Givosiran, which has been developed to be subcutaneously administered, targets aminolevulinic acid synthase 1 (ALAS1).
According to Alnylam, monthly administration of the investigational RNAi therapeutic can potentially reduce induced liver ALAS1 levels in a sustained manner by a significant extent. Owing to this capability, the investigational RNAi therapeutic can bring down neurotoxic heme intermediates, aminolevulinic acid (ALA) and porphobilinogen (PBG), towards normal levels.
FDA’s priority review designation is given to drugs that are determined by the regulator to have the potential to offer significant improvements in the treatment, prevention or diagnosis of a serious disease. The regulator’s goal is to take action within six months under a priority review compared to 10 months timeframe it has under standard review.
Givosiran has been set an action date of 4 February 2020 by the FDA under the Prescription Drug User Fee Act (PDUFA).
Alnylam said that the regulator has indicated that currently it is not planning an advisory committee meeting as part of the review of the new drug application (NDA) it had accepted for the drug.
The company also said that it has submitted the marketing authorisation application (MAA) for givosiran to the European Medicines Agency (EMA). Previously, the investigational RNAi therapeutic was granted an accelerated assessment by the European regulator, which is awarded to drugs considered to be of major public health interest and therapeutic innovation.
Accelerated assessment of the EMA potentially reduces the regulator’s evaluation time by 60 days from 210 to 150.
Previously, givosiran also secured breakthrough therapy designation and orphan drug designation in the US in addition to priority medicines (PRIME) designation from the EMA and orphan drug designation in the European Union.
Givosiran’s safety and efficacy were assessed in the ENVISION phase 3 trial which yielded positive results that are yet to be evaluated by the FDA, the EMA or any other health authority, said Alnylam.