BioMarin’s CTA for mucopolysaccharidosis drug accepted by MHRA

BioMarin Pharmaceutical has reported that its clinical trial application, or CTA, for BMN 110 or N-acetylgalactosamine 6-sulfatase, intended for the treatment of the lysosomal storage disorder mucopolysaccharidosis type IVA, or Morquio A syndrome, has been accepted by the UK Medicines and Healthcare Products Regulatory Agency or MHRA.

BioMarin expects to initiate a Phase I/II clinical trial in the next few weeks. The Phase I/II study is designed as an open-label, within-patient dose escalation trial in approximately 20 patients followed by a treatment continuation phase.

During the dose escalation phase of the study, subjects will receive weekly intravenous infusions of BMN 110 in three consecutive 12-week dosing intervals. The objectives of the Phase I/II study will be to evaluate safety, pharmacokinetics, pharmacodynamics and to identify the optimal dose of N-acetylgalactosamine 6-sulfatase (GALNS) for future studies.

Jean-Jacques Bienaime, CEO of BioMarin, said: There are a significant number of untreated Morquio patients in clinics around the world who are very anxious to receive a new treatment option. We are moving forward expeditiously with our GALNS program in the hope of bringing those patients the much-needed treatment they deserve.

BioMarin has successfully advanced two enzyme replacement therapies from investigational new drug filing to FDA approval in approximately five years each and we plan to leverage our clinical, manufacturing and regulatory expertise to develop this treatment for Morquio A syndrome.