Onyx nailed an FDA approval today for the multiple myeloma drug Kyprolis (carfilzomib), overcoming the naysayers who were skeptical that the agency would hand out an accelerated OK for a new drug on just mid-stage data drawn from a single-arm study. And the approval opens the door to a rich payout to the investors who backed the early development of the drug.
The agency made clear that this initial approval is restricted to patients who had already failed two therapies, making it a last-ditch option for many. Nevertheless, it will also likely raise expectations among other drug developers who are hoping to win quick approvals like this, where regulators will base their initial decision based on biomarker data rather than classic survival goals.
Onyx announced that it will charge close to $10,000 a month for the treatment, leaving payers facing a charge of about $40,000 for a full course of treatment.
"The approval of Kyprolis provides a treatment option to patients with multiple myeloma whose disease has progressed despite use of available therapies," said the FDA's Richard Pazdur. "We are encouraged by the continued progress in the development of drugs for multiple myeloma over the past decade, offering improved treatment of this disease."
"I would have to give the FDA a lot of credit for thoroughly reviewing this drug," Onyx CEO Tony Coles tells FierceBiotech. "For me, it's a remarkable example of how the FDA can work with a company."
The approval hands Onyx ($ONXX) a key win at a time analysts have tapped the company as a likely takeover target, with its Big Pharma partner Bayer a likely bidder. It also hands the Proteolix team, which created carfilzomib and sold it to Onyx in 2009 for $276 million in cash and $535 million in milestones, a big payday.
The original milestones included $170 million for an accelerated approval of the drug. But Coles says that the full $170 million would have been due based on an earlier approval. Today's action clears the way for an $80 million payment under the renegotiated terms of the deal.
Onyx is on a roll this summer. Regorafenib is up for an accelerated review at the FDA for colorectal cancer. Onyx won a significant slice of the royalties on the drug from Bayer recently, which only served to heat up buzz about a possible Bayer buyout.
Coles notes that he always avoids getting overconfident ahead of any regulatory decision. But, he adds, "the clinical data for regorafenib is quite stunning."
Onyx has a variety of late-stage studies for Kyprolis either under way or in planning. One of those is a trial with about 800 patients which Coles believes can provide the data needed for full U.S. and European approval. There are also two head-to-head studies with Velcade in the works,including one in a second-line setting among patients who have failed one to three therapies.
The approval today won't come as a surprise to industry observers who followed last month's advisory committee review. By a vote of 11 to 0--with one abstention--the panel of experts concluded that a host of severe risks, the death of 5 patients and a questionable benefit did not outweigh the therapy's potential to help a group of cancer victims who had run out of options.
- here's the press release