From Global Newswire. Read full release.
CAMBRIDGE, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to losmapimod for the potential treatment of facioscapulohumeral muscular dystrophy (FSHD).
“There are no approved therapies to treat patients with FSHD, and losmapimod is currently the only drug in clinical development for this serious and debilitating disease,” said Judith Dunn, PhD, Fulcrum’s president of research and development. “We are pleased that the FDA has granted Fast Track designation, which we believe demonstrates the potential for losmapimod to address unmet medical needs for people living with FSHD.”
Fast Track Designation is intended to facilitate development and expedite the review of drugs that treat serious conditions so an approved product can reach the market expeditiously. It enables the company to have more frequent interactions with the FDA throughout the drug development process and allows for eligibility for priority review and accelerated approval in certain cases, as well as a rolling review.
Fulcrum is on track to report full data from ReDUX4, a Phase 2b randomized, double-blind, placebo-controlled trial of losmapimod in FSHD patients, at the virtual FSHD International Research Congress taking place June 24-25, 2021. Data will include the primary endpoint, reduction from baseline of DUX4-driven gene expression, as well as a pre-specified sensitivity analysis assessing biopsies with the highest pre-treatment level of DUX4-driven gene expression. Additional data to be reported include secondary endpoints evaluating disease progression via skeletal muscle MRI, exploratory endpoints assessing muscle function measures and patient reported outcomes.
Losmapimod previously received Orphan Drug Designation for FSHD.
About Losmapimod
Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,600 subjects in clinical trials across FSHD and multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.
About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.
Please visit www.fulcrumtx.com.
This is wonderful news! I was part of the clinical trial in the beginning stages and went through the MRI scans but was not a candidate based on my current muscle status. I have been praying for this to be our cure for quite some time.
Does this mean it can be prescribed, then supplied through Fulcrum?
Not yet. It means the FDA will prioritize meetings with Fulcrum as it presents its Phase 2b trial data to reduce delays in the review process.
How do we get access to this?
The Phase 2b data on Losmapimod will still need to undergo review by the FDA, and the FDA will need to make a determination on how and when patients can have access to it. The Fast Track designation means that the FDA will accelerate its review process in recognition of the urgent and unmet medical need.
This sounds brilliant, my wife is currently in hospital with pnuemonia due to a wasting diaghram.
we are running lower on time, but something like this should give everyone hope. I just hope it becomes available in the U.K.
We’re sorry to hear about your wife. I hope her pulmonologist prescribed a cough-assist and BiPAP to help her. The UK patient community should be preparing to advocate for access to future treatments with the UK governmental bodies that decide which treatments will be approved and covered by the national health services.
whether it is realistic to be approved in the next 2 years
Fast Track from FDA seems to be that something that should take a couple weeks to do will take a year. The word urgency certainly applies to the need from those that suffer from FSHD but the time frame for what is happening between Fulcrum and the FDA could in no way been seen as urgent.