BMN307

Unassigned

New Medicines

Phenylketonuria in adults with PAH deficiency

Information

Advanced therapy medicinal product (ATMP)
BioMarin
BioMarin

Development and Regulatory status

Phase II Clinical Trials
None
Phase II Clinical Trials
Yes
Yes
Nov 20 · US FDA award fast-track status. [1]
Oct 19 · US FDA and EMA grant orphan designation for treatment of PKU. [1]

Category

Phenylketonuria is caused by mutations in the PAH gene. BMN307 consists of an AAV5 vector containing the gene that codes the phenylalanine hydroxylase (PAH) enzyme. Given as a single dose infusion.
One study estimated the prevalence per 10,000 live births in South East England was 1.14 among white, 0.11 among black and 0.29 among Asian ethnic groups [2].
Phenylketonuria in adults with PAH deficiency
Intravenous infusion

Trial or other data

Dec 19 · PI/II PHEARLESS open-label dose finding trial initiated (NCT04480567, n=100) to evaluate the safety and efficacy of a single dose of i.v. BMN307 in pts with PKU. Trial sites include the UK (University Hospital Birmingham NHS Foundation Trust) and USA. The study has an estimated primary completion date of Dec 2027. [1,3-5]