Tarix Orphan, a private biopharmaceutical company based in Cambridge, MA, announced that the U.S. FDA has granted Fast Track Designation to TXA127 (angiotensin 1-7) to reduce skeletal muscle damage and fibrosis and thereby improve muscle strength in Duchenne Muscular Dystrophy (DMD) patients. Tarix has previously received Orphan Drug Status for TXA127 in the DMD indication in both the U.S. and Europe. TXA127 is a pharmaceutical grade formulation of the naturally occurring peptide Angiotensin (1-7) which Tarix Orphan is developing for the treatment of a number of orphan and genetic diseases, with an initial focus on DMD.
DMD is a genetic disorder that affects approximately 1 in 3500 male births worldwide, with symptoms usually appearing before age 6. It is caused by a defective gene for dystrophin, a protein in muscle. There is no cure for DMD and current treatments are aimed at managing the symptoms and slowing the course of the disease.