New Duchenne Muscular Dystrophy Tx Granted Fast Track Status

Tarix Orphan announced that the Food and Drug Administration has granted Fast Track designation to TXA127 (angiotensin 1-7) to reduce skeletal muscle damage and fibrosis and thereby improve muscle strength in patients with Duchenne Muscular Dystrophy (DMD), a genetic disorder occurring primarily in boys that is characterized by rapidly progressive muscle degeneration and weakness.

The company expects to initiate in early 2016 a multi-site, double-blind, randomized, placebo-controlled, Phase 2 study, conducted for 48 weeks, to evaluate the safety and efficacy of TXA127 in patients with DMD, followed by a 96-week open-label extension study. Co-endpoints will include muscle quality assessment by MRI, ambulatory assessments including the 2-Minute Walk Test, and safety assessments.

RELATED: FDA Accepts Eteplirsen NDA for Duchenne Muscular Dystrophy

TXA127 is a pharmaceutical grade formulation of the naturally occurring peptide angiotensin (1-7).

For more information visit TarixOrphan.com.

Loading links....