FDA Grants Arena Pharmaceuticals’ PAH Investigational Drug Orphan Drug Status

The U.S. Food and Drug Administration has granted APD811, an investigational drug candidate internally developed by Arena Pharmaceuticals, orphan drug status. APD811 is an oral agonist of the prostacyclin (IP) receptor, which is designed for the treatment of vasospastic diseases, such as pulmonary arterial hypertension (PAH).

“The FDA Office of Orphan Products Development (OOPD) evaluates scientific and clinical data submissions from sponsors to identify and designate drug candidates that could potentially treat rare diseases to help advance the evaluation and development of such products,” explained Craig M. Audet, Ph.D., Arena’s senior vice president of operations and head of global regulatory affairs.

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The orphan drug status is granted to medications designed to treat rare diseases or conditions that affect less than 200,000 people in the Unites States, and enables companies to apply for several developmental incentives, such as tax credits for qualified clinical testing, fees exemption from the Prescription Drug User Fee Act (PDUFA), and a seven-year marketing exclusivity period after approval.

“We are pleased with the OOPD’s designation of orphan drug status for the active moiety of APD811, and we look forward to advancing this drug candidate into a Phase 2 clinical trial program later this year,” Audet added, noting that the company is engaged in improving health by seeking to bring innovative medicines targeting G protein-coupled receptors to the market through researching, developing, and commercializing drugs to address unmet medical needs.

Pulmonary arterial hypertension is a rare but severe disease with an estimated five-year survival rate of only 57% after diagnosis, according to the Registry to Evaluate Early And Long-term PAH disease management (REVEAL), which registers data from patients in the United States. The progressive and life-threatening disease is characterized by the rise of the blood pressure in the arteries that go from the heart to the lungs, causing physical limitations and heart failure.

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