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Facioscapulohumeral Muscular Dystrophy

2
Pipeline Programs
5
Companies
6
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
1
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody
1100%
+ 5 programs with unclassified modality

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Avidity Biosciences
Avidity BiosciencesCA - San Diego
1 program
1
del-braxPhase 31 trial
Active Trials
NCT07038200Recruiting200Est. Jul 2028
Scholar Rock
Scholar RockMA - Cambridge
1 program
1
ApitegromabPhase 2Monoclonal Antibody1 trial
Active Trials
NCT07435129Not Yet Recruiting60Est. Dec 2028
aTyr Pharma
aTyr PharmaSAN DIEGO, CA
2 programs
ATYR1940PHASE_1_21 trial
ATYR1940PHASE_1_21 trial
Active Trials
NCT02836418Completed8Est. Apr 2017
NCT02531217Completed9Est. May 2016
Dyne Therapeutics
1 program
FSHD-specific functional rating scaleN/A1 trial
Active Trials
NCT03458832Active Not Recruiting324Est. Dec 2027
Merck & Co.
Merck & Co.RAHWAY, NJ
1 program
ACE-083PHASE_21 trial
Active Trials
NCT03943290Terminated62Est. Mar 2020

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
Avidity Biosciencesdel-brax
Scholar RockApitegromab
Merck & Co.ACE-083
aTyr PharmaATYR1940
aTyr PharmaATYR1940
Dyne TherapeuticsFSHD-specific functional rating scale

Clinical Trials (6)

Total enrollment: 663 patients across 6 trials

NCT07038200Avidity Biosciencesdel-brax

A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

Start: Jun 2025Est. completion: Jul 2028200 patients
Phase 3Recruiting
NCT07435129Scholar RockApitegromab

Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

Start: Aug 2026Est. completion: Dec 202860 patients
Phase 2Not Yet Recruiting
NCT03943290Merck & Co.ACE-083

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

Start: May 2019Est. completion: Mar 202062 patients
Phase 2Terminated
NCT02836418aTyr PharmaATYR1940

Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)

Start: Jul 2016Est. completion: Apr 20178 patients
Phase 1/2Completed
NCT02531217aTyr PharmaATYR1940

Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension

Start: Aug 2015Est. completion: May 20169 patients
Phase 1/2Completed
NCT03458832Dyne TherapeuticsFSHD-specific functional rating scale

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Start: Mar 2018Est. completion: Dec 2027324 patients
N/AActive Not Recruiting

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 663 patients
5 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.