Anti-PD-1 cancer star nivolumab wins world's first regulatory approval

The anti-PD-1 immuno-oncology drug star nivolumab has won the race to the world's first regulatory checkered flag for an approval. Ono Pharmaceutical, which picked up the Japanese rights to the drug in a collaboration deal with Medarex back in 2005, says regulators in the Asian country approved nivolumab for sale as a treatment for unresectable melanoma.

Ono now says that it plans to make the closely-watched drug available to patients for free as it awaits a listing on the national health insurance price list. The company adds that it's planning to follow up with additional applications for other cancer uses, with plans to launch a post-marketing study on the drug's effects among patients prescribed the cancer treatment.

The immune checkpoint inhibitor will be sold in Japan as Opdivo. And it's not the only cutting-edge therapy to get a pioneering OK in Japan. Bristol-Myers today reported that Japanese regulators have approved a combo hep C therapy - packaging  Daklinza and Sunvepra for genotype 1 patients. This is the first of a number of new cocktails being developed for hep C that will exclude interferon and Ribavirin.

Medarex was bought out by Bristol-Myers Squibb ($BMY), leaving it with a cutting-edge therapy designed to disable the cloaking mechanism that cancer cells use to remain hidden from the immune system. Bristol-Myers owns all rights to the drug outside of Japan, Korea and Taiwan. Nivolumab is one of the leading IO drugs in development, racing against Merck's ($MRK) pembrolizumab (MK-3475), which is engaged in a rolling submission in the U.S. and is under review in Europe.

Merck may be leading the race for the first major market approval, but a number of analysts give Bristol-Myers the edge in developing the drug most likely to gain the biggest market share. This sector is likely to be worth billions of dollars, with combination drugs and monotherapies promising to be a game changer in treating cancer. Roche is also among the leaders in the field, with AstraZeneca coming up from behind with its own ambitious development plans.

Just two weeks ago Bristol-Myers, which has poured hundreds of millions of dollars into its development program, announced that it hit the brakes earlier than planned on a late-stage study of nivolumab after the monitoring committee overseeing interim data concluded that the PD-1 drug provided a clear survival benefit over dacarbazine for BRAF wild-type advanced melanoma when used as a frontline therapy.

"We anticipate that nivolumab will be the sales-leading agent among immunotherapies," noted Decision Resources analyst Khurram Nawaz in a recent piece for PMLive in the UK. "However, it will face direct and intense competition from other anti-PD1/PDL1 agents--notably from pembrolizumab in malignant melanoma and NSCLC--and, to a lesser extent, from MPDL-3280A in NSCL."

Gyo Sagara

"We are delighted to obtain a manufacturing and marketing approval as a drug targeting PD-1, which receives a lot of attention in tumor immunity, for the first time in the world." said Gyo Sagara, president of Ono. "Ono would like to obtain approvals for additional indications on ongoing development for other cancers to bring many patients OPDIVO as soon as possible."

- here's the release (PDF)
- here's the story on the hep C approval (sub. req.)