PYC Therapeutics (ASX:PYC) has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its drug candidate VP-001, aimed at treating Retinitis Pigmentosa type 11 (RP11). This breakthrough therapy is currently undergoing multiple clinical trials, representing a significant step forward for patients affected by this childhood disease. The ODD status confers several benefits, including tax credits and potential market exclusivity upon approval.
Retinitis Pigmentosa (RP) is a group of genetic disorders that affect the retina, leading to a gradual loss of vision. It primarily damages the light-sensitive cells in the retina known as photoreceptors, causing symptoms like difficulty seeing at night and loss of peripheral vision. RP can eventually lead to blindness as the disease progresses. .
CEO Glenn Noronha noted, “We are excited by the progress of VP-001, especially as it remains the first drug candidate targeting RP11 to advance into human trials. The FDA’s recognition of its potential is a testament to our commitment to bringing precision RNA therapies to patients who have no other options.”
PYC has also been conducting studies to complement the positive efficacy results and is on track to release further data later this year.