Early-onset LAMA2-related muscular dystrophy occurs in approximately 1 in 30,000 individuals and accounts for between 30 and 40 percent of total cases of congenital muscular dystrophy.
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, including congenital muscular dystrophies.
Tarix Orphan president and CEO Richard Franklin said: "This is the third U.S. Orphan Drug designation granted to TXA127 for muscular dystrophies, with previously received designations for Duchenne’s muscular dystrophy (DMD), our lead indication, and limb girdle muscular dystrophy (LGMD).
"We sought these Orphan Drug designations based on very positive pre-clinical data from mdx, sgcd -/- and DyW models, which correspond to DMD, LGMD and congenital muscular dystrophy (MDC1A) respectively. We now look forward to initiating the clinical evaluation of TXA127 in each of these indications, starting with our previously announced Phase 2 study in DMD, which we expect to begin later this year."
Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 Americans annually. Orphan drug designation entitles Tarix Orphan to a seven-year period of marketing exclusivity in the United States for TXA127 if it is approved by the FDA for the treatment of congenital muscular dystrophy (MDC1A), and enables the company to apply for research funding, tax credits for certain research expenses, and a waiver from the FDA’s application user fee.
About TXA127
TXA127 is a pharmaceutical grade formulation of the naturally occurring peptide Angiotensin (17) which Tarix Orphan is developing for the treatment of a number of orphan and genetic diseases, with an initial focus on DMD. Additional diseases which may benefit from treatment with TXA127 include congenital muscular dystrophies, Marfan Syndrome, and amyotrophic lateral sclerosis (ALS).
TXA127 is part of the "alternative renin angiotensin system (RAS)" and counteracts the "classical" RAS, which promotes hypertension, fibrosis, hypertrophy and inflammation.