Scholar Rock Holding Corporation filed a Form 8-K on September 14, 2026, reporting that the U.S. Food and Drug Administration 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 an SMN2 targeted treatment.

Key Highlights

  • FDA Approval: The FDA approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA).
  • Eligible Population: The approval covers adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2) targeted treatment.
  • Announcement Date: The company issued the press release announcing FDA approval on September 11, 2026.
  • Investor Call: The company hosted a webcast and conference call on September 14, 2026 at 8:00 a.m. Eastern Time to discuss the approval.
  • Exchange Listing: Scholar Rock common stock (par value $0.001 per share) trades on The Nasdaq Global Select Market under the symbol NASDAQ:SRRK.

Scholar Rock Discloses FDA Approval of ISEMBYLD for Spinal Muscular Atrophy

According to the filing, Scholar Rock Holding Corporation announced on September 11, 2026 that the FDA approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy. The approved indication covers adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2) targeted treatment, as described in both Item 7.01 and Item 8.01 of the current report.

The company disclosed that a press release announcing the FDA approval was furnished as Exhibit 99.1 and that presentation materials connected to the announcement were furnished as Exhibit 99.2. The filing noted that management reviewed those presentation materials during a webcast and conference call held on September 14, 2026 at 8:00 a.m. Eastern Time. The filing was signed by Junlin Ho, General Counsel and Corporate Secretary, on September 14, 2026.

The filing specifies that the information disclosed under Item 7.01, including Exhibits 99.1 and 99.2, "shall not be deemed 'filed' for purposes of Section 18 of the Securities Exchange Act of 1934, as amended," and is not subject to the liabilities of that section unless expressly incorporated by reference in a subsequent filing.

What Was Disclosed

Scholar Rock Holding Corporation reported via Form 8-K that the FDA approved its drug ISEMBYLD (apitegromab-mstn) on September 11, 2026 for treating SMA in adults and children 2 years of age and older currently receiving an SMN2 targeted treatment.