Disclosure Disclosure

Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.

Key Figures

Minimum patient age for ISEMBYLD treatment: 2 years Fracture alert report timing: 15 days Regulatory agency: U.S. Food and Drug Administration

3 metrics

Minimum patient age for ISEMBYLD treatment 2 years Approved indication covers adults and children 2 years of age and older receiving SMN2-targeted treatment

Fracture alert report timing 15 days All serious and non-serious domestic and foreign fracture cases must be submitted as 15-day “alert reports”

Regulatory agency U.S. Food and Drug Administration Approved ISEMBYLD and outlined post-marketing pregnancy study and fracture reporting requirements

Key Terms

Regulation FD, post-marketing requirement, pregnancy safety study, 15-day “alert reports”, +2 more

6 terms

Regulation FD regulatory

"Item 7.01. Regulation FD Disclosure."

Regulation FD is a rule that prevents company insiders, like executives, from sharing important information with some people before others get it. It matters because it helps ensure all investors have equal access to key news, making the stock market fairer and reducing chances of insider trading.

post-marketing requirement regulatory

"the FDA outlined a post-marketing requirement that the Company establish a pregnancy safety study"

A post-marketing requirement is a regulatory obligation for a company to collect more safety or effectiveness data after a product is approved and sold, similar to a follow-up check after permission is granted. Investors care because these required studies or reports can affect future sales, trigger label changes, additional costs, or even regulatory action if new risks emerge, so they influence a product’s long-term revenue and company risk profile.

pregnancy safety study medical

"a post-marketing requirement that the Company establish a pregnancy safety study"

15-day “alert reports” regulatory

"submit all serious and non-serious domestic and/or foreign cases of fractures as 15-day “alert reports.”"

spinal muscular atrophy medical

"for the treatment of spinal muscular atrophy in adults and children"

A genetic condition in which the “wiring” between the spinal cord and muscles fails, causing progressive loss of muscle strength and motor skills from infancy through adulthood as the nerve cells that move muscles deteriorate. Investors pay attention because the condition creates a clear, long-term need for specialized therapies, diagnostics and ongoing care — meaning successful drugs or tests can attract premium pricing, sustained demand and significant regulatory and reimbursement scrutiny that materially affect biotech and healthcare valuations.

SMN2-targeted treatment medical

"who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment"

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did the FDA approve for Scholar Rock (SRRK)?

The FDA approved ISEMBYLD™ (apitegromab-mstn) for treatment of spinal muscular atrophy in adults and children 2 years and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.

What error did Scholar Rock (SRRK) correct from its investor call?

Scholar Rock corrected that management had wrongly stated the FDA required no post-marketing safety commitments. The company clarified that the FDA did impose a post-marketing requirement for a pregnancy safety study and requested fracture case reporting.

What post-marketing pregnancy safety study must Scholar Rock conduct for ISEMBYLD?

The FDA required Scholar Rock to establish a pregnancy safety study to collect and analyze information on pregnancy complications and birth outcomes for pregnancies exposed to ISEMBYLD.

What fracture reporting obligations did the FDA place on Scholar Rock (SRRK)?

The FDA requested that Scholar Rock submit all serious and non-serious domestic and foreign fracture cases as 15-day “alert reports” following ISEMBYLD’s approval.

Does Scholar Rock intend to comply with the FDA’s post-marketing requests?

Yes. Scholar Rock stated it intends to conduct the pregnancy safety study and submit the required fracture “alert reports” in compliance with the FDA’s requests for ISEMBYLD.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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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 15, 2026

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 14, 2026, Scholar Rock Holding Corporation (the “Company”) hosted a live audio webcast (the “Investor Call”) announcing that the U.S. Food and Drug Administration (“FDA”) has approved ISEMBYLD™ (apitegromab-mstn) for the treatment of spinal muscular atrophy in adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. During the “questions and answers” portion of the Investor Call, the Company’s management stated in error that the FDA did not require any post-marketing safety commitments regarding bone or reproductive safety of ISEMBYLD. In connection with its approval of ISEMBYLD, the FDA outlined a post-marketing requirement that the Company establish a pregnancy safety study to collect and analyze information on pregnancy complications and birth outcomes for exposed pregnancies. Separately, the FDA also requested that the Company submit all serious and non-serious domestic and/or foreign cases of fractures as 15-day “alert reports.” The Company intends to conduct this study and submit any such reports in compliance with the FDA’s request.

The information in Item 7.01 of this Form 8-K 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.

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 15, 2026	By:	/s/ Junlin Ho
		Junlin Ho
		General Counsel & Corporate Secretary

Filing Exhibits & Attachments

3 documents

Other Documents

• EX-101 XBRL TAXONOMY EXTENSION SCHEMA 3.0 KB
• EX-101 XBRL TAXONOMY EXTENSION LABEL LINKBASE 33.4 KB
• EX-101 XBRL TAXONOMY EXTENSION PRESENTATION LINKBASE 21.8 KB