Psiron Limited (ASX: PSX) today announced that it has received orphan drug
designation from the U.S. Food and Drug Administration (FDA) for its lead oncolytic virus
Cavatak™ (coxsackievirus A21) for the treatment of stage II (T4), stage III and Stage IV
Psiron’s strategy is to pursue an aggressive timeline to advance Cavatak™ to the
market within 3-5 years. This will be achieved by targeting orphan drug indications such
as melanoma. Orphan drug status is a useful method for compressing the clinical
development timeframe from 5-9 years down to as little as 2-3 years. Other benefits
include: market protection upon registration (7 years against generic products), a
reduction in clinical data required for product registration, tax incentives and reduced
In granting orphan drug status for Cavatak™, the FDA is essentially acknowledging
Cavatak’s potential ability to improve the currently low survival rate of metastatic
“Receiving orphan drug designation for Cavatak™ is an important step forward in
Psiron’s mission to expedite the clinical development program of Cavatak™ said Julie
Nutting, Psiron’s CEO. "Orphan drug designation has the potential to markedly reduce
Cavatak’s drug registration timeframe and ultimately time to market."
In addition to the potential benefits of orphan drug designation, targeting melanoma will
allow Psiron the opportunity to apply to the FDA for “Fast Track” review once Cavatak’s
clinical program is further advanced.
Cavatak™ is currently in Phase 1 studies.
For further information contact
Ms Julie Nutting
TEL: 61 2 9889 1200