FHSD (facioscapulohumeral muscular dystrophy) is a genetic muscle disorder, characterised by affecting the muscles of the face, shoulder blades and upper arms. The cause of FSHD is thought to be the upregulation of a protein called DUX4, which is toxic to muscles.
This new product (GBC0905) was developed by Genea Biocells, a neuromuscular disease focused company working within the preclinical setting. Currently, there are no treatments for FSHD on the market, meaning there is a high unmet need. GBC0905 is a first-in-class molecule which selectively inhibits DUX4 in the affected skeletal muscles, which many experts within the field believe could be curative. The FDA's approval last week has given cause for celebration as it indicates the chance for this molecule to move forward into clinical trials.
As well as their orphan designation the FDA also gave feedback on Genea Biocell's technology platform. This platform was the first human stem cell model of FSHD, showing cellular hallmarks of the disease allowing cell-based screening of potential new molecules. The FDA lauded the platform, validating it as a highly useful platform for modeling any number of other myogenic indications. This could mean that there will be more development in this much neglected area of drug development.
“We are very pleased that the FDA granted Orphan Drug Designation to GBC0905 as an accomplishment for our team and an important milestone in our journey toward bringing our lead product candidate to the clinic,” commented said Dr. Uli Schmidt, President of Genea Biocells