Tarix Pharmaceuticals announced that the US Food and Drug Administration (US FDA) has granted TXA127, the company’s lead peptide drug candidate, orphan drug status for the treatment of Pulmonary Arterial Hypertension (PAH). Emerging science has demonstrated that the naturally occurring peptide angiotensin (1-7), the pharmaceutical ingredient in TXA127, exhibits a protective effect in animal models of PAH, suggesting a potentially beneficial role for TXA127 against this disease.

Rick Franklin, MD, PhD, chief executive officer of Tarix, commented, “We are extremely pleased that TXA127 has been granted orphan drug status for this indication by the FDA. This is an important development for Tarix and is part of our strategy to build a portfolio of products using TXA127 and our synthetic peptide PanCyte to treat a variety of serious pulmonary diseases.”

Tarix is developing TXA127 for multiple therapeutic indications, including enhancement of engraftment following peripheral blood and cord blood stem cell transplantation and Acute Respiratory Distress Syndrome (ARDS). In 2010, TXA127 was granted Orphan Drug designation as a treatment to enhance engraftment in patients receiving a stem cell transplant. TXA127 has also received Orphan Drug designation for the treatment of Myelodysplastic Syndrome (MDS).

PAH is a rare disease that is very difficult to diagnose in its early stages as many of the initial symptoms can be non-specific, resulting from insufficient cardiac output. Prognosis for the disease is extremely poor.  Untreated patients have a median survival of only two and half years, with the most common cause of death being sudden death caused by right ventricular failure. Not all patients respond to current treatments, which include calcium channel blockers and prostacyclin analogues. For the majority of patients that are treated with these drugs, five year survival is only 54%.  Lung transplantation offers the only hope of cure but it is reserved for patients with end-stage disease that have failed all other therapies and it is associated with high morbidity because of rejection, infection, and bronchiolitis obliterans.

Orphan drug designation qualifies a company for several benefits under the Orphan Drug Act of 1983 (ODA), as amended. These benefits include a 7-year period of orphan drug exclusivity upon product approval, a tax credit for certain clinical testing expenses for the orphan drug, written guidance on the non-clinical and clinical studies needed to obtain marketing approval of an orphan drug, and orphan drug grants.

Tarix Pharmaceuticals is a clinical stage biopharmaceutical company developing peptide drugs to address significant unmet medical needs.