Drug regulators in China, US and Europe out of the gates in 2020 with a whole month’s worth of action.

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Slowly at first, then accelerating recently in its domestic and global footprint in drug research and regulation, China and its National Medical Products Administration (NMPA) has been on a tear. Last week, BeiGene Ltd. Co. (Beijing), the country’s jewel of a contemporary, rising-star, is back in the news. And there’s more.

Back on December 17, 2018, Junshi Biosciences Co. Ltd. (Shanghai) garnered the landmark approval for a made-in-China PD-(L)1 checkpoint inhibitor by the NMPA. Barely a year later, the fourth homegrown PD-1 is making its way to a market that has since seen several more firsts even while getting crowded, Endpoints reports.

The approval for BeiGene Co. Ltd.’s tislelizumab just before 2019 ended is also marking a new historic event: the first time a foreign partner will be producing the marketed drug under a reformed contract manufacturing regulatory system. Boehringer Ingelheim GmbH (Ingelheim DEU), a partner since 2013, will be supplying the PD-1 antibody for commercial use using a Shanghai facility.

“This is an important milestone, not only to ensure the supply of medicines for patients in China, but also for the rapidly emerging Chinese biopharmaceutical Research & Development landscape,” said BeiGene CEO John Oyler in a statement.

In 2014 China’s drug regulator began piloting a new Marketing Authorization Holder system under which drugmakers without manufacturing capabilities can still apply for marketing approval. This move opened up the option of enlisting contract manufacturers and signified a complete break from the previous system, where developers must have manufacturing authorization to market a drug.

While BeiGene does have its own biologics manufacturing plant in Guangzhou, it’s also signed a deal with Catalent Inc. (Somerset NJ) to produce its first commercial drug, zanubrutinib (dubbed Brukinsa in the US). SVB Leerink anticipates BeiGene will launch tislelizumab in early 2020. The initial indication is third-line relapsed/refractory Hodgkin’s lymphoma, familiar to anyone who’s been following Innovent’s Tyvyt or Jiangsu Hengrui’s camrelizumab. But BeiGene will get top-line Chinese data in non-small cell lung cancer later this year and is plotting supplemental OKs for urothelial cancer and hepatocellular carcinoma.

“This is BeiGene’s first internally discovered drug approved in China,” analysts noted. “The approval was based on results from a single-arm pivotal Phase 2 trial, in which tislelizumab demonstrated a 76.9% objective response rate (ORR) via independent review committee (IRC) assessment, including a 61.5% complete response (CR) rate.”

Leerink’s model estimates $85 million in 2020 China revenue for tislelizumab, partly from “meaningful off-label sales.”

And Pfizer Inc.’s (NYC) Prevnar 13 and Merck & Co.’s (Kenilworth NJ) HPV franchise Gardasil are the world’s best-selling vaccines, and China sales have been driving growth for both of them. But now, thanks to new approvals, they’re facing competition in that very market. Last week, the NMPA approved domestic drugmaker Walvax Biotechnology Co. Ltd.’s (Yuxi) 13-valent pneumococcal conjugate vaccine and local firm Innovax’s (Fujian) bivalent HPV vaccine Cecolin.

Two weeks after publishing successful Phase 3 trial data in the New England Journal of Medicine, Allergan PLC (Dublin IRL) has landed approval from the US Food and Drug Administration for their new acute migraine pain drug ubrogepant. The drug, branded Ubrelvy, is the latest in a series of migraine drug approvals that have begun to reshape an often debilitating ailment the FDA estimates affects 37 million Americans. It is also a significant win for Allergan, who has been racing against Eli Lilly & Co. and Biohaven Inc. to develop similar pills to alleviate migraine pain.

Elsewhere, the FDA approved AstraZeneca PLC (London) and Daiichi Sankyo Co. Ltd.’s (Tokyo) Enhertu (fam-trastuzumab deruxtecan-nxki) on Friday as a treatment for patients with inoperable or metastatic HER2-positive breast cancer who have already failed on at least two other treatments. AstraZeneca and Daiichi will jointly develop and sell the drug outside of Japan, where Daiichi holds exclusive rights.

Enhertu will likely launch in January at a per-patient cost of around $13,300 per month, SVB Leerink analyst Andrew Berens said in a note to investors. At that price, Enhertu could reach $68 million in sales in 2020, with a peak sales estimate of $2.5 billion, Berens said.

Last month, FDA experts voted–barely–that the agency should approve AstraZeneca PLC (London) and Merck & Co.’s (Kenilworth NJ) Lynparza in pancreatic cancer. But that small majority was enough to convince regulators to agree. The FDA approved the drug Friday as a maintenance treatment for patients with germline BRCA-mutated disease who’ve already received a round of platinum-based chemo.

The approval followed a priority review designation, awarded over the summer, and an orphan drug designation. With the approval, AstraZeneca and Merck add a third disease area to the drug’s resume, which already includes indications in ovarian and breast cancers. And, for the third time, Lynparza is the first of its class of PARP inhibitors to break into a cancer treatment classification.

Intra-Cellular Therapies Inc. (NYC) secured the FDA’s blessing for its antipsychotic drug, Caplyta, despite two late-stage schizophrenia studies that left much to be desired. But as is custom with most antipsychotics, trials that fall short do not necessarily deter a drug’s path to approval. Caplyta, known chemically as lumateperone (or ITI-007), is engineered to “selectively and simultaneously” modulate three neurotransmitter systems implicated in a range of mental illnesses: serotonin, dopamine, and glutamate.

Thirteen years after Correvio Pharma Corp. (Vancouver CAN) first tried–and failed–to gain an approval for the heart drug vernakalant, the FDA has handed them their latest Complete Response Letter (CRL). The agency spurned the little biotech–once called Cardiome–in the wake of a lopsided 11-2 vote against marketing approval. Subjected to a clinical hold at one stage, the atrial fibrillation drug has been under a dark safety cloud. The biotech, now trading in the penny stock bargain basement, has already put up the “for sale” sign, interested in any offers for their assets.

The FDA last Thursday announced restrictions on the sale and manufacturing of all flavors of e-cigarette pods except tobacco and menthol, says Modern Healthcare. FDA Commissioner Dr. Stephen Hahn said the policy was intended to limit youth e-cigarette use but maximize the potential benefit to adults trying to quit smoking combustible cigarettes. The flavor restrictions do not apply to tank vaping systems found at vape shops that HHS officials said are more often used by adults. The Trump administration in September indicated it wanted to ban all e-cigarette flavors besides tobacco.

Across the pond, the UK drug-pricing watchdog has again rebuffed Roche Holding AG (Basel CHE) and its cancer immunotherapy Tecentriq. The National Institute for Health and Care Excellence’s (NICE) latest filing is for extensive-stage small cell lung cancer patients. The regulator determined that although Tecentriq appeared to improve patient survival and quality of life in clinical trials, that data was uncertain and based on patients who were dissimilar to the average NHS patient. The benefits Roche had shown were not worth the drug’s on average £32,800 (roughly $43,300) price, NICE said. It’s the second recent price-based rejection for Roche.

Repeating some of the concerns the FDA underscored before handing its rejection, the UK’s cost-effectiveness watchdog NICE has also rejected Akcea Therapeutics Inc.’s (Boston MA) drug, volanesorsen. On Friday, NICE issued a draft recommendation rejecting the familial chylomicronaemia syndrome (FCS) drug, citing a myriad of reasons including uncertainty of its clinical evidence as well as price. The drug is engineered to work by diminishing the production of ApoC-III, a protein that regulates plasma triglycerides. FCS is caused by insufficient or impaired function of an enzyme responsible for breaking down triglycerides. It affects between 55 and 110 people in England.

Following its Brexit-related departure from London, the European Medicines Agency’s (EMA) staff has been reduced from 897 staffers to 775, EMA said Friday in an update from a board meeting in its new home in Amsterdam. Although an October EMA management board update said the agency’s staff numbers were down to 730, the uptick comes as a 2017 report said that UK experts made up 15% of the EMA’s expert base and conducted about 20% of its scientific work.

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