Understanding What Drugs Are Used to Treat Muscle Atrophy

October 14, 2025 •

4 min read

Currently, no single therapeutic drug has been universally marketed and approved as a cure for muscle atrophy, which affects a significant portion of the population with various chronic diseases and age-related conditions. This article explores what drugs are used to treat muscle atrophy, detailing different classes of medications and their specific uses in conditions like muscular dystrophy, SMA, and sarcopenia.

Quick Summary

A diverse range of medications and experimental therapies address muscle atrophy by inhibiting protein degradation, promoting synthesis, or correcting genetic defects. Treatments are often specific to the underlying cause, and many promising agents are still under investigation in clinical trials.

Key Points

Myostatin Inhibitors and ActRII Antagonists: These compounds, like bimagrumab, block a key signaling pathway that inhibits muscle growth, leading to increased muscle mass in conditions like sarcopenia and muscular dystrophy.
Selective Androgen Receptor Modulators (SARMs): Investigational drugs like enobosarm promote muscle growth by selectively targeting androgen receptors in muscle and bone with fewer side effects than traditional steroids, though long-term safety is still under review.
Targeted Gene Therapies: For specific genetic diseases, such as SMA and DMD, FDA-approved medications exist that directly target the genetic defect, modifying gene expression or delivering a functional gene copy.
Corticosteroids: Drugs like prednisone and deflazacort are used to delay muscle degeneration in muscular dystrophies, but they carry significant long-term side effects.
Investigational Treatments: Other therapies in development include ghrelin mimetics to stimulate appetite and growth hormone, fast skeletal muscle troponin activators to increase muscle power, and HDAC inhibitors to address muscle inflammation and fibrosis.
Multimodal Management: Effective treatment for muscle atrophy typically involves a combination of medication, resistance exercise, and nutritional support, tailored to the specific cause and individual patient.
Lack of a Universal Cure: Despite ongoing research and promising therapies, there is currently no single, universally effective drug for all types of muscle atrophy, especially for non-genetic conditions like age-related muscle loss.

Current Pharmacological Approaches for Muscle Atrophy

Treating muscle atrophy is complex and often depends on the underlying cause, such as muscular dystrophy, spinal muscular atrophy (SMA), cancer-related cachexia, or age-related sarcopenia. While a cure remains elusive for many conditions, a variety of medications and investigational therapies are used to manage symptoms, slow progression, or promote muscle growth. These approaches target key biological pathways involved in muscle protein synthesis and degradation.

Myostatin Inhibitors and ActRII Antagonists

Myostatin is a protein that acts as a negative regulator of muscle growth. Blocking its activity, or the activin receptors (ActRII) it binds to, can lead to increased muscle mass.

Myostatin Inhibitors: These agents, such as monoclonal antibodies, directly neutralize the myostatin protein. Clinical trials have been conducted for various conditions, including muscular dystrophy and sarcopenia, with mixed results regarding functional improvement despite increases in muscle mass.
Activin Receptor Antagonists: Examples like bimagrumab (BYM338) target the ActRII receptors, blocking not only myostatin but also other ligands in the TGF-β superfamily that negatively impact muscle mass. Studies have shown promise in increasing lean body mass and improving physical function in specific populations.

Selective Androgen Receptor Modulators (SARMs)

SARMs are a class of nonsteroidal drugs that selectively target androgen receptors in muscle and bone tissues, promoting anabolic activity with fewer side effects than traditional anabolic steroids.

Enobosarm (Ostarine): This well-studied SARM has shown dose-dependent increases in lean body mass in elderly and cancer patients. While it increases muscle mass, results regarding improved physical function in clinical trials have been inconsistent.
Other SARMs: Compounds like LGD-4033 have also been studied, demonstrating increases in lean body mass. SARMs are still investigational, and their long-term safety and efficacy are not fully established.

Gene and Genetic-Targeting Therapies

For inherited conditions causing muscle atrophy, gene-based approaches offer targeted solutions to address the root cause of the disease. These therapies are often specific to the mutation or disease type.

Spinal Muscular Atrophy (SMA) Drugs: For SMA, which results from a defect in the SMN1 gene, several FDA-approved treatments are available.
- Nusinersen (Spinraza): An injection that modifies SMN2 gene splicing to increase functional SMN protein.
- Onasemnogene abeparvovec (Zolgensma): A gene therapy that delivers a new copy of the SMN1 gene.
- Risdiplam (Evrysdi): The first oral medication for SMA, it works as an SMN2 splicing modifier.
Duchenne Muscular Dystrophy (DMD) Drugs: Exon-skipping drugs are used for specific gene mutations in DMD.
- Eteplirsen, Golodirsen, Viltolarsen, and Casimersen: These antisense oligonucleotides cause the cell to "skip" over faulty parts of the dystrophin gene, allowing for the production of a shortened, but functional, dystrophin protein.
Histone Deacetylase (HDAC) Inhibitors: Givinostat (Duvyzat) is an HDAC inhibitor approved for DMD that works by mitigating inflammation and fibrosis, slowing disease progression.

Corticosteroids

Corticosteroids are powerful anti-inflammatory drugs that can help delay muscle degeneration and retain strength in some forms of muscular dystrophy.

Prednisone and Deflazacort (Emflaza): Often prescribed for Duchenne and Becker muscular dystrophies, they can slow the progression of muscle weakness.
Vamorolone (Agamree): A newer synthetic steroid approved for DMD that may have fewer side effects than traditional corticosteroids.

However, long-term use is associated with significant side effects, including bone loss and weight gain, requiring close medical supervision.

Comparison of Muscle Atrophy Drug Classes


Drug Class	Examples	Mechanism of Action	Target Condition(s)	Key Considerations
Myostatin/ActRII Inhibitors	Bimagrumab, Domagrozumab	Blocks myostatin or its receptor (ActRII) to promote muscle growth.	Sarcopenia, Muscular Dystrophy, Cachexia	Clinical efficacy for function can be inconsistent.
SARMs	Enobosarm (Ostarine), LGD-4033	Selectively stimulates androgen receptors in muscle and bone.	Cancer Cachexia, Sarcopenia, Hypogonadism	Investigational; potential for cardiovascular risks and liver injury.
Gene/Genetic-Targeting Therapies	Nusinersen, Zolgensma, Eteplirsen, Givinostat	Corrects genetic defects or modifies gene expression to produce functional protein.	Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD)	Highly specific to genetic mutation; not universally applicable.
Corticosteroids	Prednisone, Deflazacort, Vamorolone	Reduces inflammation and slows muscle degeneration.	Muscular Dystrophies (DMD, Becker MD)	Associated with significant long-term side effects.
Ghrelin Mimetics	Anamorelin	Stimulates appetite and growth hormone release.	Cancer Cachexia	Can cause insulin resistance and diabetes risk.

Conclusion

While there is no single cure for muscle atrophy, pharmacological treatments offer significant benefits, particularly for genetically-driven diseases like SMA and DMD. Therapies targeting myostatin, ActRII, and androgen receptors show promise for broader applications in sarcopenia and cachexia, although many remain investigational. The optimal treatment plan is often multimodal, combining medication with exercise and nutritional support, and tailored to the individual's specific condition and overall health. Continued research is essential to develop safer and more effective therapeutic options that can be widely applied to combat muscle wasting.

For more information on the research and development of pharmacotherapies for muscle wasting, consult scientific databases such as PubMed Central.

Frequently Asked Questions

SARMs are designed to selectively target androgen receptors in muscle and bone, aiming to provide anabolic benefits with fewer of the widespread side effects associated with anabolic steroids, which can affect multiple organs.

Gene therapies for SMA, such as Zolgensma, deliver a new, functional copy of the SMN1 gene to the patient's cells via a viral vector, allowing the body to produce the necessary protein for muscle control.

Myostatin inhibitors have shown promise in clinical trials by increasing muscle mass, but many investigational candidates have not yet demonstrated consistent functional improvement and are not widely approved for muscle atrophy.

Long-term corticosteroid use, particularly with drugs like prednisone and deflazacort, can lead to serious side effects including osteoporosis (bone loss), weight gain, and increased blood pressure, which require careful monitoring.

While exercise and nutrition are primary interventions for sarcopenia, investigational drugs like myostatin inhibitors and SARMs are being studied for their potential to increase muscle mass and function in older adults.

Exon-skipping drugs, such as eteplirsen and viltolarsen, are specific to certain gene mutations in DMD. They promote the skipping of a particular exon during protein synthesis, allowing the production of a shortened but partially functional dystrophin protein.

Yes, risdiplam (Evrysdi) is an oral medication that works as an SMN2 splicing modifier and is approved for adults and children with types I, II, and III SMA.