DRUGphase3antibody
apitegromab
Brand names: SRK-015, apitegromab
Mechanism
anti-myostatin (GDF8) monoclonal antibody — muscle-axis SMA therapy. BLA accepted FDA 2026-05-08, PDUFA 2026-09-30.
Related claims (26)
| Type | Predicate | Conf | Source |
|---|---|---|---|
| motor function | Participants in the apitegromab treatment groups (combined 20 mg/kg and 10 mg/kg dose) achieved statistically significant improvements in motor function compared with placebo (clinical trial result). | 65% | 40818473 |
| motor function | Apitegromab led to improved motor function in participants with later-onset types 2 and 3 SMA (clinical trial result). | 59% | 38330285 |
| motor function | Treatment with apitegromab was associated with an improved motor function in patients with Type 2 or 3 spinal muscular atrophy (SMA) (clinical trial result). | 59% | 39105058 |
| motor function | Administration of apitegromab leads to improvements in motor function in subjects with spinal muscular atrophy. | 59% | US20250326827A1 |
| motor function | Apitegromab is associated with improvements in motor function in subjects with spinal muscular atrophy (clinical trial result) | 56% | EP4518970A2 |
| motor function | Administration of apitegromab leads to improved motor function in subjects with spinal muscular atrophy. | 56% | JP2025514978A |
| safety | There were no new safety findings with apitegromab treatment at 36 months (clinical trial result). | 53% | 39105058 |
| drug target | Apitegromab is a selective inhibitor of proforms of myostatin that does not exhibit toxicities observed with other myostatin pathway inhibitors. | 53% | 34255983 |
| drug efficacy | The benefit of apitegromab treatment observed at 12 months was sustained at 36 months (clinical trial result). | 53% | 39105058 |
| drug target | Apitegromab inhibits activation of myostatin (a negative regulator of skeletal muscle growth), thereby preserving muscle mass. | 53% | 38330285 |
| drug efficacy | Apitegromab, a myostatin inhibitor, is being evaluated in a trial in SMA patients, following positive results from preclinical studies. | 53% | 35727341 |
| drug efficacy | A phase 2 trial demonstrated the potential clinical benefit of apitegromab by improving or stabilizing motor function in patients with Type 2 and Type 3 SMA. | 53% | 36329412 |
| safety | Treatment with apitegromab was associated with a favorable safety profile (clinical trial result). | 53% | 39105058 |
| drug efficacy | Apitegromab is for use in treating spinal muscular atrophy (SMA) in a human subject (clinical trial result). | 53% | JPWO2022093724A5 |
| safety | The incidence and severity of adverse events were similar between apitegromab and placebo, and consistent with spinal muscular atrophy and background spinal muscular atrophy therapy (clinical trial result). | 53% | 40818473 |
| drug efficacy | Apitegromab is for use in the treatment of spinal muscular atrophy (SMA) in a human subject. | 50% | EP4232151B1 |
| drug efficacy | Apitegromab leads to improvements in motor function and/or quality of life in subjects with spinal muscular atrophy (clinical trial result) | 50% | EP4518970A2 |
| other | Administration of apitegromab leads to improvements in quality of life in subjects with spinal muscular atrophy. | 49% | WO2023215384A2 |
| safety | Apitegromab was safe and well tolerated, on the basis of the adverse event (AE) profile with no clinically meaningful changes in baseline vital signs, electrocardiograms, or clinical laboratory parameters and no anti-drug antibody formation... | 48% | 33963971 |
| drug target | Apitegromab is a selective inhibitor of proforms of myostatin. | 47% | 34255983 |
| drug target | Apitegromab selectively inhibits myostatin activation (clinical trial result). | 47% | 40818473 |
| other | Administration of apitegromab leads to improved quality of life in subjects with spinal muscular atrophy. | 46% | JP2025514978A |
| drug efficacy | These data support the conduct of ongoing clinical studies of apitegromab in adult and pediatric patients with spinal muscular atrophy (SMA). | 45% | 34255983 |
| drug target | Apitegromab is under development to improve motor function in patients with spinal muscular atrophy. | 45% | 33963971 |
| drug target | Single and multiple apitegromab doses resulted in dose-dependent and sustained increases in serum latent myostatin. | 45% | 33963971 |
| drug efficacy | Data support the conduct of ongoing clinical studies of apitegromab in adult and pediatric patients with spinal muscular atrophy (SMA). | 45% | 34255983 |
Off-Target Findings (0)
No Boltz-2 / Chai-1 off-target panel claims recorded for this drug.