FDA orphan drug designation doubles PhaseRx shares
pharmafile | November 28, 2016 | News story | Manufacturing and Production, Research and Development, Sales and Marketing | FDA, Orphan Drug Designation, PhaseRx
PhaseRx is celebrating as its lead drug candidate PRX-OTC has been orphan drug designation by the US FDA for the treatment of ornithine transcarbamylase (OTC) deficiency, a rare genetic disorder.
The news sent the US biotech’s shares rocketing by 107% to $2.35 a share, more than double their original value.
OTC is one of six enzymes in the body involved in the breakdown and removal of nitrogen, more commonly known as the urea cycle. OTC deficiency can therefore lead to accumulation of nitrogen in the blood in the form of ammonia (hyperammonia), a neurotoxin, which can damage the central nervous system and lead to vomiting, lethargy and even permanent neurological impairment or coma. PRX-OTC is an intracellular enzyme replacement therapy (i-ERT) which acts by replacing defective enzymes in OTCD sufferers and treat the illness.
“The FDA’s decision to grant PRX-OTC orphan drug designation for OTCD is another important milestone in the development of our lead product candidate, as we prepare to file the IND by the end of 2017 and initiate our clinical trial in 2018,” PhaseRx’s president and CEO Robert W. Overell commented. “PRX-OTC is the first of three drugs in development using our Hybrid mRNA Technology, and we believe it has the potential to correct the disease in children, a population that could particularly benefit from treatment for this rare disease. Our team at PhaseRx is driving hard to advance these drugs to help the lives of families affected by this devastating liver disease that causes irreversible brain damage and potentially fatal ammonia toxicity.
Orphan drug designation is awarded to investigational drugs focused on the treatment of ultra-rare disease, as defined by the number of sufferers of the illness numbering less than 200,000 in the US, and is designed to accelerate and facilitate drug development for these illnesses.
Matt Fellows
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