Two Chinese Innovative Drugs Receive FDA Orphan Drug Designation on the Same Day

　　【Pharmaceutical Network Product InformationOn the same day (March 24), Qilu Pharmaceutical and CHANGCHUN HIGH-TECH, two pharmaceutical companies in China, announced that their innovative drugs had been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA).
 

Among them, Qilu Pharmaceutical's newly awarded FDA orphan drug designation is for the Class 1 new drug Claudin18.2/CD3 bispecific antibody QLS31905. Meanwhile, CHANGCHUN HIGH-TECH's newly awarded FDA orphan drug designation is for GenSci128 tablets, a Class 1 new chemical drug developed by its subsidiary Jin Sai Pharmaceutical, intended for the treatment of gastric cancer.
 

Data show that QLS31905 is a bispecific T-cell engager targeting Claudin18.2, which can bind to Claudin18.2 on the surface of tumor cells and CD3 on the surface of T cells. By recruiting and activating T cells near tumor cells, it continuously kills tumor cells. Currently, a Phase III clinical trial of QLS31905 combined with chemotherapy for first-line treatment of advanced pancreatic cancer is being conducted in China.
 

It is reported that pancreatic cancer is one of the most difficult cancers to cure, with a poor prognosis and a 5-year survival rate of only about 7.2%, representing a significant unmet clinical need. QLS31905 is expected to become a new first-line immunotherapy option for patients with Claudin18.2-positive pancreatic cancer.
 

GenSci128 Tablets are selective reactivators targeting the TP53 Y220C mutation, intended for the treatment of locally advanced or metastatic solid tumors carrying the TP53 Y220C mutation, including pancreatic cancer, gastric cancer, ovarian cancer, breast cancer, and colorectal cancer. GenSci128 Tablets aim to selectively bind to the pocket of the TP53 Y220C mutant protein, thereby restoring the normal conformation of the TP53 Y220C mutant protein, enhancing its stability, and reinstating transcription and tumor suppression functions.
 

It is reported that GenSci128 tablets have been approved in China and the United States for clinical trials in patients with locally advanced or metastatic solid tumors carrying the TP53 Y220C mutation. Its indication for pancreatic cancer treatment has received FDA orphan drug designation.
 

Orphan drugs, also known as rare disease drugs, refer to pharmaceuticals used for the prevention, treatment, and diagnosis of rare diseases. Orphan drug designation is a core incentive policy established by the FDA to support the accelerated research, development, and approval of innovative therapies for rare diseases. Therefore, obtaining orphan drug designation holds significant importance for new drug development. After these two innovative drugs received orphan drug designation this time, it is expected to expedite the progress of clinical trials and market registration. Additionally, they can enjoy certain policy supports, including tax credits for clinical trial expenses, waiver of new drug application fees, and a seven-year exclusive market right in the U.S. upon market entry.
 

In recent years, Chinese pharmaceutical companies have continuously increased their investment in innovation, focusing on difficult-to-treat diseases and rare disease fields, constantly breaking through core technology barriers, and entering a period of explosive growth in innovative drug research and development. If the subsequent clinical trials of these two innovative drugs proceed smoothly, it will also help the company broaden its business structure, optimize its product mix, enrich and improve the strategic product line layout, and enhance the company's core competitiveness.
 

Disclaimer: In any case, the information or opinions expressed in this article do not constitute investment advice to any person.