Chinese Innovative Drugs Secure a Wave of FDA Orphan Drug Designations in March 2026

　　【Pharmaceutical Network Industry DynamicsIn recent years, the research and development of innovative drugs in China has been showing a vigorous development trend, especially in the fields of oncology, metabolic diseases, and autoimmune diseases, where significant progress has been continuously achieved. Notably, influenced by this, the rare disease sector has also started to enter a period of accelerated development. It is reported that since March 2026, a large number of domestic pharmaceutical companies such as Changchun High & New Technology Industries (Group) Inc., AndBio, and CirCode have successively received orphan drug designations from the U.S. FDA for indications covering pancreatic cancer, achondroplasia, desmoid tumors, and other areas.
 

On March 24, Qilu Pharmaceutical's Class 1 new drug Claudin18.2/CD3 bispecific antibody QLS31905 received FDA orphan drug designation for the indication of pancreatic cancer. The drug works by simultaneously binding to Claudin18.2 on the surface of tumor cells and CD3 on the surface of T cells, precisely recruiting and activating the body's own immune cells to achieve targeted killing of tumor cells.
 

On the same day, GenSci, a subsidiary of Changchun High-Tech, also received notification from the U.S. FDA, granting orphan drug designation to GenSci128 tablets for the treatment of gastric cancer. GenSci128 tablets are a selective reactivation agent targeting the TP53 Y220C mutation and are classified as a new therapeutic chemical drug of Category 1. Previously, the drug had been approved for clinical trials in both China and the U.S. for locally advanced or metastatic solid tumors carrying the TP53 Y220C mutation.
 

On March 19, Abbisko Therapeutics announced that its self-developed highly selective small molecule FGFR2/3 inhibitor ABSK061 has been granted Orphan Drug Designation by the U.S. FDA for the treatment of achondroplasia (ACH). The drug has demonstrated significant target inhibition activity, favorable pharmacokinetic properties, and safety advantages in preclinical studies. In addition, the Phase II clinical study of ABSK061 for ACH pediatric patients aged 3-12 has completed the first patient dosing in China in December 2025, with preliminary data from this study expected to be released in the second half of 2026.
 

In mid-March, Huadong Medicine's self-developed ADC innovative drug, HDM2017 for injection, received orphan drug designation from the U.S. FDA for three indications: cholangiocarcinoma, gastric cancer, and pancreatic cancer. It is reported that HDM2017 for injection is a novel antibody-drug conjugate (ADC) targeting Cadherin 17 (LI-cadherin). Preclinical studies have shown that the drug has good druggability, safety, and efficacy; it demonstrated strong anti-tumor effects in pharmacodynamic models of colorectal cancer, pancreatic cancer, gastric cancer, and other target-positive cancers; and it exhibited good tolerability in animal tests.
 

On March 11, CirCode announced that its second core circular RNA innovative drug, HM2003 Injection, independently developed by the company, has officially received clinical trial approval from the U.S. FDA. Meanwhile, HM2003 has also been granted Pediatric Rare Disease Designation by the FDA for an undisclosed indication. It is reported that this product had previously obtained Orphan Drug Designation for the treatment of thromboangiitis obliterans.
 

On March 3, Elpiscience Biopharma officially announced that its self-developed CD39/TGF-β bispecific antibody ES014 has been granted Orphan Drug Designation by the U.S. FDA for the indication of desmoid tumors. Research results show that ES014 demonstrated good safety, with no dose-limiting toxicity (DLT) observed and most adverse events being mild.
 

On March 6, Puzhong discovered that its key product AMT-253 had received orphan drug designation from the U.S. FDA for the treatment of soft tissue sarcoma. AMT-253 is an MUC18-targeted ADC drug in the clinical stage. Preliminary clinical research results show that the drug has demonstrated positive signals in terms of efficacy and safety. Compared with existing standard treatments and potential therapeutic approaches, it exhibits certain clinical advantages, with the potential to improve tumor response and quality of life in patients with uterine leiomyosarcoma who have undergone multiple lines of prior therapy, offering a new treatment option for patients.
 

Overall, from the perspective of the technical route, these drugs each have their own characteristics. This indicates that Chinese pharmaceutical companies are moving away from homogeneous "me-too" competition and shifting towards more differentiated innovation paths. In the future, China's innovative drug assets will gain increasing recognition, and Chinese pharmaceutical companies will accelerate their growth from being "participants" in global pharmaceutical innovation to becoming "co-builders" and even "leaders" in certain fields.
 

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