Oricell's GPRC5D CAR-T receives Orphan Drug Designation from FDA
Shanghai, Oct. 25, 2022 -- Oricell Therapeutics (Shanghai) Co., Ltd. ("Oricell" or "the company”) announced that the United States (U.S) Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted Orphan Drug Designation (ODD) to Oricell's proprietary CAR-T cell therapy targeting GPRC5D (OriCar-017) for the treatment of Relapsed/Refractory Multiple Myeloma (R/R MM).
The ODD status would help accelerate OriCar-017's clinical development and registration in the U.S. This designation is primarily based on the efficacy results from Oricell's first-in-human (FIH) phase I clinical study carried out in China (NCT05016778).
Having received the ODD, OriCAR-017 is eligible to receive incentives such as FDA's guidance on clinical research, waiver of user fees, and seven-year marketing exclusivity for any approved indication in the U.S.
On September 21st of this year, Ori-C101, another OriCell’s proprietary CAR-T product targeting Glypican-3 (GPC3) for the treatment of advanced liver cancer was granted Investigational New Drug (IND) license from China’s National Medical Products Administration (NMPA).
“We are pleased to see OriCAR-017 granted ODD status by the U.S FDA, just a month after OriCAR-101 received the IND license in China“， said Ms. Yang Huanfeng, chairman and CEO of Oricell.
“OriCAR-017’s ODD status in the U.S, together with OriCAR-101’s IND license in China are both key milestones for us, driving the company into a new phase of development with both recognition by the China and U.S regulatory authority on the direction, significance and capability of our company’s R&D.” So believes Ms. Yang, “It also gives us a big boost in our pursuit of science-driven innovation intended for all patients around the world.” She added.
OriCAR-017 is one of Oricell's core products, a Chimeric antigen receptor (CAR)-T cell therapy targeting GPRC5D for the treatment of Relapsed/Refractory Multiple Myeloma. Since Apr. 30, 2022, clinical data from the phase I POLARIS study carried out by researchers in China has demonstrated its favorable safety and efficacy (100% ORR, 100% MRD 10-5 negative at day 28). All patients including those who had relapsed from prior BCMA CAR-T therapy had ongoing improvement of responses, are progression free and did not require additional therapy at data cutoff.
Results from the study are reported in oral presentation at both ASCO (American Society of Clinical Oncology) 2022 annual meeting and EHA (European Hematology Association) 2022 congress. GPRC5D (OriCAR-017) received Orphan Drug Designation by FDA office of orphan products at Oct. 24 2022. (For more information, see https://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm)
About FDA Orphan Drug Designation (ODD)
“Orphan Drug” refers to drugs developed to treat, prevent or diagnose rare diseases. The FDA has a clearly defined criteria for rare diseases — those that affect fewer than 200,000 people in the United States. Orphan drug designation is a designation granted by the U.S. FDA's Office of Orphan Drug Product Development (OOPD) to eligible drugs (including biologicals) for the prevention, treatment, and diagnosis of rare diseases. Orphan drug designation plays an important role in drug development. Designated drugs are eligible to enjoy several preferential policies, including 7 years of marketing exclusivity after approval, exemption of NDA/BLA application fees, tax deduction for clinical research expenses, and possible exemption of some clinical data declaration requirements. (For more information, see https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products)
About Multiple Myeloma
Multiple myeloma (MM), one of the most common blood cancers, is a malignant disease of abnormal proliferation of clonal plasma cells. For newly treated MM patients, commonly used first-line treatment drugs include proteasome inhibitors, immunomodulatory drugs and alkylating agents. For most patients, the commonly used first-line treatments can stabilize the patient's condition for 3-5 years, but a small number of patients show primary drug resistance at initial treatment, and the disease cannot be effectively controlled. Most of the newly treated patients with effective treatment will inevitably enter the relapse and refractory stage after the stable disease period. Therefore, there is still an unmet clinical need for patients with relapsed/refractory multiple myeloma. In the United States, MM accounts for nearly 2% of all new cancer cases and more than 2% of cancer deaths. (For more information, see https://pubmed.ncbi.nlm.nih.gov/33498356/)