A 'breakthrough' PD-1? From China? FDA says why not for rare cancer
PD-(L)1 inhibitors may be the standard of care and the cornerstone therapy for a number of cancers, but in other niches, the FDA is still happy to consider them a breakthrough.
Late Thursday — and early Friday in China — regulators granted the breakthrough designation to Junshi Biosciences. It’s a first for an anti-PD-1 from China, said the Shanghai-based, Hong Kong-listed biotech, which also won the domestic race by nabbing the first Chinese checkpoint OK back in December 2018.
A secondary listing on the STAR market in Shanghai, the formation of a joint venture focused on a CD39 drug and a Covid-19 antibody program partnered with Eli Lilly have kept Junshi execs busy. But with the FDA’s help they can move even faster in this particular indication.
First approved for the second line metastatic melanoma as Tuoyi, toripalimab’s BTD covers its application in nasopharyngeal carcinoma.
“Junshi prioritizes cancer types with high prevalence in China such as nasopharyngeal carcinoma, lung and liver cancer, but we found that great unmet medical needs also exist in other countries where patients are endangered by this deadly disease,” CEO Ning Li said in a statement.
By its count, there were 129,000 new cases of the tumor, which grows in the mucosal epithelium of the nasopharynx, worldwide in 2018. Although most patients can be cured early, few are diagnosed in that stage, meaning most patients don’t start treatment until the cancer has metastasized.
In a Phase II open-label pivotal study involving 190 patients with advanced cases who have failed systemic treatment, the drug met the primary endpoint on overall response rate.
Junshi submitted a supplemental NDA in China based on those data earlier this year, and a Phase III study is ongoing testing toripalimab plus chemotherapy (versus chemo alone) in the frontline setting for recurrent or metastatic nasopharyngeal carcinoma.
The FDA boosted toripalimab’s prospects in the US by issuing an orphan drug designation — conferring the perks that come with it — this March for mucosal melanoma in combination with Pfizer’s Inlyta. Another ODD followed for nasopharyngeal carcinoma in May.
Focusing on relatively small patient populations was an intentional strategy for Junshi, according to Li, who spent 13 years in the FDA as an oncology reviewer.
Pricing is another key part of the game plan, Li said in an interview with Loncar Funds last year — highlighting many predictions that the crowding out of the PD-(L)1 market could lead to commoditization and much lower prices. Even factoring in Merck’s China discount, Junshi’s Tuoyi costs just a third of Keytruda’s price at $29,800 per year. But Li suggested that the landscape in China may not be directly translatable to the US.
The lower price in China is proportionate with the lower cost of clinical development and manufacturing, he said, and aligns with the main goal of getting on the national drug reimbursement list:
The overall strategy for the price setting is that we are shooting to get onto the national reimbursement program as fast as possible. 200,000 renminbi is almost three to four years of the average household income in China even in the rich cities like Shanghai and Beijing. This market is not like the United States. The out of pocket expense in the United States is less because either insurance or Medicare or Medicaid will pay a lot of it. So the comparison between pricing in the United States and China is not comparable because the out of pocket part is key here. If you don’t get on the national reimbursement list in China, even if the drug is one tenth of U.S. price, it does not make any sense because the affordability isn’t there given the average income level in China.