IASO Biotherapeutics ("IASO Bio"), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, and Innovent Biologics, Inc. ("Innovent", HKEX: 01801), today jointly announced that the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to their fully human anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy (IASO Bio: CT103A, Innovent: IBI326) for the treatment of relapsed and/or refractory multiple myeloma (R/R MM).
This ODD will accelerate drug development and registration action of CT103A in the United States. CT103A will be eligible for certain development incentives, including FDA support for clinical studies, a waiver or reduction of registration application fee, and a seven-year U.S. market exclusivity granted upon product approval. In February 2021, CT103A was granted Breakthrough Therapy Designation by China's National Medical Products Administration (NMPA) for the treatment of R/R MM.
"FDA approval of ODD to CT103A is of great significance to patients with multiple myeloma and represents the FDA's recognition of CT103A and the clinical data provided by IASO Bio", said Dr. Wen Wang, Chief Executive Officer and Chief Medical Officer of IASO Bio. "Currently, our team is advancing the clinical development of CT103A to the four dimensions of strategy including frontline therapy, combination therapies, indication expansion, and ex-China development. We are looking forward to the launch of CT103A both in China and US as soon as possible to offer living-saving treatment option to more patients."
"This ODD of IBI326 is a milestone recognizing our effort to develop a novel anti-BCMA CAR with better efficacy and persistence," said Dr. Hui Zhou, Senior Vice President of Innovent, "It underscores the importance of bringing this therapeutic option to patients with multiple myeloma, and strongly motivates us to expedite the clinical development of IBI326.We hope to launch IBI326 as soon as possible which will bring hope to patients with multiple myeloma."
About the FDA's Orphan Drug Designation
The Orphan Drug Designation (ODD) is granted by the FDA's Office of Orphan Products Development (OOPD) to eligible drugs (including biologic agents) for the prevention, treatment, and diagnosis of rare diseases. The FDA defines rare diseases as those which affect fewer than 200,000 people in the United States. Due to the small number of patients, low market demand and huge R&D costs, pharmaceutical companies were less inclined to invest in the development of these drugs without specific policy support. In 1983 however, the United States Congress passed the Orphan Drug Act (ODA), stipulating that all drug candidates granted the ODD will have access to a series of supporting policies.
About Multiple Myeloma (MM)
Multiple myeloma is a deadly blood cancer that often infiltrates the bone marrow causing anemia, kidney failure, immune problems, and bone fractures. For multiple myeloma patients, common first-line drug treatments include proteasome inhibitors, immunomodulatory drugs, and alkylating agents. While treatment may result in remission, most patients will inevitably enter the relapsed or refractory stage as there's currently no cure. As a result, there is a significant unmet need for patients with relapsed/refractory multiple myeloma. In the United States, MM accounts for nearly 2% of new cancer cases, and more than 2% of all cancer-related deaths.
According to Frost & Sullivan, the number of new MM cases in the United States rose from 30,300 in 2016 to 32,300 in 2020 and is expected to increase to 37,800 by 2025. The total number of patients diagnosed with MM in the United States increased from 132,200 in 2016 to 144,900 in 2020 and is expected to rise to 162,300 by 2025.
In China, the number of new MM cases rose from 18,900 in 2016 to 21,100 in 2020 and is expected to increase to 24,500 by 2025. The total number of patients diagnosed with MM in China in...
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