Facioscapulohumeral Muscular Dystrophy (FSHD) Market

Market Overview:

The 7 major facioscapulohumeral muscular dystrophy markets reached a value of USD 32.2 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 61.4 Million by 2035, exhibiting a growth rate (CAGR) of 6.12% during 2025-2035.

The facioscapulohumeral muscular dystrophy market has been comprehensively analyzed in IMARC's new report titled "Facioscapulohumeral Muscular Dystrophy (FSHD) Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". FSHD is an inherited neuromuscular condition that is diagnosed by progressive weakening and atrophy of the muscles, mainly facial (facio), shoulder (scapulo), and arm (humeral) muscles. The condition tends to occur asymmetrically, either in adolescence or early adulthood, though its impact and rate of progression are different in each. Although FSHD is not fatal, it has a marked effect on mobility, daily life, and well-being. Symptoms are varied and include impairment of eye closure, winging of the scapulae, weakness in the upper limb, foot drop, and limb involvement in later stages. Additional symptoms include hearing loss and abnormalities in the retina. Diagnosis can be made clinically, with confirmation by electromyography (EMG), muscle biopsy, and genetic testing. The D4Z4 repeat contraction on 4q35 is a confirmed biomarker. Advances in genetic testing, biomarker research, and personalized medicine are enhancing early diagnosis and disease management, opening up opportunities for market growth.

The Facioscapulohumeral Muscular Dystrophy (FSHD) market is mainly fueled by the increasing incidence of genetic neuromuscular diseases, as well as improvements in precision medicine and gene-targeted therapies. FSHD is due to genetic mutations resulting in the aberrant expression of the DUX4 protein, which causes progressive muscle degeneration. This has prompted widespread research into disease-modifying therapies, with a number of pipeline drugs targeting epigenetic modulation, gene silencing, and DUX4 inhibition. Firms are now investing in RNA-based therapies and CRISPR gene-editing technologies to establish targeted methods addressing the cause of FSHD. Moreover, physiotherapy, rehabilitation regimes, supportive equipment, and symptomatic treatments are becoming more prominent as adjunctive care to enhance patients' mobility and well-being. Increasing utilization of anti-inflammatory drugs and myostatin inhibitors to maintain muscle function is also broadening the therapeutic pipeline. Government grants, patient advocacy programs, and rising clinical trial enrollments are driving the growth of new therapies. With the advancement of biopharmaceutical innovation, the market is anticipated to see increased treatment availability, which will spur growth throughout the forecast period.

IMARC Group's new report provides an exhaustive analysis of the facioscapulohumeral muscular dystrophy market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, Europe has the largest patient pool for facioscapulohumeral muscular dystrophy and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the facioscapulohumeral muscular dystrophy market in any manner.

Recent Developments:

• In November 2024, Novartis bought Kate Therapeutics for as much as US$1.1 billion to further develop gene therapies for neuromuscular diseases, such as FSHD. Kate Therapeutics is working on a candidate that delivers microRNA against DUX4 to skeletal muscles using their DELIVER platform for targeted delivery.
• In May 2024, Sanofi signed a collaboration and licensing deal with Fulcrum Therapeutics worth up to US$1 billion to develop and market losmapimod for FSHD outside the U.S. Although losmapimod was later discontinued, this deal highlights the pharmaceutical sector's interest in FSHD treatments.

Key Highlights:

• FSHD is among the more frequently occurring muscular dystrophies, affecting roughly 4 to 10 individuals per 100,000 people worldwide.
• Symptoms generally appear during adolescence or early adulthood, with muscle weakness starting in the face and shoulders. The progression varies, with some individuals experiencing only mild symptoms, while others face severe muscle deterioration over time.
• The disorder follows an autosomal dominant inheritance pattern, meaning a mutation in one copy of the gene can cause the disease. However, approximately 30% of cases arise from new mutations without a family history.
• Most individuals with FSHD have a normal lifespan. However, the condition can lead to mobility challenges and significantly impact daily activities.
• In addition to muscle weakness, the disorder may lead to hearing impairment, vision issues, and respiratory difficulties in advanced cases.

Drugs:

Delpacibart braxlosiran (del-brax) is an experimental Antibody Oligonucleotide Conjugate (AOC) treatment developed by Avidity Biosciences to treat the root cause of facioscapulohumeral muscular dystrophy (FSHD). In the Phase 1/2 FORTITUDE™ clinical trial, del-brax showed more than 50% decrease in DUX4 regulated genes, functional improvement trends, and good safety and tolerability.

ARO-DUX4 is Arrowhead Pharmaceuticals' experimental RNA interference (RNAi) therapy that targets the DUX4 gene, associated with facioscapulohumeral muscular dystrophy (FSHD). Through selective knockdown of DUX4, ARO-DUX4 intends to stop the muscle degeneration and enhance the function of the muscles in patients suffering from FSHD. A Phase 1/2 clinical trial has been proposed to test its efficacy and safety.

RG 6237 (GYM329) is an experimental anti-latent myostatin antibody produced by Chugai Pharmaceutical and Roche. It is designed to block myostatin, a muscle growth regulator, with the purpose of increasing muscle strength and mass. In Phase II trials for facioscapulohumeral muscular dystrophy (FSHD), RG 6237 is given subcutaneously.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the facioscapulohumeral muscular dystrophy market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the facioscapulohumeral muscular dystrophy market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current facioscapulohumeral muscular dystrophy marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the facioscapulohumeral muscular dystrophy market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the facioscapulohumeral muscular dystrophy market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the facioscapulohumeral muscular dystrophy market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (​2019-2035​) of facioscapulohumeral muscular dystrophy across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of facioscapulohumeral muscular dystrophy by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of facioscapulohumeral muscular dystrophy by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035​) with facioscapulohumeral muscular dystrophy across the seven major markets?
• What is the size of the facioscapulohumeral muscular dystrophy patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of facioscapulohumeral muscular dystrophy?
• What will be the growth rate of patients across the seven major markets?

Facioscapulohumeral Muscular Dystrophy (FSHD): Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for facioscapulohumeral muscular dystrophy drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the facioscapulohumeral muscular dystrophy market?
• What are the key regulatory events related to the facioscapulohumeral muscular dystrophy market?
• What is the structure of clinical trial landscape by status related to the facioscapulohumeral muscular dystrophy market?
• What is the structure of clinical trial landscape by phase related to the facioscapulohumeral muscular dystrophy market?
• What is the structure of clinical trial landscape by route of administration related to the facioscapulohumeral muscular dystrophy market?