FDA Grants Orphan Drug Designation for Potential Treatment of Hypoparathyroidism

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AZP-3601, Amolyt Pharma’s clinical candidate for the treatment of hypoparathyroidism.

“We believe the FDA’s granting of Orphan Drug Designation to AZP-3601 reflects the agency’s recognition that new and more effective treatment options are needed for this serious endocrine disorder,” says Endocrine Society member Thierry Abribat, PhD, chief executive officer of Amolyt Pharma. “We are pleased to have recently dosed the first subject in our Phase 1 clinical trial, and we are committed to executing an efficient development program to diligently bring this promising therapeutic to patients.”

Orphan drug status is intended to advance drug development for rare diseases. The FDA grants ODD to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of diseases or disorders that affect fewer than 200,000 people in the U.S. ODD provides certain incentives, such as tax credits toward the cost of clinical trials and prescription drug user fee waivers. If a product holding ODD receives the first FDA approval for the disease in which it has such designation, the product is entitled to seven years of market exclusivity, which is independent from intellectual property protection.