The Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the FDA voted to approve Biomarin's investigational drug Vimizim for the treatment of Morquio A syndrome, an ultra-rare lysosomal storage disorder also called Mucopolysaccharidosis Type IVA (MPS IVA). According to Biomarin, Vimizim is the first and only enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome, a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS).
Of the 21 panel members, 19 voted in favor of approval of Vimizim for use in all MPS IVA patients, one voted in favor of approval for a subgroup of MPS IVA patients, and one panel member voted not to recommend approval. The FDA assigned a PDUFA action date of February 28, 2014, for completion of its review of the BLA for Vimizim. It's highly expected that the FDA will approve Vimizim after its PDUFA date.
Meanwhile, on the cancer frontlines, Biomarin have entered a research collaboration with Myriad Genetics that will use Myriad's HRD (Homologous Recombination Deficiency) test to identify tumor types that may be sensitive to BioMarin's investigational product candidate, BMN-673. Myriad's HRD test can detect when a tumor has lost the ability to repair double-stranded DNA breaks, resulting in increased susceptibility to DNA-damaging drugs.
"The biology of cancer is complicated, and while the analysis of multiple gene targets may identify a subset of patients who will respond to PARP inhibitors, we need a more comprehensive test capable of identifying all patients who may benefit from treatment with PARP inhibitors or DNA damaging agents," said Myriad CSO Jerry Lanchbury, Ph.D., in a statement. "While it is impossible to predict all of the genetic causes of DNA repair deficiency, our HRD test solves this problem by measuring the ultimate effect, which presents as a DNA scar."
This is actually the second research collaboration between Myriad and BioMarin, as back in October, BioMarin announced it would use Myriad's BRACAnalysis® test in connection with its Phase III study of BMN-673 for breast cancer.