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Scholar Rock's Myostatin Inhibitor Potential
18 Aug
Summary
- FDA decision expected September 30 for SMA treatment.
- Apitegromab targets latent myostatin, avoiding side effects.
- This could be the first muscle-targeted SMA therapy.
Scholar Rock anticipates an FDA decision by September 30 for apitegromab, an investigational treatment for spinal muscular atrophy (SMA). The company resubmitted its biologics license application, supported by an alternate manufacturing facility.
Apitegromab has the potential to be the first therapy approved to target muscle directly in SMA patients. This drug is a myostatin inhibitor, a class of drugs that has faced challenges in development due to potential side effects.
Scholar Rock's selective approach targets the inactive form of myostatin, a protein that regulates muscle growth. Previous attempts to inhibit myostatin often targeted the active protein or its receptor, leading to unwanted effects on related proteins. The company's antibody is designed to selectively inhibit latent myostatin, aiming to avoid these off-target issues.
The drug began healthy-volunteer testing in 2018 and moved into SMA development in 2019. SMA was chosen as the target disease because muscle is its primary affected organ, whereas existing treatments focus on survival motor neuron protein production and motor neuron health.
