Another Chinese-developed CAR-T Therapy Receives FDA Orphan Drug Designation

　　【Pharmaceutical Network Industry Dynamics】CAR-T cell therapy mainly involves transferring in vitro cultured CAR-T cells into the human body to attack and kill tumor cells within the patient by activating T cells, showing great potential in cancer treatment. Data shows that the CAR-T cell market will grow at an annual rate of 20.2%, reaching a value of $20.3 billion by 2027. Faced with a vast blue ocean market, along with support from policies, talent, and capital, pharmaceutical companies in China are making continuous efforts in the CAR-T cell therapy field, showing high enthusiasm for research and development. To date, the number of CAR-T clinical trials conducted in China has exceeded 500.
 

As the track in China becomes increasingly crowded, products granted Orphan Drug Designation (ODD) by the FDA often mark a new milestone, signifying regulatory recognition of the product. In recent years, with the continuous rise of China's innovative pharmaceutical companies, more and more proprietary CAR-T drugs produced in China have been receiving good news of obtaining FDA orphan drug designation.
 

　　Another CAR-T Product Made in China Granted Orphan Drug Designation
 

Recently, Juventas and the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences jointly announced that their self-developed CD19-targeted CAR-T drug, CNCT19 Cell Injection, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of acute lymphoblastic leukemia.
 

Previously, CNCT19 cell injection has been approved in China for the treatment of relapsed or refractory acute lymphoblastic leukemia and relapsed or refractory aggressive B-cell non-Hodgkin lymphoma. Both of these clinical trials have entered the Phase 2 registrational clinical stage. In December 2020, the Center for Drug Evaluation of China’s National Medical Products Administration included CNCT19 cell injection for the treatment of relapsed or refractory acute lymphoblastic leukemia.
 

Early clinical research results show that CNCT19 cell injection demonstrates excellent efficacy and safety for both adult and pediatric r/r B-ALL patients.
 

As CNCT19 Cell Injection receives the FDA Orphan Drug Designation, industry experts believe it may help accelerate the clinical trials and registration process for this product in the United States. This also signifies that this innovative drug, independently developed in China by Juventas, will move from China to the global stage.
 

Just half a year ago, the FDA granted C-CAR039, a dual-target CAR-T product developed by Cellular Biomedicine Group, orphan drug designation for the treatment of follicular lymphoma, offering new hope to patients with follicular lymphoma and the potential for an effective treatment option. In Western countries, follicular lymphoma is a common subtype of non-Hodgkin lymphoma, affecting 2-4 people per 100,000, with its prevalence steadily increasing. Data shows that the global follicular lymphoma treatment market is projected to grow at a rate of 7.6%, from $2.08 billion in 2019 to $3.73 billion by 2027.

 

　　New Progress in CAR-T Cell Therapy Made in China Continues
 

In addition to being granted orphan drug designation by the FDA, several CAR-T cell therapies produced in China have received accelerated approval from the European Medicines Agency (EMA) in the past two years.
 

For example, in November 2021, Carsgen Therapeutics announced that its self-developed CT041 had been granted Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of gastric cancer/gastroesophageal junction cancer. This product is a potential global first-in-class, CLDN18.2-targeted autologous CAR-T candidate being developed for the clinical treatment of CLDN18.2-positive solid tumors such as gastric cancer/gastroesophageal junction cancer and pancreatic cancer. It is also the world's first solid tumor CAR-T product to be included in the PRIME program. After this product was placed in the priority pathway, it helped shorten the review time for the drug application, benefiting patients earlier.
 

In February 2021, Genscript's subsidiary Legend Biotech announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had accepted the Marketing Authorization Application (MAA) for its novel biologic product cilta-cel, used to treat refractory and relapsed multiple myeloma, for accelerated assessment. Previously, the product had received Breakthrough Therapy Designation in the United States, Priority Medicine (PRIME) designation and Orphan Drug Designation from the European Commission, and Breakthrough Therapy Designation in China in August 2020.
 

It can be seen that China-produced CAR-T therapies are gradually ushering in new advancements. Currently, the number of CAR-T cell therapies available globally is still very limited. If China-produced products successfully tap into overseas markets one after another, it will create an incredibly inspiring new landscape.