On September 18, AbClon, a company specializing in antibody-based drugs, announced that its next-generation CAR-T cell therapy, Nespe-cel (AT101), was designated as an orphan drug in development by the Ministry of Food and Drug Safety (MFDS).

Nespe-cel (AT101) is a personalized treatment for patients with relapsed or refractory blood cancers. The process involves extracting a patient's T-cells, genetically modifying them with AbClon 's proprietary h1218 antibody to specifically target cancer cells, and then re-infusing them back into the patient.

Nespe-cel is attracting attention due to its exceptional therapeutic efficacy. Interim results from a Phase 2 clinical trial showed an objective response rate (ORR) of 94%, a significant improvement over the 52% ORR of existing treatments currently used in Korea. Additionally, the complete response rate (CRR) was 68%, demonstrating a markedly superior outcome compared to the 40% seen with the conventional therapy.

The core technological difference of Nespe-cel lies in AbClon's unique antibody and CAR-T technology. Unlike existing global CAR-T therapies that use the murine 'FMC63' antibody, AbClon has applied its self-developed, CD19-specific, humanized antibody, 'h1218'. The use of the h1218 antibody improves the drug's persistence and triggers a more powerful anti-cancer effect, securing a high level of technological competitiveness on the global stage.

Orphan drug designation in development by the Ministry of Food and Drug Safety (MFDS) is a system to support the creation of treatments for rare and intractable diseases with fewer than 20,000 patients in Korea.

This designation allows significant benefits:

• The product license validity period is extended from 5 years to 10 years.

• Administrative advantages, such as simplified data submission, are granted during the application process.

• The exclusive data protection period for the new drug is extended from the previous 4-6 years to 10 years.

These benefits increase development efficiency, secure market exclusivity, and give the company a competitive advantage in commercializing the drug.

According to the Korea Biotechnology Industry Organization, the global CAR-T cell therapy market was valued at $3.74 billion in 2023. It is projected to grow at an average annual rate of approximately 40% to reach $29 billion by 2029.

Specifically, CD19-targeted therapies held a market share of 74% in 2023, valued at $2.76 billion, and are expected to expand to $22 billion by 2029.