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news.BioMarin Pharmaceutical has submitted a clinical trial application (CTA) with the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for BMN-190 for the treatment of Batten disease.
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The investigational recombinant human tripeptidyl peptidase 1, BMN-190, is being developed as a medication for a form of Batten disease called neuronal ceroid lipofuscinosis type 2 (NCL-2).
The company anticipates commencement of NCL-2 patient enrollment in 2013 in a Phase 1/2 trial.
BioMarin executive vice president and chief medical officer Hank Fuchs said, "We hope to leverage our expertise in enzyme replacement therapy development to advance this program and bring a therapeutic option to patients suffering from NCL-2."
Evaluation of BMN-190’s safety and tolerability in addition to efficacy are the primary objectives of the open-label, dose-escalation study.
Secondary objectives include investigating the impact of treatment on brain atrophy against NCL-2 natural history besides characterizing the pharmacokinetics and immunogenicity.