Scholar Rock Has Received FDA Fast Track Status for Apitegromab
More encouraging news for the FSHD community. Scholar Rock has started dosing FSHD patients in their Phase 2 clinical trial for apitegromab, and the U.S. Food and Drug Administration (FDA) has just given the drug two special designations that can help speed up its development: “Fast Track” and “Orphan Drug”
What’s happening?
Scholar Rock is testing apitegromab as a possible muscle regeneration treatment for patients with FSHD. The drug is currently not approved for FSHD; this is an early-stage trial to find out if it’s safe and if it works.
This Phase 2 clinical trial, called FORGE, has just begun giving its first doses to FSHD participants.
What is apitegromab?
Apitegromab is a lab-made antibody designed to block a protein in the body called myostatin. Myostatin normally limits how much muscle your body builds. By blocking it, the idea is that muscles may grow larger and stronger.
This isn’t a brand-new drug, and has already been tested in people with spinal muscular atrophy (SMA), a different neuromuscular disease, where it showed real improvements in motor function in a large, late-stage study. Scholar Rock is now hoping it can help people with FSHD too.
In mouse studies of FSHD, a similar version of the drug increased muscle mass, strength, and endurance, which is part of why researchers are hopeful about testing it in people.
What does the FORGE trial involve?
- About 60 adults with genetically confirmed FSHD will take part.
- Participants are randomly assigned to receive either apitegromab or a placebo, given as an IV infusion every 4 weeks for about a year (52 weeks).
- Neither participants nor researchers will know who is getting the real drug until after the study (this is called “double-blind” and helps make results more reliable).
- Researchers will mainly be measuring changes in muscle volume, using MRI scans, along with muscle fat content and overall safety.
What does “Fast Track” and “Orphan Drug” designation mean?
These are special statuses the FDA can grant to promising treatments for serious or rare diseases:
- Fast Track is meant to speed up the review process for drugs that address a serious unmet medical need.
- Orphan Drug designation is given to treatments for rare diseases (like FSHD) and can come with additional support and incentives for the company developing it.
Importantly, neither designation means the drug is approved or guaranteed to work, it simply means the FDA sees enough promise to prioritize working with the company as development continues.
What does this mean for the FSHD community?
There are currently no approved treatments for FSHD or muscle regeneration in FSHD patients, so any new trial is meaningful news. This trial will take time but it represents real progress toward a future muscle regeneration option.
Read the full announcement below: