Rozanolixizumab
- Generic Name
- Rozanolixizumab
- Brand Names
- Rystiggo
- Drug Type
- Biotech
- CAS Number
- 1584645-37-3
- Unique Ingredient Identifier
- P7186074QC
- Background
Rozanolixizumab is a humanized high-affinity anti-human neonatal Fc receptor (FcRn) monoclonal antibody (IgG4P) targeting the immunoglobulin G (IgG). Rozonolixizumab itself is an IgG4P, an inactive isotype, to reduce the likelihood of unwanted chain exchange. It is investigated for use in autoimmune and alloimmune diseases with pathologic IgG, particularly generalized myasthenia gravis. Generalized myasthenia gravis is characterized by the formation of IgG antibodies against the nicotinic acetylcholine receptor (AChR) or muscle-specific tyrosine kinase (MuSK). Approximately 80% of myasthenia gravis patients tested positive for the AChR autoantibodies, and 40% of these AChR-negative or seronegative patients were found to have MuSK autoantibodies.
AChR is vital for signal transduction in the neuromuscular junctions (NMJ) by generating muscle end plate potentials to propagate action potential. Therefore, the presence of AChR-antibodies can interfere with the ACh-mediated downstream signaling, thus reducing the likelihood of end plate potentials reaching the threshold needed to trigger an action potential. As a result, the main clinical manifestation of myasthenia gravis is easily fatigable or persistent muscle weakness. On the other hand, MuSK activation can trigger the clustering of AChR at the NMJ, guide the innervation of motor neurons toward AChR-dense areas, and anchor acetylcholinesterase. Therefore, autoantibodies against MuSK can also affect the signal propagation at the NMJ.
Rozanolixizumab-noli is available under the brand name RYSTIGGO and was developed by UCB. It was granted orphan drug designation by the FDA in 2019, by the European Medicines Agency (EMA) in April 2020, and by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) in November 2020. In June 2023, Rozanolixizumab-noli was approved by the FDA under Priority Review for the treatment of adult patients with generalized myasthenia gravis who are positive for the anti-acetylcholine receptor (AchR) or anti-muscle-specific tyrosine kinase (MuSK) antibody. This is due to the efficacy demonstrated in the pivotal Phase 3 MycarinG study (NCT03971422).
- Indication
Rozanolixizumab-noli is indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
- Associated Conditions
- Generalized Myasthenia Gravis
UCB's RYSTIGGO® (Rozanolixizumab) Approved in Japan for At-Home Self-Administration in gMG Patients
• UCB has received approval from Japan's PMDA for at-home self-administration of RYSTIGGO® (rozanolixizumab) via infusion pump or manual push syringe for patients with generalized Myasthenia Gravis (gMG).
• The approval is complemented by UCB's launch of the ONWARD program in Japan, which includes home delivery service for RYSTIGGO, portable syringe pumps, and personalized patient support with dedicated Care Coordinators.
• This development follows Japan's September 2023 approval of RYSTIGGO for gMG treatment, making it the first country to simultaneously approve both RYSTIGGO and ZILBRYSQ® (zilucoplan), UCB's two targeted therapies for gMG.
Immunovant Shifts Focus to Next-Gen Therapy Despite Positive Phase III Results for Batoclimab in Myasthenia Gravis
• Immunovant's batoclimab demonstrated statistically significant improvement in myasthenia gravis symptoms in Phase III trial, with high-dose patients showing a 5.6-point improvement on the MG-ADL scale versus 3.6 points for placebo.
• Despite positive clinical outcomes, Immunovant announced it will not pursue regulatory approval for batoclimab in myasthenia gravis or chronic inflammatory demyelinating polyneuropathy due to competitive market pressures.
• The company is pivoting resources to its next-generation anti-FcRn antibody IMVT-1402, with pivotal trials expected to begin imminently for both myasthenia gravis and CIDP indications.
FDA Grants Fast Track Designation to Nipocalimab for Sjögren's Disease Treatment
• Johnson & Johnson's investigational monoclonal antibody nipocalimab has received FDA Fast Track designation for treating moderate-to-severe Sjögren's disease, following its Breakthrough Therapy designation in November 2024.
• The Phase 2 DAHLIAS study showed significant results with the 15 mg/kg dose demonstrating over 70% relative improvement in systemic disease activity compared to placebo, representing the first positive results for an FcRn blocker in Sjögren's disease.
• Sjögren's disease affects approximately four million people worldwide with no FDA-approved treatments that address its underlying causes, leaving a significant unmet need for the predominantly female patient population.
Amgen's Uplizna Shows Sustained Efficacy in Myasthenia Gravis Patients Through One Year
• Amgen's Uplizna demonstrated durable efficacy in patients with acetylcholine receptor antibody-positive generalized myasthenia gravis, with 72.3% of treated patients showing significant improvement in daily living activities versus 45.2% on placebo at 52 weeks.
• The anti-CD19 antibody therapy maintained its efficacy with an infrequent dosing schedule of just two doses per year following an initial loading dose, potentially offering a competitive advantage over current treatments requiring more frequent administration.
• Regulatory filing for Uplizna in generalized myasthenia gravis is anticipated in the first half of 2025, with the FDA having already granted the therapy Orphan Drug Designation for this indication.
Eculizumab Shows Promise in Treating Thymoma-Associated Myasthenia Gravis
• A real-world study demonstrates that eculizumab significantly reduces Myasthenia Gravis Activities of Daily Living (MG-ADL) scores in patients with thymoma-associated myasthenia gravis.
• The research indicates a substantial decrease in the need for corticosteroids among patients treated with eculizumab by week 12.
• Clinically meaningful improvements were observed in 81.8% of patients, with initial benefits appearing within an average of 1.7 weeks after starting eculizumab.
• The study suggests eculizumab is a safe and effective treatment option for this severe myasthenia gravis subtype, warranting further investigation through larger randomized controlled trials.
Nipocalimab Receives FDA Priority Review for Generalized Myasthenia Gravis Treatment
• The FDA granted Priority Review to nipocalimab for treating gMG in antibody-positive patients, expediting its potential availability to patients.
• Phase 3 Vivacity-MG3 study results supported the application, demonstrating sustained disease control and significant MG-ADL score reduction.
• Nipocalimab, a monoclonal antibody, aims to reduce IgG autoantibodies, addressing the underlying cause of gMG without broad immunosuppression.
• Johnson & Johnson also submitted a Marketing Authorisation Application to the EMA, seeking approval of nipocalimab in gMG in Europe.
VYVDURA Approved in Japan for Chronic Inflammatory Demyelinating Polyneuropathy
• Japan's MHLW has approved VYVDURA (efgartigimod alfa and hyaluronidase-qvfc) for treating adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP).
• VYVDURA is the first and only neonatal Fc receptor (FcRn) blocker approved for CIDP treatment, administered as a weekly subcutaneous injection.
• The approval is based on the ADHERE study, which demonstrated a 61% reduction in relapse risk compared to placebo (p<0.0001) and clinical improvement in 69% of patients.
• This approval expands treatment options for CIDP patients in Japan, offering a convenient at-home self-injection alternative.
FcRn Inhibitors Show Promise in Autoimmune Disease Treatment, Market Set for Growth
• FcRn inhibitors are emerging as a targeted therapeutic approach for pathogenic IgG reduction, offering a less invasive alternative to traditional treatments for autoimmune diseases.
• Several companies, including Immunovant, Johnson & Johnson, and UCB Biopharma, are advancing FcRn inhibitors, with positive trial results and regulatory submissions driving market growth.
• Efgartigimod alfa (VYVGART) leads in the myasthenia gravis market, while nipocalimab and batoclimab show promise in trials for various IgG-mediated autoimmune disorders, including Graves' disease and CIDP.
• The FcRn inhibitor market is expected to grow significantly due to the increasing prevalence of autoimmune diseases and the potential for these drugs to address unmet needs in targeted IgG reduction.
UCB Pharma Eyes Further Expansion in South Korea Following Recent Drug Approvals
• UCB Pharma aims to expand its portfolio in South Korea after securing approvals for Bimzelx (bimekizumab) for plaque psoriasis and Zilbrysq (zilucoplan) for myasthenia gravis.
• Fintepla (fenfluramine) is under review for Dravet syndrome and Lennox-Gastaut syndrome, potentially receiving expedited approval via the GIFT program.
• Rystiggo (rozanolixizumab), a monoclonal antibody for myasthenia gravis, may also see accelerated approval if designated under Korea’s GIFT program.
• Bimzelx is poised to compete in the plaque psoriasis market, showing superior efficacy in trials against Humira, Stelara, and Cosentyx.
FDA Approves Rystiggo (rozanolixizumab-noli) for Generalized Myasthenia Gravis
• The FDA has approved Rystiggo (rozanolixizumab-noli) for treating generalized myasthenia gravis (gMG) in adults with specific antibody-positive subtypes.
• Rystiggo, a humanized IgG4 monoclonal antibody, reduces circulating IgG levels by binding to the neonatal Fc receptor (FcRN).
• Clinical trials demonstrated statistically significant improvements in MG-ADL and QMG scores with Rystiggo compared to placebo.
• The most common adverse reactions reported were headache, infections, diarrhea, pyrexia, hypersensitivity reactions, and nausea.
Bexicaserin Shows Sustained Seizure Reduction in Developmental Epileptic Encephalopathies
• Bexicaserin demonstrated a 59.3% median reduction in countable motor seizures over 12 months in patients with developmental and epileptic encephalopathies (DEEs).
• The PACIFIC open-label extension study showed consistent seizure reduction in both patients who switched from placebo to bexicaserin and those who continued treatment.
• Longboard Pharmaceuticals, now acquired by Lundbeck, is advancing bexicaserin into Phase 3 trials, including the DEEp SEA study for Dravet syndrome.
• Bexicaserin's favorable safety profile and tolerability were maintained throughout the 12-month extension, supporting its potential as a treatment for DEEs.
Inebilizumab Shows Promise in Myasthenia Gravis, Phase 3 Data Suggests
• Phase 3 MINT trial data indicates inebilizumab significantly improves Myasthenia Gravis Activities of Daily Living (MG-ADL) scores compared to placebo.
• The study included patients with both anti-acetylcholine receptor (AChR) and muscle-specific tyrosine kinase (MuSK) antibody-positive generalized myasthenia gravis (gMG).
• Amgen is planning regulatory submissions for inebilizumab as a potential new treatment option for generalized myasthenia gravis (gMG).
• Ongoing trials are exploring new therapeutic approaches, including bispecific nanoantibodies and complement inhibitors, to address unmet needs in gMG treatment.
Nipocalimab Shows Sustained Disease Control in Adolescents with Generalized Myasthenia Gravis
• Nipocalimab, combined with standard of care, demonstrates sustained disease control in adolescents (12-17 years) with generalized myasthenia gravis (gMG) over 24 weeks.
• The Phase 2/3 Vibrance-MG study shows a significant reduction in immunoglobulin G (IgG) levels and improvements in MG-ADL and QMG scores.
• Nipocalimab is well-tolerated, with no serious adverse events reported, offering a potential new treatment option for adolescents with gMG.
• Johnson & Johnson has submitted applications to the FDA and EMA seeking approval for nipocalimab for the treatment of gMG.
J&J's Nipocalimab Shows Promise in Treating Autoimmune Diseases
Johnson & Johnson reports positive outcomes for its experimental drug nipocalimab in treating two autoimmune diseases, Sjögren's disease and generalized myasthenia gravis (gMG), highlighting its potential as a blockbuster therapy.
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
© Copyright 2025. All Rights Reserved by MedPath