PhaseRx Inc. said the European Medicines Agency's Committee for Orphan Medicinal Products issued a positive opinion recommending orphan medicinal product designation for PRX-ASL to treat argininosuccinate lyase deficiency, or ASLD.
ASLD is a rare liver disorder caused by an inherited single-gene deficiency that results in elevated ammonia in the blood. This can lead to irreversible neurological impairment, coma and death. The only known cure for ASLD is a liver transplant.
In a preclinical model, the drug has shown therapeutic potential, including reduction in the levels of compounds whose elevation are the hallmark of ASLD such as plasma citrulline, argininosuccinic acid and blood ammonia.
The EMA will forward its opinion to the European Commission, which is responsible for formally granting the orphan designation.
