Cambridge, Massachusetts, January, 3, 2025 — Anida Pharma, a preclinical stage biopharmaceutical company developing first-in-class treatments for vision loss, today announced that the US Food and Drug Administration (F.D.A.) has granted Orphan Drug Designation to AP-001 for the treatment of neurotrophic keratopathy (NK). NK is a potentially blinding condition due to progressive loss of the corneal integrity. The condition is due to the loss of the sensory innervation to the cornea which provides necessary trophic support to maintain its integrity. Loss of innervation is commonly caused by viral infection, HSV-1 and HVZ, but increasingly also diabetes and in rare cases trauma and surgery.
“With the paucity of treatment options and restoration of corneal innervation the only long term treatment solution, we believe there could be a significant role for AP-001,” said Per Gjorstrup MD PhD, Anida Founder and CEO. “The broad activity profile of AP-001 demonstrated as stimulation of nerve regrowth together with promoting corneal epithelial closure and controlling progressive late stage inflammation creates a unique ‘activity triad‘ for the treatment of NK. The treatment is expected to be patient compliant with twice daily dosing and treatment for two months”.
The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation would provide Anida certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees and tax credits for qualified clinical trials.