Zilucoplan

Inozyme's INZ-701 Shows Promise in ENPP1 Deficiency Trial with Sustained Phosphate Increases

8 days ago

• Interim data from Inozyme Pharma's ENERGY 3 pivotal trial demonstrates INZ-701's potential to modify disease course in pediatric ENPP1 Deficiency patients, with sustained phosphate increases and favorable safety profile. • The trial is progressing well with no patient dropouts, dose adjustments, or discontinuations, and remains on track for topline data in Q1 2026, positioning INZ-701 to potentially become the first approved therapy for this rare disease. • Inozyme has appointed Petra Duda, M.D., Ph.D. as Chief Medical Officer, bringing over two decades of expertise in rare disease clinical development as the company advances toward late-stage development milestones.

Eculizumab Shows Promise in Treating Thymoma-Associated Myasthenia Gravis

4 months ago

• 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

4 months ago

• 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

5 months ago

• 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.

UCB Pharma Eyes Further Expansion in South Korea Following Recent Drug Approvals

6 months ago

• 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.

Inebilizumab Shows Promise in Myasthenia Gravis, Phase 3 Data Suggests

8 months ago

• 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.

J&J's Nipocalimab Shows Promise in Treating Autoimmune Diseases

a year ago

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.