This section highlights FDA-related milestones and regulatory updates for drugs developed by Scholar Rock (SRRK).
Over the past two years, Scholar Rock has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
apitegromab-mstn and Apitegromab. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Apitegromab-mstn FDA Regulatory Events
Apitegromab-mstn is a drug developed by Scholar Rock for the following indication: treatment of spinal muscular atrophy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- apitegromab-mstn
- Announced Date:
- September 11, 2026
- Indication:
- treatment of spinal muscular atrophy
Announcement
Scholar Rock announced that the FDA has approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children two years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.
Read Announcement
Apitegromab FDA Regulatory Timeline and Events
Apitegromab is a drug developed by Scholar Rock for the following indication: Type 2 and Type 3 Spinal Muscular Atrophy (SMA).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Apitegromab
- Announced Date:
- September 2, 2026
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that the FDA has granted Fast Track and Orphan Drug designations to apitegromab for the treatment of people living with FSHD.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- September 2, 2026
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that the FDA has granted Fast Track and Orphan Drug designations to apitegromab for the treatment of people living with FSHD.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- August 21, 2026
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced it has successfully removed Catalent Indiana LLC (part of Novo Nordisk) as a commercial fill-finish facility from its apitegromab Biologics License Application (BLA) for the treatment of spinal muscular atrophy (SMA) in the U.S., under guidance from FDA.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- August 7, 2026
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that the FDA review of the apitegromab Biologics License Application (BLA) for the treatment of children and adults with spinal muscular atrophy (SMA) is progressing with the Company’s second fill-finish facility.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- July 20, 2026
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock provided an update on the anticipated timing of a Committee for Medicinal Products for Human Use (CHMP) opinion for the apitegromab marketing authorisation application (MAA) for children and adults with spinal muscular atrophy (SMA).
AI Summary
Scholar Rock said it now expects a Committee for Medicinal Products for Human Use, or CHMP, opinion on its apitegromab marketing application for spinal muscular atrophy in children and adults during the third quarter of 2026. The company said the review is moving forward and that it is working with European regulators as part of the approval process.
Scholar Rock also noted that its second fill-finish facility for apitegromab is a U.S.-based site that already makes several commercial products. The company said the facility is in good standing with both the FDA and the EMA, which supports its manufacturing plans for apitegromab.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- March 31, 2026
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced it has resubmitted the Biologics License Application (BLA) for apitegromab for the treatment of children and adults with SMA to the U.S. Food and Drug Administration (FDA).
AI Summary
Scholar Rock announced it has resubmitted the Biologics License Application (BLA) to the U.S. Food and Drug Administration for apitegromab, a muscle-targeted therapy for children and adults with spinal muscular atrophy (SMA). Apitegromab is the first and only muscle-targeted therapy to show statistically significant and clinically meaningful improvements in motor function in a pivotal Phase 3 trial among patients already receiving an SMN-targeted therapy.
The resubmission was limited in scope and mainly included a standard safety update. Scholar Rock expects the FDA to accept the BLA within 30 days of resubmission and anticipates a review period of up to six months. A PDUFA action date is expected in late September 2026.
Separately, the European Medicines Agency review of the marketing application is progressing, with a decision anticipated in mid-2026. Scholar Rock emphasized the focused nature of the update as it awaits regulatory decisions.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- September 23, 2025
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the apitegromab Biologics License Application (BLA) for the treatment of patients with spinal muscular atrophy (SMA).
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- August 14, 2025
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that positive study results from the pivotal Phase 3 SAPPHIRE trial (NCT05156320) were published in the peer-reviewed journal The Lancet Neurology.
AI Summary
Scholar Rock announced that its pivotal Phase 3 SAPPHIRE trial results were published in The Lancet Neurology. The randomized, double-blind study (NCT05156320) enrolled 188 children and adults with spinal muscular atrophy (SMA) on existing SMN-targeted treatments. Over 52 weeks, participants received apitegromab or placebo to evaluate motor function.
Data showed a statistically significant, clinically meaningful improvement in the Hammersmith Functional Motor Scale Expanded (HFMSE) for those on apitegromab (mean difference 1.8 points, p=0.019). At 52 weeks, 30.4% of treated patients achieved ≥ 3-point gains versus 12.5% on placebo, and 19.6% achieved ≥ 4-point gains versus 6.3% with placebo.
The study found consistent benefits across age groups, types and timing of SMN-targeted treatment, and regions. Apitegromab was well tolerated with no new safety concerns, highlighting the value of a muscle-targeted approach alongside neuron-preserving therapies.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- June 18, 2025
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced positive results from the Phase 2 EMBRAZE proof-of-concept trial assessing apitegromab in combination with tirzepatide to preserve lean mass during tirzepatide-induced weight loss.
AI Summary
Scholar Rock announced encouraging results from its Phase 2 EMBRAZE trial, which studied the combination of apitegromab and tirzepatide to preserve lean mass during weight loss. In the trial, patients receiving both treatments for 24 weeks preserved an extra 4.2 pounds of lean mass—54.9% more than those using tirzepatide alone (p=0.001). This trial, designed for overweight or obese patients, showed that while the overall weight loss was similar in both groups, the combination therapy led to a higher quality of weight loss. Specifically, the treatment with apitegromab resulted in an 85% loss from fat mass and only 15% from lean mass, compared to 70% fat loss and 30% lean loss with tirzepatide alone. Moreover, apitegromab was generally well tolerated, with a safety profile that aligned with earlier clinical studies.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- March 25, 2025
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational muscle-targeted treatment that is being developed to provide clinically meaningful improvement in motor function for people living with spinal muscular atrophy (SMA) who are receiving an SMN-targeted treatment.
AI Summary
Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab. This investigational treatment is designed to help people with spinal muscular atrophy (SMA) who are already receiving SMN-targeted therapies. Apitegromab works by targeting muscles to potentially improve motor function, addressing the decline many SMA patients experience despite current treatments.
The FDA will review the application under a priority review process, with a target action date set for September 22, 2025. This designation suggests that, if approved, apitegromab could provide a significant advancement in treatment options for the SMA community. Scholar Rock is hopeful that this breakthrough muscle-targeted therapy will offer meaningful improvements for patients and pave the way for its future commercial launch in the United States and abroad.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- March 25, 2025
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that FDA to review BLA application under priority review, with a PDUFA date of September 22, 2025
AI Summary
Scholar Rock announced that the FDA has accepted its Biologics License Application (BLA) for apitegromab and will review it under priority review. This designation signifies that the FDA believes apitegromab could offer a significant improvement in the treatment and safety profile for patients suffering from spinal muscular atrophy (SMA). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2025. This review process highlights the potential of apitegromab as the first muscle-targeted therapy to improve motor function in SMA patients who continue to decline despite SMN-targeted treatments.
The priority review status acknowledges the drug’s promise and speeds up the evaluation process, emphasizing its importance as a transformative therapy in the SMA community. Scholar Rock is optimistic about this crucial milestone as it continues its efforts toward a future commercial launch.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- March 16, 2025
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that new efficacy and safety data from the Phase 3 pivotal SAPPHIRE trial (NCT05156320) will be presented in multiple clinical presentations at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Dallas, Texas.
AI Summary
Scholar Rock, a late-stage biopharmaceutical company focused on neuromuscular diseases, announced that new efficacy and safety data from its Phase 3 pivotal SAPPHIRE trial (NCT05156320) will be presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Dallas, Texas. The trial evaluates apitegromab, an investigational muscle-targeted therapy for spinal muscular atrophy (SMA) in patients who are already receiving SMN-targeted treatments. At the conference, multiple clinical presentations will highlight key data, including secondary endpoint analyses that demonstrate consistent improvements in motor function across various patient subgroups. This opportunity will allow researchers and clinicians to learn more about how apitegromab may provide an added benefit for individuals living with SMA, potentially improving daily activities and overall independence.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- February 21, 2025
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that it will present additional data from its Phase 3 SAPPHIRE clinical trial (NCT05156320) at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 16-19 in Dallas, Texas.
AI Summary
Scholar Rock, a late-stage biopharmaceutical company dedicated to neuromuscular disorders, announced plans to present additional data from its Phase 3 SAPPHIRE clinical trial at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. The SAPPHIRE trial evaluated apitegromab, an investigational therapy designed to improve motor function in patients with spinal muscular atrophy (SMA) who are already receiving SMN-targeted treatments. Dr. Thomas O. Crawford from Johns Hopkins University will deliver an oral presentation detailing the trial’s efficacy and safety findings on March 19, 2025. In addition, further insights will be shared through a poster presentation during the conference, which runs from March 16-19 in Dallas, Texas. This event offers an important platform for discussing emerging treatment approaches for SMA and enhancing our understanding of therapies that target neuromuscular diseases.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- January 29, 2025
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced the submission of a Biologics License Application to the U.S. Food and Drug Administration (FDA) for apitegromab, a muscle-targeted therapy designed and developed to provide clinically meaningful improvement in motor function for people living with SMA who are receiving SMN-targeted treatments.
AI Summary
Scholar Rock, a late-stage biopharmaceutical company, has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for apitegromab. This muscle-targeted therapy is designed to provide meaningful improvements in motor function for patients with spinal muscular atrophy (SMA) who are already receiving SMN-targeted treatments. The submission is based on encouraging results from the Phase 3 SAPPHIRE trial, which showed participants on apitegromab achieved a statistically significant improvement compared to those on placebo. The therapy aims to enhance quality of life for those with SMA by improving their motor skills, an important step in addressing the unmet needs of these patients.
Scholar Rock’s ongoing work with apitegromab highlights its commitment to developing innovative treatments, with future steps including further regulatory submissions and additional clinical studies to bring this promising therapy closer to patients in need.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- October 7, 2024
- Target Action Date:
- Q1 2025
- Estimated Target Date Range:
- January 1, 2025 - March 31, 2025
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced that Scholar Rock plans to submit a U.S. Biologics License Application and European Union marketing authorisation application in Q1 2025
AI Summary
Scholar Rock announced encouraging results from its Phase 3 SAPPHIRE trial testing apitegromab, a novel treatment for spinal muscular atrophy (SMA). The study showed that patients receiving apitegromab experienced a statistically significant and clinically meaningful improvement in motor function compared to those given placebo on top of standard care. Benefits were seen as early as 8 weeks and were maintained through 52 weeks, with improvements observed across multiple age groups.
Looking ahead, Scholar Rock plans to submit its U.S. Biologics License Application and European Union marketing authorisation application in the first quarter of 2025. This key submission is an important step toward making apitegromab available for patients with SMA, aiming to transform treatment options and support improved motor function for children and adults battling this challenging condition.
Read Announcement- Drug:
- Apitegromab
- Announced Date:
- October 7, 2024
- Indication:
- Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Announcement
Scholar Rock announced positive topline results from the Phase 3 SAPPHIRE clinical trial (NCT05156320) evaluating the efficacy and safety of apitegromab, an investigational muscle-targeted therapy, in patients with SMA.
AI Summary
Scholar Rock recently announced positive topline results from its Phase 3 SAPPHIRE trial, evaluating the efficacy and safety of apitegromab, an investigational muscle-targeted therapy for spinal muscular atrophy (SMA). The trial met its primary endpoint, showing statistically significant and clinically meaningful improvements in motor function, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE). Approximately 30.4% of patients receiving apitegromab achieved over a 3-point improvement compared to 12.5% for those on placebo. Notably, improvements were seen early at 8 weeks and maintained at 52 weeks, with benefits observed across patients aged 2 to 21. The study also confirmed a favorable safety profile consistent with previous findings. Based on these encouraging results, Scholar Rock plans to submit a U.S. Biologics License Application and an EU marketing authorization application in Q1 2025.
Read Announcement
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