RemeGen Gains Orphan Drug Designation for Telitacicept in Myasthenia Gravis

China-based RemeGen has received FDA’s Orphan Drug designation for its investigational drug telitacicept for patients with myasthenia gravis, a chronic autoimmune disease which causes muscle weakness.

Telitacicept is a novel fusion protein that targets two cell-signaling molecules critical for B-lymphocyte development, to reduce B-cell mediated autoimmune responses that are implicated in several autoimmune diseases.

The company said it completed a phase 2 study in the first quarter of this year in Chinese patients, with positive results.

The FDA grants Orphan Drug status to investigational drugs with potential to address an unmet clinical need for treating a rare disease. The prevalence of myasthenia gravis in the U.S. is estimated at between 36,000 to 60,000 cases.