Rilzabrutinib

In 2024, the European Medicines Agency received a record number of marketing authorization applications for advanced therapy medicinal products. Notable filings include UCB’s orphan drug for thymidine kinase 2 deficiency and Novo Nordisk’s Alhemo for hemophilia. The FDA approved 50 novel agents and 11 biologics. EMA recommended several new products for EU approval, including treatments for hereditary angioedema and transthyretin amyloidosis. Sanofi’s rilzabrutinib and Alvotech/Advanz Pharma’s golimumab are under review. EMA also reconsidered Eisai/Biogen’s Alzheimer’s drug for approval. The UK approved Eli Lilly’s Alzheimer’s drug, despite NICE's reservations.

Sanofi's recent achievements include Sarclisa's success in a phase 3 study, FDA Breakthrough Therapy for Tolebrutinib, Fast Track for influenza and COVID-19 vaccines, and Dupixent's EU approval for eosinophilic esophagitis. Sanofi also reported strong Q3 sales growth and strategic partnerships.

The IgG4-related disease market is expected to grow significantly by 2034, driven by increasing prevalence and awareness. Current treatments rely on off-label therapies, with emerging drugs like obexelimab and inebilizumab poised to revolutionize treatment. The US leads the market, with key players including Zenas BioPharma and Horizon Therapeutics.

The IgG4-related disease treatment market is set to grow significantly by 2034, driven by increasing prevalence, awareness, and the launch of new therapies. Current treatments rely on off-label drugs like glucocorticoids and rituximab, with no approved therapies specifically for IgG4-RD. Key players like Zenas BioPharma and Horizon Therapeutics are advancing treatments, with promising pipeline drugs expected to revolutionize the market. The 7MM market size was around USD 150 million in 2023, with the US leading. The disease, affecting mainly middle-aged to elderly men, presents challenges in diagnosis and treatment, emphasizing the need for early intervention to prevent organ damage.

The IgG4-related disease market, involving companies like Zenas BioPharma and Sanofi, is projected to grow significantly by 2034, driven by increasing prevalence and awareness. Current treatments rely on off-label therapies, with emerging drugs like obexelimab and inebilizumab expected to revolutionize treatment patterns. The US leads the market, with a focus on addressing unmet medical needs and improving patient outcomes.

Principia Biopharma Inc., a Sanofi company, completed patient enrollment for the global Phase 3 PEGASUS trial of rilzabrutinib, an oral BTK inhibitor, for treating pemphigus. The trial aims to evaluate rilzabrutinib's efficacy in achieving durable complete remission in pemphigus patients, with potential for a 24-week open-label extension. Rilzabrutinib targets immune mediated diseases by blocking inflammatory responses without depleting B cells.

Hemolytic anemias include PNH, aHUS, and wAIHA. Anti-C5 therapy is key for PNH and aHUS. New treatments target upstream complement cascade components. Novel therapies for wAIHA include fostamatinib and sovleplenib.

Sanofi and Teva's Phase IIb RELIEVE UCCD study of duvakitug for ulcerative colitis (UC) and Crohn’s disease (CD) met primary endpoints, showing significant remission rates. UC patients achieved 36.2% (low dose) and 47.8% (high dose) remission; CD patients showed 26.1% and 47.8% endoscopic response. Duvakitug was well-tolerated, with no major safety concerns. Sanofi and Teva will equally share development costs and profits in major markets.

Rilzabrutinib showed superior efficacy over placebo in treating ITP, with 65% of patients achieving platelet response compared to 33% on placebo. It met primary and secondary endpoints, including durable platelet response, reduced bleeding, and improved quality of life. Presented at ASH 2024, its safety profile was consistent with previous studies.

ASH 2024 highlights include GSK's Blenrep Phase 3 data supporting re-market, J&J's Darzalex Faspro delaying progression in smoldering multiple myeloma, Arcellx's anito-cel showing comparable safety to Carvykti, and J&J/Legend's Carvykti improving survival outcomes. Kura Oncology's ziftomenib showed high response rates in leukemia, Eli Lilly's Jaypirca reduced disease progression risk, and Merck's zilovertamab vedotin achieved high complete response rates in lymphoma. Beam Therapeutics' BEAM-101 showed durable effects in sickle cell disease, Novo Nordisk's etavopivat reduced crises, and Bristol Myers Squibb's arlo-cel demonstrated durable responses in multiple myeloma. Galapagos' GLPG5101 showed encouraging cell therapy results, Orca Bio's Orca-T improved survival in blood cancers, and Sanofi's rilzabrutinib improved platelet response in thrombocytopenia. Regeneron's drug combo showed better disease control than Ultomiris in PNH.