Cambridge, MA (January 18, 2017): Tarix Orphan LLC today announced that the U.S. Food and Drug Administration has granted a Rare Pediatric Disease (RPD) designation for the company’s drug candidate TXA127 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), a rare genetic skin disorder. There are currently no approved therapies for RDEB and treatment is limited to supportive care.
“The RPD designation for TXA127 in RDEB supplements the Orphan Drug Designation previously granted by the FDA in this indication,” said Richard Franklin, President and Chief Executive Officer of Tarix Orphan LLC. “RDEB 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.”
The FDA defines ’Rare Pediatric Disease’ as a disease that primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 persons in the United States. Under the Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval for a drug or biologic for a ‘rare pediatric disease’ may qualify for a voucher which can be redeemed to receive a priority review of a subsequent marketing application for a different product. The Priority Review Voucher is requested at the time of the marketing application and awarded upon approval of the product. The voucher may only be used once, but may be sold or transferred an unlimited number of times.
TXA127, a pharmaceutical formulation of the natural Angiotensin (1-7) peptide, interferes with the TGF beta pathway, which is involved in the pathophysiology of DEB as well as several other serious orphan diseases including Marfan Syndrome and muscular dystrophy. Previous preclinical studies with TXA127 have shown its ability to reduce the fusion of digits, a symptom in RDEB mice that is analogous to mitten deformity common in patients with severe RDEB.
Recessive dystrophic epidermolysis bullosa (RDEB) is one of the more severe subtypes of EB, a group of rare genetic connective tissue-fragility disorders, characterized by extremely fragile skin and mucosa, and recurrent blister formation, resulting from minor mechanical friction or trauma.
The skin has two layers; the outer layer is called the epidermis and the inner layer the dermis. Normally, there are protein ‘anchors’ between the two layers that prevent them from separating from one another. In people with RDEB, the two skin layers lack the anchors that hold them together, and any action that creates friction between the layers (like rubbing or pressure) will create blisters and painful sores. People with severe RDEB have compared the sores to third-degree burns.
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 orphan and Fast Track designations and is expected to enter Phase II in DMD in early 2017.
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). TX127 has shown therapeutic activity in animal models of Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophy (LGMD), congenital muscular dystrophy (MDC1A), and Marfan syndrome. Tarix Orphan has broad IP protection for TXA127, and Orphan Drug Designations (ODDs) have been granted for DMD, LGMD, and RDEB in the United States, and for DMD in Europe. For more information on Tarix Orphan, please visit our website at www.tarixorphan.com.
Elizabeth Wagner, Vice President
Kureczka/Martin Associates (415) 821 2413