Treatment for FSHD named orphan drug, put on FDA fast track
Infusion therapy for MD affecting face, upper body now in clinical testing
Written by |
The U.S. Food and Drug Administration (FDA) has granted both fast track and orphan drug designations to Scholar Rock‘s apitegromab as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD), a disease type that characteristically affects muscles of the face and upper body.
The experimental muscle-targeted therapy, which aims to improve motor function in FSHD, is now being tested in a mid-stage U.S. clinical trial that’s still recruiting participants.
The FDA grants fast track status to experimental therapies that aim to fill unmet needs in the treatment of serious health conditions. This regulatory tool aims to speed the development of important new medications, and gives a drug’s developer access to perks such as more frequent feedback from the FDA.
Orphan drug status, meanwhile, is designed to help incentivize companies to invest in developing treatments for rare diseases. This designation offers incentives that would include a guaranteed seven years of market exclusivity for apitegromab if it ultimately wins FDA approval for FSHD.
“We are very pleased to receive both Fast Track and Orphan Drug designations for our apitegromab FSHD program, which underscores the urgency of advancing new treatment options for the FSHD community,” David L. Hallal, chairman and CEO of Scholar Rock, said in a company press release.
FSHD is a form of muscular dystrophy divided into two types. They’re caused by different underlying genetic changes, but the end result is the same: muscle weakness that mainly affects the face, shoulders, and upper arms.
FORGE trial of apitegromab for FSHD now enrolling
Scholar Rock is running a Phase 2 trial dubbed FORGE (NCT07435129) to test apitegromab in adults with FSHD. The study aims to enroll 60 people, ages 18 to 60, at a single center in Austin, Texas. To be eligible, participants must have a confirmed genetic diagnosis of FSHD type 1 or type 2, and also meet certain criteria related to disease status and walking ability. The trial is already dosing patients.
Participants in FORGE are being randomly assigned to receive infusions of apitegromab or a placebo every four weeks for about a year.
The main goal is to evaluate the impact of treatment on total lean muscle volume — that is, the total amount of muscle tissue in the body, as measured by MRI scan.
“With participant dosing now underway in our Phase 2 FORGE study, we are one step closer to realizing the broader potential of our world-leading myostatin platform and bringing a potentially transformative muscle-targeted therapy to people living with FSHD worldwide,” Hallal said.
Myostatin is a protein that normally helps to prevent excessive muscle growth. Apitegromab is an antibody-based medication designed to block the precursor and latent forms of this protein in skeletal muscle. In doing so, the therapy aims to improve muscle strength in people with conditions such as FSHD, which are marked by muscle weakness.
According to Scholar Rock, apitegromab treatment increased muscle mass, strength, and endurance in a mouse model of FSHD.
“Our preclinical data from the [mouse] model and prior literature suggest that, in people living with FSHD, [blocking myostatin] can result in muscle [growth] and improved motor function. Apitegromab therefore holds important potential to impact this disease,” said Akshay Vaishnaw, MD, PhD, president of research and development at Scholar Rock.
With participant dosing now underway in our Phase 2 FORGE study, we are one step closer to … bringing a potentially transformative muscle-targeted therapy to people living with FSHD worldwide.
“We believe FORGE, which assesses changes in lean muscle volume as well as a range of exploratory functional endpoints, will enable robust evaluation of apitegromab and inform on the drug’s potential to drive meaningful functional outcomes,” Vaishnaw added.
The FDA is reviewing an application seeking apitegromab’s approval for spinal muscular atrophy, a genetic disease that, like FSHD, is marked by muscle weakness. A decision from the FDA on that application is expected later this month.
Leave a comment
Fill in the required fields to post. Your email address will not be published.