Facioscapulohumeral muscular dystrophy (FSHD) is a rare genetic muscle disorder characterized by progressive muscle weakness, primarily affecting the face, shoulder blades, and upper arms. According to Matthew K. Preston et al. (2025), FSHD1 is inherited in an autosomal dominant manner, with approximately 70%-90% of patients having a parent with the condition. On the other hand, 10%-30% result from de novo mutations. According to the facioscapulohumeral muscular dystrophy pipeline analysis by Expert Market Research, the rising focus on genetic therapies and RNA-targeted therapeutics is expected to drive pipeline growth in the coming years.
Major companies involved in the facioscapulohumeral muscular dystrophy pipeline analysis include Avidity Biosciences, Inc., Epicrispr Biotechnologies, Inc., and others.
Leading drugs currently in the pipeline include AOC-1020, EPI-321, and others.
The facioscapulohumeral muscular dystrophy drug pipeline is advancing with increased gene therapy research, novel molecular targets, and rising clinical trial activity, supporting significant growth in therapeutic development.
The Facioscapulohumeral Muscular Dystrophy Pipeline Analysis Report by Expert Market Research gives comprehensive insights into facioscapulohumeral muscular dystrophy therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for facioscapulohumeral muscular dystrophy. The facioscapulohumeral muscular dystrophy report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The facioscapulohumeral muscular dystrophy pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with facioscapulohumeral muscular dystrophy treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to facioscapulohumeral muscular dystrophy.
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Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder characterized by progressive weakening and wasting of muscles, especially in the face, shoulders, and upper arms. It occurs due to the abnormal activation of the DUX4 gene, which produces toxic proteins that damage muscle cells over time.
Facioscapulohumeral muscular dystrophy treatment focuses on symptom management through physical therapy, orthopedic interventions, and assistive devices. Research into gene-targeted therapies is ongoing, offering potential future options. In July 2024, Avidity Biosciences’ del-brax (delpacibart braxlosiran) showed promising results in a Phase I/II trial. The drug significantly reduced DUX4 expression and related protein levels, improved muscle strength, and decreased creatine kinase, suggesting strong therapeutic potential for FSHD.
As stated by Andrew LaPelusa et al., 2024, muscular dystrophies affect an estimated 1 in 5,000 to 10,000 individuals globally, with prevalence varying by subtype. According to Matthew K. Preston, facioscapulohumeral muscular dystrophy (FSHD) affects approximately 1 in 20,000 individuals worldwide. FSHD1 is inherited in an autosomal dominant manner, with 70%–90% of cases showing a familial history and 10%–30% resulting from de novo mutations. Each child of an affected parent has a 50% chance of inheriting the pathogenic variant. FSHD2 involves complex inheritance.
This section of the report covers the analysis of facioscapulohumeral muscular dystrophy drug candidates based on several segmentations, including:
By Phase
By Drug Class
By Route of Administration
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II covers a major share of the total facioscapulohumeral muscular dystrophy clinical trials with 55%, reflecting robust clinical momentum. Phase I accounts for 36%, while phase III holds 9%, signaling early yet meaningful progress. This dynamic pipeline underscores strong research efforts, with each phase paving the way for innovative therapies that are poised to positively transform the treatment landscape.
The drug molecule categories covered under the facioscapulohumeral muscular dystrophy pipeline analysis include small molecules, oligonucleotides, gene therapies, monoclonal antibodies, and peptides. The facioscapulohumeral muscular dystrophy report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for facioscapulohumeral muscular dystrophy. Small molecule drug therapies are under active investigation for the treatment of facioscapulohumeral muscular dystrophy. For instance, losmapimod, a selective p38α/β mitogen-activated protein kinase inhibitor, was evaluated in Fulcrum Therapeutics' Phase 3 REACH trial. The trial provided key insights into therapeutic development strategies for this rare neuromuscular disorder.
The EMR report for the facioscapulohumeral muscular dystrophy pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed facioscapulohumeral muscular dystrophy therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in facioscapulohumeral muscular dystrophy clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for facioscapulohumeral muscular dystrophy. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of facioscapulohumeral muscular dystrophy drug candidates.
Del-brax (AOC 1020), developed by Avidity Biosciences, Inc., is currently under evaluation in a Phase 3 clinical trial to assess its efficacy and safety in patients with facioscapulohumeral muscular dystrophy (FSHD). This investigational therapy aims to reduce DUX4 mRNA and protein expression, the root cause of FSHD. Del-brax combines a monoclonal antibody targeting TfR1 with siRNA, delivering treatment directly to muscle tissue.
EPI-321, developed by Epicrispr Biotechnologies, Inc., is an investigational gene therapy being evaluated in an open-label Phase 1/2 trial for facioscapulohumeral muscular dystrophy (FSHD). The study is assessing the safety, tolerability, and biological activity. EPI-321 delivers a non-cutting CRISPR-based epigenetic editor—dCasONYX fused with methylation enzymes, via the AAVrh74 vector. It targets the D4Z4 region near the DUX4 gene to restore methylation, repress the expression of the toxic DUX4 protein, and protect muscle function.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Facioscapulohumeral Muscular Dystrophy Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for facioscapulohumeral muscular dystrophy. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into facioscapulohumeral muscular dystrophy collaborations, regulatory environments, and potential growth opportunities.
Facioscapulohumeral Muscular Dystrophy Epidemiology Forecast
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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