AbClon, a Korean antibody drug developer, announced on September 18 that its next-generation CAR-T (chimeric antigen receptor T-cell) therapy Nespe-cel (AT101) has been designated as a developmental-stage orphan drug by the Ministry of Food and Drug Safety (MFDS).
Nespe-cel is a personalized treatment for patients with relapsed or refractory blood cancers. The therapy works by extracting a patient’s T-cells, genetically modifying them with AbClon’s proprietary H1218 antibody to specifically target cancer cells, and reinfusing them back into the patient.
The drug has drawn attention for its clinical efficacy. Interim results from a Phase 2 clinical trial showed an objective response rate (ORR) of 94%, compared to 52% with currently available therapies in Korea. The complete remission rate (CRR) also reached 68%, significantly higher than the 40% seen with existing treatments.
A key differentiator of Nespe-cel lies in its proprietary antibody and CAR-T platform. While most global CAR-T therapies use the mouse-derived “FMC63” antibody, AbClon applied its own H1218 antibody, which targets the CD19 protein commonly expressed in blood cancers. According to the company, this enhances drug persistence and induces a more potent anti-cancer effect.
The MFDS’s developmental orphan drug designation is granted to treatments for rare and intractable diseases with fewer than 20,000 patients in Korea. Benefits include an extended marketing authorization period (10 years instead of 5), streamlined review procedures, and exclusive data protection of up to 10 years, compared to the current 4–6 years. These incentives are designed to improve R&D efficiency and help secure market exclusivity.
According to the Korea Biotechnology Industry Organization, the global CAR-T cell therapy market reached USD 3.74 billion in 2023 and is expected to grow at a compound annual rate of about 40%, reaching USD 29 billion by 2029. CD19-targeting therapies account for the majority, with a market size of USD 2.76 billion (74% share) in 2023, projected to expand to USD 22 billion by 2029.
Development of Nespe-cel is being supported by the Korea Drug Development Fund (KDDF). In Türkiye, biotech company TCT has licensed the technology and is working on local process development, while in Korea, Chong Kun Dang holds priority commercialization rights and is collaborating with AbClon on co-development.
An AbClon official commented, “Nespe-cel represents new hope for patients with relapsed or refractory blood cancers, offering the potential to overcome the limitations of existing third-line therapies. With the orphan drug designation, we will make every effort to accelerate commercialization through expedited and conditional approval pathways.”
