CAMBRIDGE, Mass., June 12, 2017 /PRNewswire/ -- Tarix Orphan LLC today announced that the European Medicines Agency has granted Orphan Drug status to the company's lead compound, TXA127, for the treatment of epidermolysis bullosa (EB), a rare and often severe genetic connective tissue disorder. EB's main symptom is extremely fragile skin that blisters and tears from minor friction or trauma. The disease can also seriously affect internal organs and bodily systems. 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 EB.
Clare Robinson, Head of Research at DEBRA International, a leading EB patient organization, responded to the announcement, "People with EB frequently face disabling scarring and contractures as a result of the fibrosis that is a consequence of their extreme skin fragility and chronic wounds. A safe, effective treatment to reduce fibrosis would have an immense positive benefit on patients' quality of life, and data on TXA127 so far indicate its promise as a candidate."
Rick Franklin, Chief Executive Officer of Tarix Orphan, commented, "Orphan designation in Europe for TXA127 for the treatment of EB complements the designation we have received in the United States, and forms an integral part of our strategy to address unmet needs in rare pediatric diseases. We are moving forward with confirmatory pre-clinical studies being done by Professor Bruckner-Tuderman's lab the University of Freiburg, with the support from DEBRA Austria, in preparation for the initiation of a clinical development program for this important condition."
Orphan status is granted by the EMA to promote the development of products that demonstrate promise for the treatment of rare diseases. Orphan drug designation entitles Tarix Orphan to a 10-year period of marketing exclusivity in Europe for TXA127 if it is approved by the EMA for the treatment of epidermolysis EB.
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 Duchenne muscular dystrophy (DMD). TXA127 has U.S. Orphan Drug and Fast Track designations and is expected to enter Phase 2 clinical testing in DMD in early 2018.
About Tarix Orphan
Tarix Orphan LLC is a private biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases. The company's lead drug candidate is TXA127, a pharmaceutical formulation of the naturally occurring peptide Angiotensin (1-7), for the treatment of rare neuromuscular and connective tissue diseases. TX127 has shown therapeutic activity in animal models of Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophy (LGMD), congenital muscular dystrophy (MDC1A), epidermolysis bullosa (EB) and Marfan syndrome. Tarix Orphan has broad IP protection for TXA127, and Orphan Drug Designations (ODDs) have been granted for DMD, LGMD, CMD and EB in the United States, and for DMD and EB in Europe. For more information on Tarix Orphan, please visit our website at www.tarixorphan.com.
Elizabeth Wagner, COO
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SOURCE Tarix Orphan LLC