Oncology Dominates As China Drug, Biologic Approvals Surge
Executive Summary
China saw a 150% increase in new drug approval applications in 2018, with oncology taking the lion's share of investigational new drug filings, notes a newly released annual report from China’s Center for Drug Evaluation. Meanwhile Darzalex has become the latest foreign drug to be approved.
In a bid to bring more cancer and rare disease treatments to the country, China has significantly increased approvals related to novel therapies.
Throughout 2018, a total of 25 Category 1.1 new drug approval (NDA) applications were accepted by regulators, an increase of 150%. Among these, small molecule drugs accounted for 16, up by 100%, and large molecule biologics for nine, surging by 450%, notes a new annual review released by the National Medical Products Administration’s drug review wing, the Center for Drug Evaluation (CDE).
Investigational new drug (IND) filings also rose, among which oncology took the absolute lead. Of a total of 457 INDs filed by both domestic and international applicants, anticancers was the leading therapeutic area, followed by endocrinology and gastrointestinal and cardiovascular medicines. It was a similar situation for biologics, where 70% of the INDs were for cancer. [Click to see the report: Chinese language]
Oncology Remains Front And Center
The CDE annual report report highlights a heavy focus on oncology and increasing attention on rare disease treatments in China.
Oncology accounted for 70% of new biologics approvals, which included four PD-1 checkpoint inhibitors, two of which were from multinationals: Bristol-Myers Squibb Co.’s Opdivo (nivolumab) and Merck & Co. Inc.’s Keytruda (pembrolizumab). The other two were Tuoyi (toripalimab) from Junshi Pharma and Tyvyt (sintilimab) from Innovent Biologics Inc.
In fact domestic drug makers appear to be catching up fast, securing several NDAs for their novel cancer treatments. These included Jiangsu Hengrui Medicine Co. Ltd. with Iruini (pyrotinib) and Hutchison MediPharma Ltd. with Elunate (fruquintinib), along with anlotinib from Chiatai Tianqing, a part of China Biologic Products Inc.
Meanwhile, Roche/Chugai Pharmaceutical Co. Ltd. ’s Alecensa (alectinib) also gained approval in China for ALK mutation-positive patients with non-small cell lung cancer.
Global First Approvals
As China speeds up the introduction of novel therapies to the country, regulators are eagerly highlighting several world-first new drug approvals granted to products filed first in China.
One of these was FibroGen Inc.’s roxadustat, for anemia associated with chronic kidney disease (CKD) and patients on dialysis. It was the first approval worldwide for the first-in-class hypoxia-inducible factor prolyl hydroxylase inhibitor, which is marketed by AstraZeneca PLC in China.
With an estimated 120 million sufferers, the country has one of the largest CKD patient populations in the world, and the associated burden of dialysis is increasing. (Also see "First Approval For AZ's Roxadustat With China Green Light " - Scrip, 18 Dec, 2018.)
Similarly, domestic biotech startup Nanjing Pioneer gained the first approval worldwide for albuvirtide, its peptide therapy for HIV infections. The long-acting injection is billed as China’s first domestically-developed drug for combination use in patients who have been on antiviral treatment.
Orphans Getting More Attention
The annual report also highlighted the Chinese regulator’s increasing focus on rare disease treatments. Last May, the National Health Commission in China released the first-ever Rare Diseases Catalog, ushering in a new chapter for orphan drugs development in the country. Orphan drugs have also been selected for priority reviews and clinical trials waivers as long as they have already been approved in the US, EU and Japan.
Biogen Inc.’s spinal muscular atrophy treatment Spinraza (nusinersen) became the first approved treatment for the devastating neuromuscular degenerative condition following a speedy review and without local clinical trials. Alexion Pharmaceuticals Inc.’s Soliris (eculizumab) also gained nods for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, both included by the commission in the list of 122 designated rare conditions.
Also approved during the year were Roche/Chugai’s Hemlibra (emicizumab) for hemophilia A and Sanofi’s Aubagio (teriflunomide) for relapsing multiple sclerosis, and other notable orphan clearances included Amgen Inc.’s PCSK9 inhibitor Rapatha (evolumab) for rare heterozygous familial hypercholesterolemia, and Actelion Pharmaceuticals Ltd.'s Uptravi (selexipag) for pulmonary arterial hypertension. China has few available treatment options for the latter disease, a severe and life-threatening condition, noted the CDE report.
J&J Scores Darzalex Approval
Other notable approvals included Johnson & Johnson’s antibody Darzalex (daratumumab) as a single therapy for relapsed and intractable multiple myeloma; the product was also the first CD38-targeting antibody to gain approval in China. Darzalex was originally developed by Genmab AS of Denmark, and J&J has licensed exclusive rights in the US and China among other territories.
Multiple myeloma has overtaken acute leukemia/lymphoma as the second mostly common hematological malignancy in the country, especially among middle aged and older people, and mortality rates are high due to frequent recurrence and relapse.
Several companies have been active in China with new therapies, including Takeda Pharmaceutical Co. Ltd. and Celgene Corp.. Takeda’s Ninlaro (ixazomib) gained the green light in China last April as the first approved oral-only treatment for multiple myeloma, while Celgene’s Revlimid (lenalidomide) was cleared last February in combination with dexamethasone for previously untreated, transplant-ineligible multiple myeloma patients.
Marketed in China by BeiGene Ltd. under a previously-agreed deal, Revlimid has however seen only a slow uptake in China despite its strong showing in the US.
Last May, the US FDA approved Darzalex in combination with Velcade (bortezomib), a proteasome inhibitor, for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant. “As the first CD38-targeting antibody approved in China, the novel treatment is expected to redefine the multiple myeloma treatment in China,” J&J said in a statement.
The next step the product will face is market access, given that there is no immediate reimbursement coverage for newly approved drugs in China.