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Tarix Orphan receives rare pediatric disease designation from FDA for TXA127 candidate
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Tarix Orphan has been granted a rare pediatric disease designation from the FDA for its drug candidate TXA127 for the treatment of recessive dystrophic epidermolysis bullosa, according to a press release.
TXA127 is a pharmaceutical formulation of the naturally occurring peptide angiotensin (1-7), which the company is developing to treat a number of orphan and genetic diseases, with an initial focus on Duchenne muscular dystrophy.
TXA127 interferes with the TGF-beta pathway, which is involved in the pathophysiology of dystrophic epidermolysis bullosa as well as several other serious orphan diseases including Marfan Syndrome and muscular dystrophy. The formulation is expected to enter phase 2 in early 2017.
“The [rare pediatric disease] designation for TXA127 in [recessive dystrophic epidermolysis bullosa] supplements the Orphan Drug Designation previously granted by the FDA in this indication,” Richard Franklin, president and CEO of Tarix Orphan, said in the release. “[Recessive dystrophic epidermolysis bullosa] is a devastating disease. We have been very encouraged by the preliminary animal data we have generated and are looking forward to confirming these results in an additional model through the support of DEBRA International, the European patient organization focused on epidermolysis bullosa.”
Reference : www.tarixpharma.com