On December 26, 2022, Huaxia Yingtai announced that it had received a written response from the Office of Orphan Products Development (OOPD) of the United States Food and Drug Administration (FDA). The integrative T-cell receptor Antigen Receptor (STAR T) injection (development code: YTS104) designed by the company was granted Orphan Drug Designation (ODD) for the treatment of relapsed/refractory acute myeloid leukemia (R/R AML).
FDA orphan drug status can help accelerate clinical development and registration of drugs in the United States. With the FDA's ODD designation, YTS104 will be eligible for a number of favorable policies, including FDA guidance support for the product candidate, special fee waivers, and seven years of market exclusivity in the United States.
Dr. Guohua Pan, CEO of Huaxia Yingtai, said:
"I am very proud of our team. Following the completion of the Phase I clinical trial of our dual-target STAR-T product and the enrollment of the first patient in early December, YTS104 received FDA orphan drug status, which not only confirms the approval of our product by the Chinese NMPA and the US FDA review authorities, It also marks that Huaxia Yingtai has entered a new stage of clinical development from new drug research and development. This recognition of FDA orphan drug status is an important milestone in the development process of YTS104, which will play a positive role in accelerating the clinical development and future registration and marketing of YTS104 in China and the United States. We are confident that through unremitting exploration and efforts, we will soon bring new treatment options to patients with acute myeloid leukemia worldwide."
About YTS104
YTS104 cell injection is an autologous cell therapy drug based on the innovative STAR T platform developed by Huaxia Yingtai. The primary target indication is relapsed/refractory acute myeloid leukemia (R/R AML). This product is a cell therapy drug obtained by introducing specific STAR genes into autologous T cells by means of lentivirus gene transduction, and after in vitro cell amplification preparation, function and quality control testing. The advantage of the product is that two self-screened nano antibodies with different epitopes are connected to α chain and β chain of STAR structure, which is expected to have better antigen recognition and killing effect.
About FDA Orphan Drug Designation (ODD)
The Designation, Orphan Drug Designation, ODD, was designed by the Office of Orphan Products Development, FDA. OOPD) A qualification granted to qualified drugs (including biologics) for the prevention, treatment and diagnosis of rare diseases. Orphan drugs, also known as orphan drugs, refer to drugs used for the prevention, treatment and diagnosis of rare diseases. The FDA has a clear definition of rare diseases, namely diseases affecting less than 200,000 people in the United States. Because of the small number of people affected by rare diseases, low market demand and high research and development costs, few pharmaceutical companies pay attention to the development of their treatments, so these drugs are called "orphan drugs". In 1983, the Orphan Drug Act (ODA) was issued in the United States, stipulating that candidate drugs eligible for orphan drug qualification have the opportunity to obtain a series of supporting policies.
About Acute Myeloid Leukemia (AML)
Acute myeloid leukemia (AML) is a malignant disease of clonal proliferation of myeloid primitive cells in the hematopoietic system, characterized by abnormal proliferation of primitive and naive myeloid cells in the bone marrow and peripheral blood. The clinical manifestations are anemia, bleeding, infection and fever, visceral infiltration, metabolic abnormalities, etc., with strong heterogeneity, limited therapeutic means, and poor prognosis. In 2017, the number of AML cases worldwide was 119,570. China ranked among the world's top three in terms of both cases and deaths, with 13,200 cases and 7,100 cases, respectively. Compared with non-monocyte subtypes, monocyte AML (M4 and M5) had a greater risk of bone marrow and extramaterial recurrence after stem cell transplantation (34% and 50%, respectively). For relapsed and refractory AML patients, there is no clear treatment that can significantly prolong survival. The median OS of elderly relapsed refractory patients is 5-9 months after low-dose chemotherapy, while the median OS of patients with ≥2 relapses is only 3 months.
Huaxia Yingtai
Huaxia Yingtai is an innovative pharmaceutical enterprise with a global vision, focusing on the development of T cell immunotherapy products and addressing unmet clinical needs such as advanced cancer. Founded in March 2018, Huaxia Yingtai was led by a famous immunologist. The company has established two innovative technology platforms, STAR-T and enTCR-T, as well as mature industrialization platforms for process development and quality control, and laid out a rich product pipeline covering hematoma, solid tumor and viral infection. More than 10 "First-in-Human" clinical studies have been initiated and positive clinical safety and efficacy data have been obtained, which have been successively published in international academic conferences and journals. Many projects are actively promoting registered clinical studies. The company's mission is to "focus on science, strive for perfection, relieve cancer patients' pain, and become a pioneer and leader of global innovative cellular immunotherapy products based in China and benefiting patients around the world".
