FDA grants orphan drug status for novel hypoparathyroidism drug
The FDA granted orphan drug designation to an investigational therapy to treat hypoparathyroidism, according to an industry press release.
AZP-3601 (Amolyt Pharma) is a therapeutic peptide designed to target a specific configuration of the parathyroid hormone receptor to safely produce sustained levels of calcium to manage the symptoms of hypoparathyroidism. As Healio previously reported, several analogues of parathyroid hormone in different formulations are being developed to provide more long-lasting effects of the molecule to treat hypoparathyroidism, including AZP-3601. Amolyt announced in October that it enrolled the first participant in a phase 1 clinical trial, designed to demonstrate proof-of-concept for AZP-3601 as a treatment for hypoparathyroidism and to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy following single and multiple ascending doses in healthy participants as well as adults with hypoparathyroidism. Approximately 130 healthy volunteers and patients are expected to be enrolled.
“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,” Thierry Abribat, PhD, CEO of Amolyt Pharma, said in the release. “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.”
The selective action of AZP-3601 is also intended to limit urine calcium excretion by stimulating renal calcium reabsorption, consequently preventing chronic kidney disease. The drug’s unique receptor profile and short half-life are expected to preserve bone integrity, according to the release. Most patients with hypoparathyroidism are middle-aged women, often at increased risk for osteoporosis.
The FDA grants orphan drug designation to drugs and biologics 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. The status provides certain incentives, such as tax credits toward the cost of clinical trials and prescription drug user fee waivers. If a product holding orphan drug designation receives the first FDA approval for the disease in which it has such designation, the product is entitled to 7 years of market exclusivity, which is independent from intellectual property protection.