UCB has received approval from Japan's Pharmaceuticals and Medical Devices Agency (PMDA) for two new administration methods for RYSTIGGO® (rozanolixizumab), allowing patients with generalized Myasthenia Gravis (gMG) to self-administer the treatment at home. The approval, announced on May 9, 2025, covers both infusion pump and manual push syringe methods, providing greater flexibility and autonomy for patients managing this rare neuromuscular disease.
New Administration Options Enhance Patient Autonomy
The newly approved administration methods enable patients to self-administer RYSTIGGO at home following training from healthcare professionals. This development addresses a significant challenge for gMG patients, whose unpredictable symptoms can severely impact daily activities and quality of life.
"For people living with gMG, unpredictable symptoms can significantly impact daily life, leading to feelings of vulnerability and loss of control," said Donatello Crocetta, Chief Medical Officer and Head of Global Medical Affairs at UCB. "Subcutaneous self-administration can help address these challenges, providing better control over treatment schedules and enhancing autonomy and satisfaction."
ONWARD Support Program Launches in Japan
Complementing the approval, UCB Japan is launching a comprehensive patient support initiative called the ONWARD program. This service includes:
- Home delivery of RYSTIGGO and portable syringe pumps
- Dedicated Care Coordinators for personalized patient support
- Symptom tracking tools
- 24/7 online resources
The ONWARD program is designed to reduce treatment burden and provide tailored support for individuals living with gMG and other rare diseases. Patient information is strictly managed by subcontractors, with UCB having no access to personally identifiable information.
Background on RYSTIGGO and gMG
RYSTIGGO was initially approved in Japan in September 2023 for treating gMG in adult patients who do not adequately respond to steroids or nonsteroidal immunosuppressants. Japan made history as the first country to simultaneously approve both RYSTIGGO and UCB's other gMG therapy, ZILBRYSQ® (zilucoplan).
RYSTIGGO is a humanized monoclonal antibody that specifically binds to the fetal Fc receptor (FcRn), inhibiting the interaction between FcRn and immunoglobulin G (IgG). This mechanism promotes the catabolism of antibodies and reduces the concentration of pathogenic IgG autoantibodies that contribute to gMG.
The efficacy of RYSTIGGO was demonstrated in the Phase 3 global MycarinG trial, which showed statistically significant reductions in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score compared to placebo.
Understanding Generalized Myasthenia Gravis
gMG is a rare, chronic autoimmune disease characterized by dysfunction at the neuromuscular junction. The condition affects an estimated 100 to 350 people per million worldwide, with approximately 29,210 patients in Japan.
Patients with gMG experience various symptoms including ptosis (drooping eyelids), diplopia (double vision), difficulty swallowing and speaking, and potentially life-threatening respiratory muscle weakness. The disease occurs when pathogenic autoantibodies impair synaptic transmission at the neuromuscular junction, disrupting nerve signals to skeletal muscles and resulting in muscle weakness.
Safety Profile
The most commonly reported adverse reactions in clinical studies of RYSTIGGO include headache (48.4%), diarrhea (25.0%), and pyrexia (12.5%). In the MycarinG trial, headache was the most common reaction, reported in 48.4% of patients treated with rozanolixizumab compared to 19.4% of those receiving placebo. Most headaches were mild to moderate in severity, and there was no increase in incidence with repeated cyclic treatment.
Expanding Global Presence
This approval in Japan represents another milestone in UCB's commitment to addressing the needs of patients with rare neurological conditions. RYSTIGGO has previously received approval in the United States and Europe, with the Japanese approval for self-administration methods further enhancing the medication's accessibility and convenience.
"We are delighted to achieve this approval in Japan and the roll out of the ONWARD home delivery service, which together provide personalized support and reduce the treatment burden for those affected by gMG," added Crocetta.
For gMG patients in Japan, these developments offer new options for managing their condition with greater independence and support, potentially improving treatment adherence and quality of life.
