On February 14, Nanjing Iaso, an innovative biopharmaceutical company dedicated to the development and industrialization of cell therapy and antibody drugs, announced today with Innovent that the U.S. Food and Drug Administration (FDA) Office of Orphan Drug Development has officially issued a written reply granting two Orphan drug designation for fully human autologous B cell maturation antigen chimeric antigen receptor autologous T cell injection for the treatment of relapsed/refractory multiple myeloma (R/R MM) jointly developed by two companies.
FDA orphan drug designation can help accelerate the clinical development and registration of drugs in the United States. The CT103A product candidate will be eligible for a number of preferential policies, including FDA guidance support for clinical studies, special fee waivers, and seven-year market exclusivity in the U.S. for post-approval products. In February 2021, CT103A's therapy for R/R MM has been included in the "Breakthrough Therapy Drug" category by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA).
Dr. Wen Wang, CEO and Chief Medical Officer of Iaso, said: "The FDA's grant of 'Orphan Drug' designation to CT103A is of great significance to patients with multiple myeloma, representing the FDA's affirmation of CT103A's product candidate and the validation of the previous clinical data provided by Iaso. Approved. At present, the reindeer team is advancing the clinical development of CT103A in four dimensions: front-line treatment, combination therapy, autoimmune disease indication expansion, and overseas deployment. We look forward to the early launch of this candidate product to save the lives of more patients!”
"The FDA's orphan drug designation for IBI326 is a milestone in our commitment to developing BCMA-targeted CAR-Ts with greater efficacy and durability, and underscores the need for this therapeutic option," said Hui Zhou, Senior Vice President and Dr. The importance it brings to patients with multiple myeloma also motivates us to further accelerate the clinical development of IBI326. We hope that this product candidate will be launched soon, bringing hope to patients with multiple myeloma.”
About FDA Orphan Drug Designation
Orphan drug designation is a designation granted by the FDA's Office of Orphan Drug Product Development to eligible drugs for the prevention, treatment, and diagnosis of rare diseases. Orphan drugs, also known as rare disease drugs, refer to drugs used for the prevention, treatment, and diagnosis of rare diseases. The FDA has clearly defined criteria for rare diseases, that is, diseases that affect less than 200,000 people in the United States. Due to the small number of patients with rare diseases, low market demand and high research and development costs, few pharmaceutical companies pay attention to the research and development of their therapeutic drugs, so these drugs are called "orphan drugs". In 1983, the United States promulgated the Orphan Drug Act (ODA), which stipulates that any candidate drug that has obtained orphan drug qualification has the opportunity to obtain a series of supporting policies.
About Multiple Myeloma (MM)
Multiple myeloma (MM), one of the most common blood cancers, is a malignant disease of abnormal proliferation of clonal plasma cells. For newly treated patients with multiple myeloma, commonly used first-line treatment drugs include proteasome inhibitors, immunomodulatory drugs and alkylating agents. For most patients, the commonly used first-line treatment can stabilize the patient's condition for 3-5 years, but a small number of patients show primary drug resistance at the initial treatment, and the disease cannot be effectively controlled. For most of the newly treated patients with effective treatment, they will inevitably enter the relapse and refractory stage after the stable disease period. Therefore, there is still an unmet 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.
According to the Frost & Sullivan report:
• The number of new cases of MM in the US increased from 30,300 in 2016 to 32,300 in 2020 and is expected to increase to 37,800 in 2025. The number of people with MM in the United States increased from 132,200 in 2016 to 144,900 in 2020 and is expected to increase to 162,300 in 2025.
• The number of new cases of MM in China increased from 18,900 in 2016 to 21,100 in 2020 and is expected to grow to 24,500 in 2025. The prevalence of MM in China increased from 69,800 in 2016 to 113,800 in 2020 and is expected to increase to 182,200 in 2025.
About CT103A (BCMA CAR-T)
CT103A is an innovative product candidate jointly developed by Iaso and Innovent Pharmaceuticals. This product candidate uses lentivirus as a gene carrier to transfect autologous T cells. The CAR contains fully human scFv, CD8a hinge and transmembrane, 4-1BB costimulatory and CD3ζ activation domains. Based on rigorous screening and comprehensive in vitro and in vivo functional evaluation, the CT103A CAR-T product candidate has potent and rapid efficacy with outstanding durability. In February 2021, CT103A was approved by the State Food and Drug Administration as a "breakthrough therapy drug" for the treatment of relapsed and refractory multiple myeloma. In February 2022, CT103A was granted "orphan drug" designation by the U.S. Food and Drug Administration for the treatment of relapsed and refractory multiple myeloma.
About Nanjing Iaso
Iaso is an innovative biopharmaceutical company focused on the development and industrialization of cell therapy and antibody drugs. The company takes the development of hematological tumor cell-based drugs and antibody drugs as the cornerstone of innovation, and expands to solid tumors and autoimmune diseases. Multiple technology platforms including human antibody discovery platform, high-throughput CAR-T drug optimization platform, general CAR technology platform, production technology platform, and clinical translational research platform. At present, 10 products under development are in different stages of research and development. Among them, the most rapidly progressing candidate product CT103A (full human BCMA chimeric antigen receptor autologous T cell injection) is in the late stage of clinical development and has been approved by the company in February 2021. The Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) has included the "Breakthrough Therapy Drug" category; the company's self-developed innovative candidate product CT120 (full human CD19/CD22 dual-target CAR-T cell injection) has been It has entered the clinical research stage and has obtained FDA orphan drug designation (ODD).
With a strong executive management team, rich product pipeline, unique innovative R&D and business model, Iaso aspires to become an influential innovative pharmaceutical company in the industry, bringing innovative drugs that truly solve clinical pain points and have market competitiveness to the clinic. Until the market, it will open up new treatment paths for subjects and bring new hope.