BioMarin Pharmaceutical Inc. (BMRN: Quote) announced that it has submitted Biologics License Application or BLA to the U.S. Food and Drug Administration or FDA for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.
The company said it intends to submit an application for registration in the European Union or EU by the end of April 2013.
Mucopolysaccharidosis IVA is a disease characterized by deficient activity of N-acetylgalactosamine-6-sulfatase or GALNS causing excessive lysosomal storage of glycosaminoglycans such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease.
In a separate press release, BioMarin Pharmaceutical announced that it has submitted a Clinical Trial Application or CTA with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN-190, a recombinant human tripeptidyl peptidase 1 (rhTPP1) for the treatment of patients with neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten disease. The company expects to start enrolling patients in a Phase 1/2 trial in mid-2013.
The Phase 1/2 study is an open-label, dose-escalation study in patients with NCL-2. The primary objectives are to evaluate the safety and tolerability of BMN-190 and to evaluate effectiveness using an NCL-2-specific rating scale score in comparison with natural history data. Secondary objectives are to evaluate the impact of treatment on brain atrophy in comparison with NCL-2 natural history, and to characterize the pharmacokinetics and immunogenicity.
The study will enroll approximately 22 subjects worldwide for a treatment duration of 48 weeks.
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by RTT Staff Writer
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