PhaseRx, Inc (NASDAQ:PZRX) shares soared on Monday as the US Food and Drug Administration (FDA) granted its lead candidate drug, PRX-OTC, orphan drug designation.
The designation effectively allows the development of PRX-OTC, which is being developed for the treatment of an inherited liver disease that causes ammonia to accumulate in the blood, to be fast-tracked.
The designation is given to drugs that address diseases or circumstances that occur so infrequently in the USA there is no reasonable expectation that the cost of bringing the drug to market will be recovered from sales.
Orphan drugs qualify for tax credits to offset clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.
Shares in PhaseRX, which is developing messenger ribonucleic acid (mRNA) treatments for life-threatening inherited liver diseases in children, shot up 124% to US$2.5316 in early deals.
Story by ProactiveInvestors