Type of information: Acceptation for review of a NDA
Product name: pegvaliase
Compound: PEGylated recombinant phenylalanine ammonia lyase enzyme
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases
- enzyme replacement therapy. PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase or ‘PAL’, pegvaliase) is an investigational drug that substitutes for the PAL enzyme in phenylketonuria (PKU) with the PEGylated version of the enzyme phenylalanine lyase, to break down phenylalanine. It is being developed as a potential treatment for adults with inadequately controlled blood phenylalanine levels.
Company: Biomarin Pharmaceutical (USA - CA)
- • On August 29, 2017, BioMarin Pharmaceutical announced that the FDA has accepted for Priority Review the Biologics License Application (BLA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, to reduce blood phenylalanine (Phe) levels in adult patients with phenylketonuria who have uncontrolled blood Phe levels on existing management. The PDUFA action date is February 28, 2018. However, the FDA has requested additional Chemistry, Manufacturing, and Controls (CMC) information, which the company expects, when submitted, will be classified as a major amendment and result in a three month extension of the PDUFA date. The Agency has not informed the company whether an advisory committee meeting to discuss the application will be needed.
- • On June 30, 2017, BioMarin Pharmaceutical announced that the company submitted a Biologics License Application (BLA) to the FDA for pegvaliase to reduce blood phenylalanine (Phe) levels in adult patients with PKU who have uncontrolled blood Phe levels on existing management. Following receipt of the BLA, the FDA conducts an initial assessment of the application to determine its fileability. The FDA typically notifies the applicant of their filing decision and planned Prescription Drug User Fee Act (PDUFA) action date within 60 to 74 days after receipt of the application. The company also intends to submit an application for registration in the European Union (EU) by year end 2017.
Submission of marketing authorization application USA : 2017-06-30
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: