Amryt, a clinical-stage specialty pharmaceutical company focused on treatments for orphan diseases, has received orphan drug designation from U.S. Food and Drug Administration (FDA) for its drug compound, AP102, which has the potential to treat patients with acromegaly.
Acromegaly is a rare endocrine disorder in which the body produces excessive growth hormone, leading to abnormal growth throughout the body over time.
The FDA’s Orphan Drug Designation program provides orphan status to drugs and biologics that are being developed to address rare diseases or disorders that affect fewer than 200,000 people in the United States. With orphan designation, Amryt qualifies for various incentives, including tax credits for qualified clinical trials and market exclusivity upon regulatory approval, the company said on Monday.
Joe Wiley, CEO, commented:
“We are pleased that our drug compound, AP102, has received orphan drug designation from the FDA. AP102 has the potential to treat acromegaly, a rare disorder which is estimated to affect four to 13 in every 100,000, and causes excess growth of body tissue.
We believe that AP102 has the potential to improve outcomes for patients facing this debilitating disease and receiving the FDA’s orphan drug designation marks an important regulatory milestone for Amryt as we prepare to bring AP102 into the clinic next year.”
Acromegaly is a rare endocrine disorder that most commonly results from an adenoma, a benign tumour of the pituitary gland that secretes excessive growth hormone (GH) and leads to excess production of the hormone IGF-1. The progression of acromegaly is typically slow and diagnosis is often delayed by years. As the disease advances, patients typically exhibit abnormal growth throughout the body.