The US Food & Drug Administration (FDA) has granted Orphan Drug Designation to Selten Pharma's lead compound tacrolimus (SPI-026) has been granted Orphan Drug Designation by for the treatment of pulmonary arterial hypertension (PAH).
Leo Gu, Ph.D., Co-CEO and president of Selten Pharma, said, "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 programme for the treatment of PAH. The results of the proof-of-concept, safety, and tolerability study in this indication have been very encouraging."
"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," commented Narinder S. Banait, Ph.D., J.D., Co-CEO and general counsel of Selten Pharma.
Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the US. The designation provides the drug developer with a seven-year period of US marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees. The OOPD also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.
Selten's SPI-026 programme is an investigational BMPR2 (Bone morphogenetic protein receptor type II) pathway activator being evaluated for the treatment of patients with pulmonary arterial hypertension. It was discovered by Dr. Edda Speikerkoetter, and is being developed with the help of Dr. Roham Zamanian, both at Stanford University. It 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. It has been shown to be safe and tolerable in patients with PAH.
Selten Pharma is a privatively held biopharmaceutical company formed in 2013 that is focused on the development and commercialization of therapies for the treatment of rare diseases.