FDA Grants Orphan Drug Designation to Selten Pharma’s Pulmonary Arterial Hypertension Candidate

Today, privately-held, rare disease focused Selten Pharma, Inc. announced that US health regulators granted its investigational pulmonary arterial hypertension (PAH) drug orphan drug designation.

The company said that its lead compound tacrolimus (SPI-026) has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of PAH. The agency grants ODD to novel drugs or biologics that treat a rare disease or condition, defined as affecting fewer than 20,000 patients in the US. If the drug is approved by the FDA, Selten will receive a seven-year period of US marketing exclusivity, as well as financial benefits.

“We are pleased to receive Orphan Drug Designation for SPI-026, which highlights the significant need for new medications for patients suffering from PAH. We greatly appreciate the FDA’s support of our efforts to evaluate our SPI-026 program for the treatment of PAH. The results of the proof-of-concept, safety, and tolerability study in this indication have been very encouraging,” said Leo Gu, PhD, Co-CEO and President of Selten Pharma.

PAH is a life-threatening disease which is characterized by elevated blood pressure in the pulmonary arteries. Severe constriction of the blood vessels in the lungs causes very high pulmonary arterial pressures, which makes it difficult for the heart to pump blood through the lungs to be oxygenated. It is very common for patients with PAH to develop significant increases in pulmonary vascular resistance (PVR) and sustained elevations in pulmonary artery pressure (PAP), which ultimately lead to right ventricular failure and death. The current treatments for PAH involve calcium channel antagonists, prostacyclins, prostacyclin receptor agonist, endothelin receptor antagonists, phosphodiesterase-5 (PDE5) inhibitors, and long-term anticoagulant therapy, and aim to reduce symptoms, improve quality of life and slow disease progression.

SPI-026 is an investigational BMPR2 (Bone morphogenetic protein receptor type II) pathway activator being evaluated for the treatment of patients with PAH. The drug prevented the development of PAH in mice with a deletion of BMPR2 endothelial cells in a chronic hypoxia model, and reversed PAH and neointimal/occlusion in the lungs in rats with neointima formation following VEGF receptor blockade and chronic hypoxia.

“We are excited to begin the Phase 2b clinical trial of SPI-026, which we believe has the potential to be the first disease-modifying treatment for patients with this life threatening disease. SPI-026 has been shown to activate the BMPR2 pathway and possibly could even reverse the effects of the disease,” said Narinder S. Banait, PhD, JD, Co-CEO and General Counsel of Selten Pharma.

Source: Selten Pharma, Inc.

Last updated: 3/18/15; 9:55am EST

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