2 September 2026
Scholar Rock’s investigational therapy, apitegromab, has received Fast Track and Orphan Drug designations from the US Food and Drug Administration (FDA) for facioscapulohumeral muscular dystrophy (FSHD). The company has also announced that the first participants have begun receiving the investigational treatment in the Phase 2 FORGE clinical trial.
Scholar Rock has announced two important developments for its FSHD research programme. The US Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to apitegromab for FSHD. In addition, participants will begin receiving apitegromab in the Phase 2 FORGE clinical trial.
Apitegromab is an investigational therapy, which means that it is still being studied and has not been approved for the treatment of FSHD. The FORGE study is the first clinical trial to study apitegromab specifically in people living with FSHD.
What are FDA Fast Track and Orphan Drug designations?
The FDA’s Fast Track designation, given to investigational therapies for serious health conditions, is intended to help speed up the development and review process. However, Fast Track designation does not mean that a potential therapy has been proven to work or that it will be approved.
The FDA has also granted Orphan Drug designation to apitegromab for FSHD. This designation is designed to encourage the development of treatments for rare diseases.
These two designations are important steps for the development programme, but they do not provide evidence that apitegromab is effective or safe for people living with FSHD.
What is apitegromab?
Apitegromab is designed to affect myostatin, a protein that helps regulate muscle growth. Myostatin acts as part of the body’s way of controlling how much muscle grows. Apitegromab is designed to block the activation of myostatin in skeletal muscle. The aim is to explore whether blocking myostatin could increase or preserve muscle mass and potentially improve physical function.
It is important to remember that an increase in muscle mass does not automatically mean that a person will become stronger or find everyday activities easier. Clinical trials are needed to find out whether changes in muscle lead to meaningful benefits for people living with FSHD.
The Phase 2 FORGE clinical trial
The FORGE trial is a Phase 2 clinical trial. Phase 2 studies are usually designed to provide more information about a potential therapy’s effects and safety.
The study will include approximately 60 adults with genetically confirmed FSHD. Participants will be randomly assigned to receive either apitegromab or a placebo. Treatment will be given intravenously every four weeks for 52 weeks.
The trial is randomised, double-blind and placebo-controlled. This means participants are randomly assigned to a treatment group. Neither the participants nor the researchers know who is receiving the investigational therapy and who is receiving the placebo (“sugar pill”) during the study.
The main outcome being studied is the change in total lean muscle volume after 52 weeks. This will be measured using MRI.
The study will also look at other changes in muscle, as well as safety and tolerability. It will include additional exploratory outcomes, including functional measures.
Where are the study sites that are conducting the clinical trial?
Check the FORGE study website (scholarrockforge.com) or your local clinical trial registry, such as clinicaltrials.gov or eudract.ema.europa.eu, for the location of participating sites and eligibility criteria. Although all study locations are not yet running, there are expected to be about 20 sites across North America and Europe.
What previous research tells us
The FSHD programme is supported by research done on mice. Scholar Rock reports that a form of apitegromab increased their muscle mass, strength and endurance.
Apitegromab has also been studied in people with spinal muscular atrophy (SMA), another neuromuscular condition. European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. However, SMA and FSHD are different diseases. Results in people with SMA cannot tell us whether the potential therapy will work in people with FSHD.
The FORGE trial will provide the first clinical evidence about how the investigational therapy affects people living with FSHD.
Why does this research matter for the FSHD community?
This announcement shows progress in two areas. First, apitegromab has received two important regulatory designations from the FDA. Second, the Phase 2 FORGE clinical trial is now underway.
The trial will help researchers understand whether blocking myostatin has measurable results in people living with FSHD. It will also provide important information about the investigational therapy’s safety and its possible effects on muscles and their function.
As with all clinical research, the contribution of trial participants, families, clinicians and researchers is essential. Their involvement helps generate the evidence needed to understand whether new treatments could one day provide meaningful benefits for people living with FSHD.
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