(Reuters) – BioMarin Pharmaceutical Inc said a late-stage trial of its experimental genetic disorder drug met the main goal of improving patients’ walking ability when administered weekly, sending the company‘s shares up 25 percent in premarket trading.
The drug, called GALNS, however, did not show any improvement over a placebo when administered every other week.
The trial, named MOR-004, showed that weekly doses of the drug at 2 mg/kg improved the six-minute walk distance of patients by 22.5 meters over the placebo.
However, patients getting the same dose every other week did not show any meaningful or statistically significant change compared to the placebo.
The disorder – Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome – is a rare, inherited disorder caused by the deficiency of a particular enzyme, leading to skeletal dysplasia, short stature and joint abnormalities.
The drug was also tested in another extension trial that showed its benefits continued to improve with further dosage, BioMarin said in a statement.
GALNS was generally well-tolerated and adverse events were similar to those seen in the trials of other enzyme replacement therapies. There were no deaths and no patients withdrew from the study due to adverse events.
BioMarin now plans to submit marketing applications for the drug, starting from the first quarter of 2013.
The company currently has four drugs in the market, two of which are for treating other metabolic disorders caused by the absence of certain enzymes.
Shares of the company closed at $ 37.41 on the Nasdaq on Friday, and were trading at $ 47 before markets opened on Monday. (Reporting by Esha Dey in Bangalore; Editing by Joyjeet Das)
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