FDA orphan designation, page-11

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PYC Therapeutics gets FDA approval

PYC Therapeutics (ASXYC) has received an Orphan Drug Designation (ODD) from the US FDA for its drug candidate, PYC-001, aimed at treating OPA1-associated vision loss.
The ODD is a special designation given to drugs targeting rare diseases. It comes with perks like tax credits for clinical trials, exemptions from certain regulatory fees, and the possibility of market exclusivity for up to 7 years post-approval.
PYC’s investigational drug candidate, PYC-001, is said to be a significant advancement in precision therapy.
The drug was designed to restore the expression of the OPA1 gene to levels necessary for optimal retinal function.
Utilising PYC’s proprietary drug delivery technology, the company has tackled a major hurdle for RNA drugs by ensuring effective delivery to the targeted cells affected by ADOA ( Autosomal Dominant Optic Atrophy).
ADOA is a progressive and irreversible blinding eye disease. It affects approximately 1 in every 35,000 people representing a market size of ~$2 billion per annum.
ADOA stems from a mutation in one copy of the OPA1 gene, which results in inadequate levels of OPA1 gene expression, impacting the normal function of retinal ganglion cells.
The resulting cellular stress and eventual cell death disrupt the transmission of visual signals from the retina to the brain, ultimately causing vision loss in ADOA patients.