FDA review backs Cornerstone's new heart drug - with a hitch

Near the beginning of the year Cornerstone Therapeutics ($CRTX) snapped up Cardiokine and its late-stage heart drug lixivaptan for a mere $1 million down and $147 million in back-ended milestones. Today the investors at Cardiokine took a big step forward in their quest to get that back-end payoff as an FDA staffer recommended approval of lixivaptan, with a significant caveat on its use.

The drug is designed to treat hyponatremia, a condition characterized by low sodium levels in the blood which can trigger some severe physical reactions, such as the swelling of the brain and respiratory problems, which can be potentially fatal. Six million Americans suffer from the ailment, which is linked to congestive heart failure and erratic chemical syndromes spurred by syndrome of inappropriate antidiuretic hormone secretion.

Now, two days ahead of an expert panel review, FDA reviewer Nancy Xu issued a report which concludes that lixivaptan "was associated with a significant increase in serum sodium concentrations compared to placebo." And while she thinks the outside experts should provide a thumb's up for the drug, an elevated risk of death seen in the drug arm--not firmly linked to the drug itself--spurred Xu to recommend against using it in patients "requiring intervention to raise sodium concentration urgently to prevent or to treat serious neurological symptoms."

"There is currently insufficient information about the drug to determine whether the product is safe for use," noted Xu. 

- here's the FDA report (PDF)
- read the Reuters story