HanAll Biopharma has announced that batoclimab, its investigational anti-FcRn antibody, has received Orphan Drug Designation (ODD) from Japan's Ministry of Health, Labor and Welfare (MHLW) for the treatment of active Thyroid Eye Disease (TED).
The designation represents a significant regulatory milestone for the South Korean biopharmaceutical company as it advances batoclimab through late-stage clinical development. In Japan, ODD is granted to therapies targeting conditions affecting fewer than 50,000 people, with TED estimated to impact approximately 35,000 individuals across the country.
"We are thrilled to have received Orphan Drug Designation for batoclimab in Japan, marking an important milestone in our efforts to bring this promising treatment to patients in need," said Sean Jeong, MD, MBA, CEO of HanAll Biopharma. "This designation highlights the potential impact batoclimab could have on the lives of patients with TED. We remain dedicated to advancing the development of this treatment and are focused on bringing it closer to the market."
Mechanism of Action and Administration Advantages
Batoclimab is a monoclonal antibody specifically designed to target and inhibit the neonatal Fc receptor (FcRn), which plays a crucial role in recycling immunoglobulin G (IgG) antibodies. By selectively binding to FcRn, the drug reduces circulating levels of pathogenic IgG antibodies that drive autoimmune conditions like TED.
A key differentiator for batoclimab is its subcutaneous (SC) formulation, which could potentially allow for at-home self-administration. This approach offers significant advantages over intravenous therapies that require clinical settings for administration, potentially improving treatment adherence and reducing healthcare system burden.
Clinical Development Program
HanAll and its licensees are currently conducting a Phase 3 clinical trial of batoclimab in active TED, with patient enrollment ongoing in Japan. The study aims to evaluate the efficacy and safety of the drug as a potential new treatment option for this debilitating condition. Top-line results from this pivotal trial are expected in the second half of 2025.
Beyond TED, batoclimab is being investigated globally for multiple autoimmune conditions, including generalized myasthenia gravis (gMG), chronic inflammatory demyelinating polyneuropathy (CIDP), and Graves' disease, demonstrating the broad potential of FcRn inhibition across IgG-mediated autoimmune disorders.
Understanding Thyroid Eye Disease
Thyroid Eye Disease, also known as Graves' orbitopathy, is a rare and debilitating autoimmune disorder primarily affecting individuals with hyperthyroidism or Graves' disease. The condition is characterized by inflammation and tissue expansion in the orbit, leading to a range of severe symptoms including proptosis (eye bulging), periorbital edema, diplopia (double vision), and in severe cases, vision loss due to optic nerve compression.
The disease significantly impacts quality of life, limiting daily activities such as reading, driving, and working. Many patients also experience substantial psychological distress related to facial disfigurement, leading to anxiety, depression, and social withdrawal.
Current treatment options for moderate to severe TED remain limited, with corticosteroids serving as first-line therapy despite variable efficacy and significant side effects. While one biologic therapy has been approved in recent years, there remains substantial unmet need for additional effective and convenient treatment options.
Expanding Pipeline and Global Presence
HanAll Biopharma has been expanding its focus beyond its traditional therapeutic areas of endocrine, circulatory, and urologic diseases to include immunology, oncology, neurology, and ophthalmology. The company has established presence in Korea, the United States, Japan, and Indonesia.
In addition to batoclimab (HL161), the company is developing HL161ANS (IMVT-1402), another anti-FcRn antibody being evaluated in Graves' Disease and Rheumatoid Arthritis. HanAll's ophthalmology program includes HL036 (tanfanercept), a TNF inhibitor protein that has commenced a Phase 3 VELOS-4 study in the US for dry eye disease.
The Orphan Drug Designation in Japan provides various benefits including priority review, potential for conditional early approval, and extended market exclusivity, which could accelerate batoclimab's path to patients with this challenging condition.
