BioMarin skates through FDA panel for rare disease drug

The Bay Area's BioMarin Pharmaceutical ($BMRN) had an easy time at the FDA, winning the support of an agency panel for its latest rare-disease treatment and feeding expectations for a near-term approval.

A committee of agency advisers voted 20-1 in favor of approval for Vimizim, which treats the rare enzyme deficiency Morquio A. The majority of panelists said BioMarin's drug demonstrated convincing safety and efficacy, and their recommendation, while nonbinding, likely spells a full approval once BioMarin's PDUFA date comes around in February.

Morquio A affects about 3,000 patients and leads to a host of skeletal and bone disorders that can seriously restrict mobility. Vimizim, in its pivotal trials, helped patients walk 22.5 meters beyond what they could manage on placebo, and despite a lukewarm reception from an FDA staff reviewer, that benefit was enough to sway the agency's advisers and will likely carry BioMarin's drug through to market.

"I am very pleased with the outcome of today's panel vote and look forward to continuing to work with the FDA to bring this much-needed therapy to these patients," BioMarin CEO Jean-Jacques Bienaimé said in a statement.

And, as has been the case for some time with BioMarin, any spot of good news rekindles long-standing M&A speculation. Now that trumped-up rumors of a $15 billion check from Roche ($RHHBY) have fizzled and a reported tire-kicking from GlaxoSmithKline ($GSK) amounted to nothing, BioMarin is more valuable than ever and is well on its way to getting a 5th drug to market. Biotechs around the world have demonstrated how lucrative rare-disease drug can be, as payers are often willing to pick up hefty checks for effective treatments, and Vimizim could be BioMarin's ticket to a takeover.

Last week, Shire ($SHPG) signed a $4.2 billion deal to pick up the rare disease-focused ViroPharm, paying a steep premium on the promise of a deeper pipeline of promising drugs with small patient populations. Meanwhile, many members of the year's burgeoning class of biotech IPOs wooed investors with strong data for in-development rare-disease drugs.

BioMarin halted trading of its shares all day Tuesday but jumped about 5% to $70 after hours. The company is up roughly 13% since last week.

- read BioMarin's statement

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