UPDATED: Bayer, Onyx nab speedy FDA OK for regorafenib, prices at $9,350

Only three months after nabbing FDA fast-track review status for regorafenib, Bayer's top cancer prospect, the agency handed out a speedy approval this afternoon as a new treatment for metastatic colorectal cancer. The double-quick regulatory OK completes Bayer's quick sprint through the late-stage development process, paving the way to commercialization work expected to generate peak sales of $1.25 billion a year or more.

The newly-approved drug will be marketed as Stivarga. And the FDA's release was careful to note that the agency's approval comes more than a month ahead of the agency's accelerated PDUFA date.

In keeping with the speedy regulatory decision, Bayer officials tell FierceBiotech that the drug will be available on Friday, with a wholesale acquisition cost of $9,350 per 28 day cycle. Shannon Campbell, the vice president of oncology for Bayer, says the company's patient support program will assist anyone who can't afford the treatment.

"The approval of Stivarga reflects Bayer's commitment to confronting the challenges of difficult-to-treat cancers," said Pamela A. Cyrus, MD,  the head of U.S. Medical Affairs, Bayer HealthCare Pharmaceuticals.  "Patients with metastatic colorectal cancer whose disease has returned after treatment will now have a new option that has been shown to prolong survival and keep the cancer from progressing."

Cyrus says that 2012 marks a major turning point in establishing Bayer as a powerhouse cancer drug developer. In addition to expanding on the Stivarga approval with additional cancer studies, both as a monotherapy and in combination with other drugs, Bayer also is wrapping up the pivotal work on Alpharadin, its radium-223 dicloride program for prostate cancer wich is slated to head to regulators later this year. Bayer licensed the program from Algeta in 2009 with an $800 million pact. And there's more ahead. Bayer filed applications in late August looking for regulatory approval to market Stivarga for gastrointestinal tumors. 

The approval for regorafenib, a multi-kinase inhibitor sometimes referred to as "son of Nexavar," also marks the start of a new revenue stream for Onyx Pharmaceuticals ($ONXX), which gained $160 million upfront and a commitment of a hefty 20% royalty stream from Bayer after filing a lawsuit to pursue its ownership claims. The two companies have been teamed for years on Nexavar and Onyx claimed that Bayer scientists had quietly gone to work developing a close therapeutic companion of their partnered drug. Regardless, the two companies made up after the legal scrap and are still married at the hip on the cancer front, so much so that speculation over a potential Bayer buyout has percolated for months. The buyout buzz can only grow louder with today's approval news.

The excitement about regorafenib's prospects has a lot to do with its effectiveness in Phase III colorectal cancer studies. Investigators reported in early January that the treatment improved the median overall survival rate of metastatic patients by 29%, with the drug arm demonstrating a 6.4 month overall survival rate while advanced patients who had already failed standard therapy survived a median average of 5 months on a placebo. Metastatic patients in the drug arm also experienced high rates of fatigue, skin reactions and diarrhea, according to Bayer.

For Bayer, the approval marks an eagerly sought after expansion of its cancer drug franchise, which so far has relied on Nexavar, which earned $1.08 billion worldwide last year. 

- here's the FDA release

Timeline on regorafenib (Stivarga) development

May 15, 2009 
Onyx Pharmaceuticals files a complaint in the U.S. District Court for Northern California alleging that the compound regorafenib can be distinguished from Nexavar by only a single atom. Report

May 7 2010
Germany's Bayer Schering Pharma marches ahead into a Phase III study of regorafenib for colorectal cancer, gambling it can successfully fend off a legal attack from its U.S. partner Onyx. Onyx filed suit against Bayer in 2009 saying the new cancer drug can be distinguished from Nexavar by only a single atom. Report

February 4, 2011
Regorafenib is granted orphan drug status for the treatment of patients with gastrointestinal stromal tumors by the FDA. Report

May 10, 2011
Bayer Healthcare receives the FDA's fast track designation for regorafenib. This comes after the company initiated a randomized, double-blind, placebo-controlled Phase III study of the drug in January. Report

October 12, 2011
Bayer and Onyx patch up the long-running dispute over who owns the rights to a Phase III cancer drug. Bayer retains control of regorafenib's development and commercialization efforts, while Onyx gets a hefty $160 million check and a promise of up to $15 million more, a 20% royalty share on worldwide sales and rights to co-promote the treatment in the U.S. Report | See the 8-K

October 26, 2011
Investigators say regorafenib hit its primary endpoint for improving overall survival among metastatic patients, triggering a move to unblind the study and offer the drug to patients in the placebo arm. Report

December 20, 2011
Bayer predicts that four of its late-stage programs--including regorafenib--are likely blockbusters. The company commits 2 billion euros, or two thirds of its total R&D budget, for drug research in 2012. Report

January 18, 2012
Investigators report that regorafenib improved the median overall survival rate of metastatic patients by 29%, with the drug arm demonstrating a 6.4 month overall survival rate while advanced patients who had already failed standard therapy survived a median average of 5 months on a placebo. Report

April 3, 2012
In a trial, Dana Farber's George Demetri studied regorafenib in advanced cancer patients who weren't responding to existing medications. Bayer says the treatment hit its primary endpoint on progression-free survival. Report

June 3, 2012
Regorafenib offers late-stage data which show the drug put the brakes on stomach cancer among a group of patients who had failed two standards of care. The median progression-free survival time for the regorafenib arm of the study was 4.8 months compared to only 0.9 months in the placebo arm. Report

June 28, 2012
FDA gives regorafenib promising treatment priority review status, which is projected to shave four months off the standard 10-month review. That lands the deadline for a marketing decision in late October. Report

August 30, 2012
Bayer files for approval of regorafenib to treat gastrointestinal cancer. Report

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