NM-003 receives orphan drug designation from FDA

The U.S. Food and Drug Administration (FDA) announced that NM-003, a long-acting glucagon-like peptide-2 (GLP-2) receptor agonist, received orphan drug designation for the treatment of acute graft-versus-host disease (aGvHD).¹ NM-003 has previously been approved for use in another rare disease, short bowel syndrome, in the US and Europe following the results of phase III studies (NCT00798967 and NCT02682381).

Orphan drug designation is granted to agents for the treatment, prevention, or diagnosis of rare diseases that affect < 200,000 people in the US. The designation qualifies NM-003 for tax credits for clinical testing, among other benefits.

NM-003, also known as teduglutide, acts on the GLP-2 receptor, which is a G-protein-coupled receptor and is found in multiple cell types within the intestinal tract. The ligand GLP-2 has been shown to have gastrointestinal protective effects by promoting regeneration of Paneth cells and intestinal stem cells. Activation of the GLP-2 receptor causes the secretion of growth factors such as insulin-like growth factors-1 and -2 and stimulates expansion of different intestinal cell populations.²