Drugs / Apitegromab
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | arthrogryposis spinal muscular atrophy | 2026-03-31 | “has resubmitted the Biologics License Application (BLA) for apitegromab for the treatment of children and adults with SMA” scholarrock.com ↗ |
| Rejected (CRL) | US (FDA) | — | arthrogryposis spinal muscular atrophy | 2025-09-23 | “the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the apitegromab Biologics License Application (BLA)” scholarrock.com ↗ “In September 2025 , Scholar Rock received a Complete Response Letter (CRL) from the FDA for apitegromab” scholarrock.com ↗ |
| Filed | EU (EMA) | — | arthrogryposis spinal muscular atrophy | 2025-03-25 | “submitted and received validation for its Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for apitegromab for the treatment of SMA” scholarrock.com ↗ “European Medicines Agency (EMA) validated Marketing Authorisation Application (MAA) for apitegromab for patients with SMA” scholarrock.com ↗ |
| Filed | US (FDA) | — | arthrogryposis spinal muscular atrophy | 2025-03-25 | “FDA has accepted its Biologics License Application (BLA) for apitegromab” scholarrock.com ↗ |
| Filed | US (FDA) | — | arthrogryposis spinal muscular atrophy | 2025-01-29 | “The Company submitted a Biologics License Application (BLA) application for apitegromab to the FDA, which has been accepted under priority review” scholarrock.com ↗ “today announced the submission of a Biologics License Application to the U.S. Food and Drug Administration (FDA) for apitegromab” scholarrock.com ↗ “The FDA has accepted the Biologics License Application (BLA) for apitegromab under priority review” scholarrock.com ↗ “Scholar Rock’s apitegromab BLA submission in March 2026 included two fill-finish facilities” scholarrock.com ↗ “Scholar Rock submitted a BLA to the FDA in January 2025” scholarrock.com ↗ “the U.S. FDA accepted its BLA for apitegromab” scholarrock.com ↗ |
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 24 trials · 1 met primary | ||||||
| Phase 2 | NCT07435129 | Jul 2026 → Jun 2028 expected | facioscapulohumeral muscular dystrophy | Scholar Rock, Inc. | Recruiting | No outcome recorded |
| Phase 2 | NCT07047144 | Sep 2025 → Nov 2028 expected | spinal muscular atrophy, type II, spinal muscular atrophy, type III | Scholar Rock, Inc. | Recruiting | No outcome recorded |
| Phase 2 | NCT06445075 | May 2024 → Mar 2025 | — | Scholar Rock, Inc. | Completed | Met primary |
| Phase 2 | NCT03921528 | Apr 2019 → Jan 2021 | muscular atrophy, spinal muscular atrophy, type II, spinal muscular atrophy, type III | Scholar Rock, Inc. | Completed | Mixed |
| Phase 32 trials · 1 met primary | ||||||
| Phase 3 | NCT05626855 | Apr 2023 → Nov 2026 expected | muscular atrophy, spinal muscular atrophy, type II, spinal muscular atrophy, type III | Scholar Rock, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05156320 | Apr 2022 → Dec 2024 | muscular atrophy, spinal muscular atrophy, type II, spinal muscular atrophy, type III | Scholar Rock, Inc. | Completed | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT06877689 | — | spinal muscular atrophy | Scholar Rock, Inc. | No longer available | No outcome recorded |
News releases announcing trial results or a regulatory action · 41
| Date | Issuer | Release |
|---|---|---|
| 2026-10-08 | Scholar Rock, Inc. | Regulatory Scholar Rock Resubmits Apitegromab Marketing Authorisation Application (MAA) to European Medicines Agency (EMA) for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA) scholarrock.com ↗
Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology, today announced it has resubmitted the Marketing Authorisation Application (MAA) for apitegromab for the treatment of children and adults with spinal muscular atrophy (SMA) to the European Medicines Agency (EMA). |
| 2026-09-11 | Scholar Rock, Inc. | Regulatory Scholar Rock Announces FDA Approval of ISEMBYLD™ (apitegromab-mstn), the First and Only Muscle-Targeted Treatment for Children and Adults with Spinal Muscular Atrophy (SMA) scholarrock.com ↗
today 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. |
| 2026-09-02 | Scholar Rock, Inc. | Regulatory Scholar Rock Receives FDA Fast Track Designation for Apitegromab Facioscapulohumeral Muscular Dystrophy (FSHD) Program as Participant Dosing Commences in Phase 2 FORGE Trial scholarrock.com ↗
today announced that the FDA has granted Fast Track and Orphan Drug designations to apitegromab for the treatment of people living with FSHD. |
| 2026-08-21 | Scholar Rock, Inc. | Regulatory Scholar Rock Provides Update on Global Apitegromab Regulatory Progress Across U.S., Europe, and Japan scholarrock.com ↗
Japan Pharmaceuticals and Medical Devices Agency (PMDA) has agreed no additional clinical studies required for apitegromab regulatory submission; Scholar Rock now on track to submit apitegromab Japanese New Drug Application (JNDA) by YE 2026 |
| 2026-08-07 | Scholar Rock, Inc. | Regulatory Scholar Rock Announces FDA Review of Apitegromab Biologics License Application (BLA) for Spinal Muscular Atrophy (SMA) to Progress with Second Fill-Finish Facility; Approval Decision Anticipated by September 30, 2026 Action Date scholarrock.com ↗ |
| 2026-03-31 | Scholar Rock, Inc. | Regulatory Scholar Rock Resubmits Biologics License Application (BLA) to FDA for Apitegromab for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA) scholarrock.com ↗
Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today 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). |
| 2025-09-23 | Scholar Rock, Inc. | Regulatory FDA Issues Complete Response Letter (CRL) for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA) Solely Related to Observations Identified at Catalent Indiana LLC Fill-Finish Facility scholarrock.com ↗
Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today 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). |
| 2025-08-14 | Scholar Rock, Inc. | Results Pivotal SAPPHIRE Trial Data Published in The Lancet Neurology: Apitegromab Demonstrated Significant Motor Function Gains for Children and Adults with SMA on SMN-Targeted Treatment scholarrock.com ↗
As reported, the Phase 3 SAPPHIRE trial met its primary endpoint, achieving a statistically significant and clinically meaningful improvement in motor function (measured by HFMSE) for patients receiving apitegromab versus placebo, despite all participants receiving chronic, ongoing SMN-targeted treatment. |
| 2025-06-23 | Scholar Rock, Inc. | Results Scholar Rock to Present Comprehensive Update at 2025 Annual Cure SMA Research and Clinical Care Meeting, Including Positive Results from Pivotal Phase 3 SAPPHIRE Trial scholarrock.com ↗
Oral presentation on the positive Phase 3 SAPPHIRE trial of apitegromab for people living with spinal muscular atrophy (SMA), which demonstrated statistically significant improvement and clinically meaningful benefit as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) |
| 2025-06-18 | Scholar Rock, Inc. | Results Scholar Rock Reports Positive Phase 2 EMBRAZE Trial Results Demonstrating Statistically Significant Preservation of Lean Mass with Apitegromab During Tirzepatide-Induced Weight Loss scholarrock.com ↗
Apitegromab therapy (10 mg/kg) with tirzepatide preserved an additional 4.2 pounds (1.9 kilograms) or 54.9% (p=0.001) of lean mass compared to tirzepatide alone, leading to higher quality weight loss. |
| 2025-03-25 | Scholar Rock, Inc. | Regulatory FDA Grants Priority Review for Biologics License Application (BLA) and EMA Accepts Marketing Authorisation Application (MAA) for Apitegromab as a Treatment for Spinal Muscular Atrophy scholarrock.com ↗ |
| 2025-03-16 | Scholar Rock, Inc. | Results Scholar Rock Presents New Phase 3 SAPPHIRE Data at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference scholarrock.com ↗
New analysis performed in the pooled population (ages 2-21) showed clinically meaningful and consistent improvement in HFMSE across pre-specified subgroups (type of SMN-targeted therapy, age at SMN-targeted therapy initiation) and geographic region. |
| 2025-01-29 | Scholar Rock, Inc. | Regulatory Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA) scholarrock.com ↗ |
| 2024-10-07 | Scholar Rock, Inc. | Results Scholar Rock Reports Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA) scholarrock.com ↗
30.4% of patients receiving apitegromab had >3 point improvement in HFMSE versus 12.5% of patients on placebo |
| 2024-08-08 | Scholar Rock, Inc. | Results Scholar Rock Reports Second Quarter 2024 Financial Results and Highlights Business Progress scholarrock.com ↗
Reported that long-term apitegromab data continued to show substantial and sustained motor function improvements over 48 months 1 . |
| 2024-03-19 | Scholar Rock, Inc. | Regulatory Scholar Rock Reports Full Year 2023 Financial Results and Highlights Business Progress scholarrock.com ↗
Announced FDA clearance of IND application to initiate Phase 2 proof-of-concept trial with apitegromab to treat obesity; expected to commence in mid-2024 |
| 2024-01-23 | Scholar Rock, Inc. | Regulatory Scholar Rock Announces FDA Clearance of IND Application to Initiate Phase 2 Proof-of-Concept Trial with Apitegromab to Treat Obesity scholarrock.com ↗ |
| 2023-06-30 | Scholar Rock, Inc. | Results New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy scholarrock.com ↗
Detailed results were presented today by Thomas Crawford , M.D., of Johns Hopkins Medicine , and the lead principal investigator of the TOPAZ trial, during two podium presentations at the Cure SMA Research & Clinical Care Meeting in Orlando, Florida . |
| 2023-03-01 | Scholar Rock, Inc. | Results Scholar Rock to Present 24-Month Data from the Phase 2 TOPAZ trial at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference scholarrock.com ↗
This analysis of our 24-month data continues to provide insights into apitegromab’s potential effects on motor function and quality of life measures over time. |
| 2022-10-22 | Scholar Rock, Inc. | Results New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA scholarrock.com ↗
The tertiary endpoint data from these measures show trends of continuous improvement over 24 months. |
| 2022-06-17 | Scholar Rock, Inc. | Results Positive Phase 2 Topaz Trial Extension Data Demonstrate Sizable and Sustained Motor Function Improvement at 24 Months with Apitegromab for Non-Ambulatory Patients with Types 2 and 3 Spinal Muscular Atrophy (SMA) scholarrock.com ↗
Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24-months of treatment, which support sustained and continued improvement with apitegromab for non-ambulatory patients with Types 2 and 3 SMA receiving an SMN therapy. |
| 2021-10-03 | Scholar Rock, Inc. | Results Scholar Rock to Present Apitegromab TOPAZ Phase 2 Pharmacologic Data at the 2021 World Congress of Neurology scholarrock.com ↗
This poster will showcase positive correlations between the magnitude of target engagement (as measured by serum levels of latent myostatin) and motor function improvements following treatment with apitegromab. |
| 2021-09-23 | Scholar Rock, Inc. | Results Scholar Rock Presents Additional Data Analyses from the Apitegromab TOPAZ Phase 2 Trial at the World Muscle Society 2021 Virtual Congress scholarrock.com ↗
An additional exploratory analysis evaluating time to achieving various thresholds of improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) scores further support the dose response in clinical efficacy. |
| 2021-06-18 | Scholar Rock, Inc. | Results Scholar Rock to Present Apitegromab TOPAZ Phase 2 Trial Results Highlighting Pharmacokinetic (PK) and Pharmacodynamic (PD) Data at the 2021 European Academy of Neurology Congress scholarrock.com ↗
In the TOPAZ trial, apitegromab showed therapeutic potential to further improve motor function in patients with non-ambulatory Type 2 and 3 SMA who were already receiving chronic maintenance therapy with a background SMN upregulator (nusinersen). |
| 2021-06-11 | Scholar Rock, Inc. | Results Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 Spinal Muscular Atrophy (SMA) at the 2021 Virtual SMA Research & Clinical Care Meeting scholarrock.com ↗
In the TOPAZ trial, treatment with apitegromab in conjunction with nusinersen in patients with Type 2 and 3 SMA led to meaningful motor function improvements of up to 20 points as measured by HFMSE. |
| 2021-05-24 | Scholar Rock, Inc. | Regulatory Scholar Rock Receives Fast Track Designation from the U.S. FDA for Apitegromab for the Treatment of Patients with Spinal Muscular Atrophy scholarrock.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for apitegromab, a selective inhibitor of myostatin activation, for the treatment of patients with Spinal Muscular Atrophy (SMA). |
| 2021-05-11 | Scholar Rock, Inc. | Results Scholar Rock Announces Publication of Phase 1 Clinical Trial Data Evaluating Apitegromab in Healthy Volunteers in the Journal Advances in Therapy scholarrock.com ↗
Single- and multiple-ascending doses of apitegromab were safe and well-tolerated at IV doses up to 30 mg/kg. |
| 2021-04-06 | Scholar Rock, Inc. | Results Scholar Rock Announces Positive 12-Month Top-Line Results From the TOPAZ Phase 2 Clinical Trial Evaluating Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy (SMA) scholarrock.com ↗
today announced positive top-line data from the TOPAZ Phase 2 clinical trial evaluating apitegromab (SRK-015) in patients with Type 2 and Type 3 spinal muscular atrophy (SMA). |
| 2020-10-27 | Scholar Rock, Inc. | Results Scholar Rock Announces Positive Proof-of-Concept Data from TOPAZ Phase 2 Trial Interim Analysis of SRK-015 in Patients with Type 2 and Type 3 Spinal Muscular Atrophy scholarrock.com ↗
Treatment with SRK-015 led to improvements in Hammersmith scale scores (primary efficacy endpoint) in all three cohorts of patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA). |
| 2020-08-13 | Scholar Rock, Inc. | Regulatory Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of Spinal Muscular Atrophy scholarrock.com ↗
Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA), a progressive, rare genetic disease that leads to motor function impairments. |
| 2020-02-06 | Scholar Rock, Inc. | Results Scholar Rock Presents Data for SRK-015 at the SMA Europe 2nd International Scientific Congress scholarrock.com ↗
Preliminary demographic and baseline characteristic data are in-line with published data with nusinersen and ensure appropriate inclusion of patients with Type 2 and Type 3 SMA. |
| 2019-11-19 | Scholar Rock, Inc. | Results Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy scholarrock.com ↗
SRK-015 demonstrated robust and dose-dependent target engagement |
| 2019-10-02 | Scholar Rock, Inc. | Results Scholar Rock to Present Preclinical and Phase 1 Clinical Data on SRK-015 at the World Muscle Society Congress scholarrock.com ↗
Preclinical and Phase 1 data support the evaluation of SRK-015 in ongoing Phase 2 trial for the treatment of patients with Type 2 and Type 3 spinal muscular trophy |
| 2019-06-03 | Scholar Rock, Inc. | Results Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers scholarrock.com ↗
Consistent with previously announced interim findings, the final results showed robust and sustained target engagement and no apparent safety signals were observed across all tested doses. |
| 2019-02-26 | Scholar Rock, Inc. | Results Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans scholarrock.com ↗
SRK-015, a highly specific inhibitor of myostatin activation, was observed to be well tolerated with no apparent safety signals. |
| 2018-12-03 | Scholar Rock, Inc. | Regulatory Scholar Rock Announces Positive Opinion by the European Medicines Agency on Orphan Drug Designation for SRK-015 for the Treatment of Spinal Muscular Atrophy scholarrock.com ↗
“We are delighted with the EMA COMP’s adoption of a positive opinion for SRK-015 orphan drug designation, which represents another important milestone for this clinical program,” said Nagesh Mahanthappa, PhD, President and CEO of Scholar Rock. |
| 2018-11-27 | Scholar Rock, Inc. | Results Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of Spinal Muscular Atrophy scholarrock.com ↗
The publication details preclinical studies demonstrating that a highly specific inhibitor of myostatin activation effectively increased muscle mass and function in mouse models of Spinal Muscular Atrophy (SMA). |
| 2018-03-29 | Scholar Rock, Inc. | Regulatory Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy scholarrock.com ↗ |
| 2018-02-02 | Scholar Rock, Inc. | Results Scholar Rock Announces Publication of Comprehensive Pharmacological Study of SRK-015, a Novel Inhibitor of Myostatin Activation, Supporting the Treatment of Muscle Atrophy Disorders scholarrock.com ↗
The preclinical research published today by Scholar Rock researchers demonstrates that inhibiting the activation of the latent precursor form of myostatin using monoclonal antibodies offers a potentially superior approach to address this important therapeutic target. |
| 2017-07-10 | Scholar Rock, Inc. | Results Scholar Rock and the Miami Project to Cure Paralysis Present Preclinical Data Demonstrating Therapeutic Potential of SRK-015 in Spinal Cord Injury at 35th Annual National Neurotrauma Symposium scholarrock.com ↗
More specifically, the antibody decreased muscle atrophy, reduced fat infiltration into muscle, and improved muscle function. |
| 2017-06-28 | Scholar Rock, Inc. | Results Scholar Rock to Develop SRK-015 to Improve Muscle Function in Patients with Spinal Muscular Atrophy scholarrock.com ↗
Treatment with SRK-015 resulted in significantly increased muscle strength in a genetic model of SMA |
All press releases naming this drug 59 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Apitegromab | “Apitegromab, an investigational, fully human monoclonal antibody, inhibits activation of myostatin (a negative regulator of skeletal muscle growth), thereby preserving muscle mass.” PMID 38330285 ↗ Feb 2024 |
| Known as | SRK-015 | “Apitegromab (SRK-015) is an investigational, fully human immunoglobulin G4 monoclonal antibody that specifically binds to human proforms (i.e., inactive precursor forms) of...” NCT05626855 ↗ |
| Action | Inhibit | “Growth/differentiation factor 8 inhibitor” CHEMBL4594326 ↗1“inhibits activation of myostatin” PMID 38330285 ↗ Feb 2024 |
| Modality | Monoclonal antibody | “fully human anti-proMyostatin monoclonal antibody (mAb)” NCT03921528 ↗ |
| Route | Intravenous | “administered every 4 weeks by intravenous infusion” NCT03921528 ↗ |
| Target | MSTN | “Growth/differentiation factor 8 inhibitor” CHEMBL4594326 ↗ |