Drug Profile

Domofenogene zalfaparvovec - BioMarin Pharmaceutical

Alternative Names: BMN-307

Latest Information Update: 07 Sep 2021

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At a glance

Originator BioMarin Pharmaceutical
Class Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Phenylketonuria
New Molecular Entity No

Highest Development Phases

Phase I/II Phenylketonuria

Most Recent Events

05 Sep 2021 The USFDA places clinical hold on the phase I/II PHEARLESS study for Phenylketonuria in USA (NCT04480567) (EudraCT2019-001878-28)
05 Sep 2021 Safety data from a preclinical trial in Phenylketonuria released by BioMarin
05 Sep 2021 BioMarin suspends enrolment in the phase I/II PHEARLESS trial for Phenylketonuria (In adolescents, In adults) in USA and the UK (IV, Infusion) (NCT04480567) (EudraCT2019-001878-28)

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