After PhIII odyssey, the FDA finally gives Medicines Co. a thumbs-up for cangrelor

After close to a decade of late-stage studies and stinging setbacks, The Medicines Co. may finally be on the verge of seeing its blood-thinner cangrelor make it to the market. But the late comeback may be far too little and too late to do much good as sales of its franchise therapy Angiomax drop ahead of what is expected to be brutal generic competition later this year.

Just a year ago the FDA and its outside experts keelhauled The Medicines Co.'s application for cangrelor. But regulators looked over the company's new, simplified pitch for cangrelor and decided that it appears acceptable for use as a second-line therapy in a narrow indication.

"Our conclusions regarding the benefit risk of cangrelor remain the same as in our original NDA review," the in-house reviewers noted in an analysis released today. "The benefit of cangrelor compared to clopidogrel (Plavix) is small, but the risk is smaller. Treating 171 patients prevents one clinically meaningful periprocedural MI. In comparison, treating 1106 patients causes one GUSTO severe bleed … Using a less severe bleed to assess benefit risk, such as a GUSTO moderate or TIMI minor bleed still favors the use of cangrelor. We recommend that cangrelor be approved as an adjunct to PCI for the reduction in risk of periprocedural ischemic complications including myocardial infarction and stent thrombosis in patients in whom treatment with an oral P2Y12 platelet inhibitor prior to PCI is not feasible and when glycoprotein IIb/IIIa receptor antagonists are not anticipated to be used."

The company's shares ($MDCO) surged 6% on the news this morning.

The cautious but clear thumbs up from the FDA came after the agency and independent observers blasted the design and execution of the Phase III studies for cangrelor. The first two Phase III studies actually failed, but in a major followup study involving close to 11,000 patients, The Medicines Co. trumpeted data outlining a reduction in serious adverse events when compared with Plavix.

The Medicines Co. has spent tens, if not hundreds, of millions of dollars on this drug, with a Phase III program that dates back 9 years. The first Phase III effort ended in an embarrassing failure back in 2009, which prompted a halt, followed by a followup trial and the initial setback at the FDA.

Adding time to the development process has not been kind to The Medicines Co. At one point analysts expected that cangrelor could reap about $400 million a year. But with the delay and a decision by the biotech to drop an effort to use the drug in a bridge indication, the peak sales estimates have been shrinking.

"This drug was SUPER complementary to MDCO's existing Angiomax franchise … but if Angiomax goes away in mid-2015, the incremental impact of cangrelor has become less important," notes Evercore ISI's Umer Raffat. He pegs peak sales at $100 million, "with a fairly healthy penetration estimate."

The FDA's cautious acceptance of cangrelor comes three months after the EU provided its sanction for the therapy. Generic versions of Angiomax, meanwhile, are expected as early as mid-June.

The FDA's Cardiovascular and Renal Drugs Advisory Committee meets on Wednesday.

- here's the FDA review

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