Novel GLP-1 Receptor Agonist Designated Orphan Drug for Polymyositis

The Food and Drug Administration (FDA) has granted Orphan Drug designation to PF1801 for the treatment of polymyositis.

Polymyositis is a type of inflammatory myopathy characterized by chronic muscle inflammation and weakness. The exact cause of the disease is unknown. PF1801 is a novel long-acting glucagon-like peptide-1 (GLP-1) receptor agonist. Study findings suggest that activation of GLP-1 receptor signaling may be beneficial in the treatment of atrophy-related muscular diseases.

In a press release, ImmunoForge stated: “This Orphan Drug Designation is of great significance in confirming the potential of PF1801 as a treatment for rare musculoskeletal diseases, and we hope that it will serve as a source of hope for patients suffering from rare diseases with the various new drugs we are developing.”

The FDA previously granted Orphan Drug designation to PF1801 for the treatment of Duchenne muscular dystrophy.

References

1. ImmunoForge Inc. receives US FDA ODD for treatment of polymyositis. News release. ImmunoForge Inc. Accessed September 27, 2021. https://www.prnewswire.com/news-releases/immunoforge-inc-receives-us-fda-odd-for-treatment-of-polymyositis-301384587.html. 
2. Pipeline: PF1801. ImmunoForge Inc. Accessed September 27, 2021. https://www.immunoforge.com/m21.php?slide=1&#section2.