Third Orphan Drug Designation Granted to TXA127 in Muscular Dystrophy

February 17, 2016

FDA withdraws warning letter after resolution

The Food and Drug Administration (FDA) has granted Orphan Drug designation to TXA127 (Tarix Orphan) for the treatment of laminin-deficient congenital muscular dystrophy (LAMA2 MD or MDC1A).

Tarix Orphan announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to TXA127 for the treatment of laminin-deficient congenital muscular dystrophy (LAMA2 MD or MDC1A).

Approximately 1 in 30,000 people develop early-onset LAMA2-related muscular dystrophy, which accounts for 30–40% of total cases of congenital muscular dystrophy.

TXA127 previously received Orphan Drug designations for other muscular dystrophies including Duchenne’s muscular dystrophy (DMD) and limb girdle muscular dystrophy (LGMD).

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

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