The US FDA has granted orphan drug designation to Shanghai-headquartered biotech CARsgen Therapeutics’ CAR-T cell therapy CT053 for multiple myeloma after positive results were observed in the phase I trial efficacy evaluation.


19 of 24 patients with relapsed and refractory multiple myeloma showed complete response

Zonghai Li, CARSgen Therapeutics

CARsgen Therapeutics made headlines as its multiple myeloma CAR-T cell therapy CT053 has been granted orphan drug status by the US FDA on August 30 2019. CT053 is composed of fully human anti-BCMA (B Cell Maturation Antigen) autologous chimeric antigen receptor T cells. BCMA is a protein found on the surface of myeloma cells and is considered a primary target for cell therapy.


CARsgen, an immune-oncology biotech company, currently has 11 cancer-targeting CAR-T therapies in its pipeline, five of which are currently in clinical trial stages. CT053, alongside GPC3 for hepatocellular carcinoma, was approved earlier this year as an investigational new drug (IND) by the Chinese National Medical Products Administration, allowing it to be tested in patients with relapsed or refractory multiple myeloma in clinical trials. 


Phase I trials of CT053 began in China in June 2019, and late August 2019 marked the first efficacy evaluation upon 12 weeks of CT053 cell infusion in which the overall response rate is measured. The results of this evaluation concluded that “19 of 24 patients with relapsed and refractory multiple myeloma showed complete response… and importantly, no event of [side effect] grade 3 or higher cytokine release syndrome (CRS) was observed,” according to CEO Zonghai Li. 


Orphan drug designation is granted to treatments that target rare diseases that affect fewer than 200,000 people in the United States.  It is an important regulatory milestone that offers financial incentives around development and commercialization, as well as US market exclusivity for seven years following its approval.