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 30 percent to a life-high.
Analysts said the trial data was strong enough for an approval, especially given the lack of treatment options for the disorder.
"Given this is the largest Phase III enzyme replacement therapy conducted to-date, being statistically significant on the primary endpoint should strongly support approval," RBC Capital Markets analyst Michael Yee said in a note.
BioMarin, which has been repeatedly named as one of the most promising biotechnology companies and a potential takeover target for the Big Pharma, now plans to submit marketing applications for the drug GALNS, starting from the first quarter of 2013.
"With an estimated 3,000 GALNS patients worldwide, we believe this represents a $500 million to $800 million opportunity for BioMarin," Cowen and Co analyst Phil Nadeau wrote in a note.
He expects the drug to have sales of $75 million in 2014.
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.
"Our consultants have said that given the unmet need in (the disorder) the FDA could approve the drug even if the statistics in the primary endpoint were missed," Nadeau said.
However, patients getting the same dose of GALNS every other week, did not show any meaningful or statistically significant change compared to the placebo.
"The every other week dosing schedule was a 'long shot,' which if it had worked, would increase patient convenience," Yee said.
"All GALNS trials conducted prior to Phase III dosed in patients on a weekly schedule, so expectations for (the every other week) arm working were very low."
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. There is currently no approved treatment for MPS IVA.
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, which has a market capitalization of $4.63 billion according to Thomson Reuters data, 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 rose to $48.64 on the Nasdaq in heavy trading on Monday morning. More than 5.6 million shares changed hands by 1020 ET — double their 10-day moving average volume.
(Reporting by Esha Dey in Bangalore; Editing by Joyjeet Das)