Novartis has achieved significant regulatory successes on two fronts, with its oral therapy Rhapsido (remibrutinib) receiving marketing authorisation from the European Commission (EC) for specific patients with chronic spontaneous urticaria (CSU), and its antimalarial medication, Coartem Baby (artemether-lumefantrine), earning prequalification status from the World Health Organization (WHO). These dual approvals underscore Novartis’s commitment to addressing unmet medical needs across diverse therapeutic areas, from chronic inflammatory conditions to critical infectious diseases.
The European Commission’s decision marks a pivotal moment for Rhapsido, establishing it as the first oral treatment option for adult CSU patients who have not found adequate relief with first-line H1-antihistamine therapies. This oral therapy, taken twice daily, represents a novel approach to managing a condition characterized by persistent, itchy hives and swelling, often significantly impacting patients’ quality of life. Rhapsido functions as a Bruton’s tyrosine kinase (BTK) inhibitor, a class of drugs designed to modulate immune responses by disrupting signaling pathways crucial for the release of histamine and other inflammatory mediators that drive urticarial symptoms.
Chronic spontaneous urticaria is a debilitating condition affecting an estimated 0.5% to 1% of the global population, characterized by the sudden appearance of hives (wheals) and/or swelling (angioedema) that persist for six weeks or longer, without an identifiable external trigger. The underlying pathophysiology is complex but largely involves the activation of mast cells in the skin, leading to the release of histamine and a cascade of inflammatory events. For many patients, conventional antihistamines provide insufficient relief, leaving them with chronic symptoms that can disrupt sleep, work, and social interactions.
The European approval for Rhapsido is supported by robust clinical trial data from two pivotal Phase III studies, REMIX-1 (NCT05030311) and REMIX-2 (NCT05032157). These trials collectively enrolled 926 adult CSU patients who were refractory to H1-antihistamines. The results demonstrated that Rhapsido significantly improved both itch and hive symptoms, offering substantial relief to patients. Importantly, the drug’s favorable safety profile was also highlighted, with studies indicating that no routine laboratory monitoring is required. Common adverse events observed in the trials included nasopharyngitis (nasal congestion, sore throat, and runny nose), suggesting a generally well-tolerated treatment.
This European marketing authorisation follows closely on the heels of a similar regulatory milestone in the United States, where the Food and Drug Administration (FDA) approved Rhapsido for the same patient population in September 2025. This parallel regulatory progression in major markets underscores the drug’s perceived clinical value and the scientific rigor behind its development.
Novartis’s entry into the CSU market with an oral BTK inhibitor positions the Swiss pharmaceutical giant in direct competition with established injectable biologic therapies. Notably, Sanofi and Regeneron’s Dupixent (dupilumab) and Roche’s Xolair (omalizumab) are currently leading treatments for moderate-to-severe CSU. These therapies, while effective for many, require subcutaneous injections, which can present adherence challenges and patient preference issues. Rhapsido’s oral administration offers a potential competitive advantage, providing a more convenient and potentially preferred treatment option that could allow Novartis to capture significant market share from these blockbuster injectable therapies.
Industry analysts are optimistic about Rhapsido’s commercial prospects. GlobalData’s Pharma Intelligence Centre forecasts that global sales for Rhapsido in the CSU indication are projected to reach an impressive $2.6 billion by 2032. This projection reflects the significant unmet need in the CSU market and the potential of an oral therapy to address this demand effectively. GlobalData is the parent company of Pharmaceutical Technology.
Beyond its current indication, Rhapsido’s therapeutic potential is being further explored. Novartis is investigating the drug’s efficacy in treating multiple sclerosis (MS), a chronic, inflammatory disease of the central nervous system. A Phase III trial (NCT05156281) is currently underway to evaluate Rhapsido’s impact on MS progression. Furthermore, research is ongoing to assess its utility in other immune-mediated conditions, including chronic inducible urticaria, food allergy, and hidradenitis suppurativa (HS), suggesting a broad potential for this BTK inhibitor across various inflammatory and autoimmune disorders.
WHO Prequalifies Novartis’s Antimalarial Coartem Baby
In parallel with the Rhapsido approval, Novartis has received World Health Organization (WHO) prequalification for Coartem Baby, a critical antimalarial medication specifically formulated for newborns and infants. This WHO endorsement is a crucial step towards expanding access to this life-saving treatment in malaria-endemic regions, particularly for the most vulnerable patient population. Coartem Baby is the first antimalarial treatment designed and dosed for infants weighing between 2 kilograms and 5 kilograms, addressing a significant gap in pediatric malaria care.
The WHO prequalification process is a rigorous evaluation of a medicine’s safety, efficacy, and quality. This independent assessment ensures that treatments meet international standards, providing confidence to public health organizations, procurement agencies, and healthcare providers. For diseases like malaria, HIV/AIDS, and tuberculosis, WHO prequalification is a vital component of global health initiatives, informing purchasing decisions by entities such as the United Nations and various global health funds.
Coartem Baby, also known as Riamet Baby in some markets, was developed through a strategic partnership between Novartis and Medicines for Malaria Venture (MMV). This collaboration exemplifies a successful public-private partnership aimed at tackling a major global health challenge. The WHO prequalification will facilitate wider public sector procurement, enabling greater availability and affordability of Coartem Baby in regions where malaria poses a significant threat to infant mortality.
Novartis has committed to supplying Coartem Baby on a primarily not-for-profit basis in malaria-affected areas, reinforcing its dedication to global health equity. Dr. Lutz Hegemann, Novartis Global Health President, expressed his enthusiasm, stating, "This decision takes us one step closer to ensuring that the tiniest babies have access to the first antimalarial designed specifically for them. We have already introduced the treatment in Ghana and are pleased to now be going further together with our partners to reach even more of the smallest malaria patients."
The development of Coartem Baby was a key initiative within the PAMAfrica consortium, which received vital funding from the European & Developing Countries Clinical Trials Partnership and the Swedish International Development Cooperation Agency. This collaborative effort highlights the importance of international funding and partnerships in advancing medical solutions for diseases disproportionately affecting low-resource settings.
Novartis has a long-standing commitment to global health, with significant investments in research and development for neglected diseases. Since 2021, the company has invested over $500 million in global health R&D, including the pursuit of new compounds to combat drug-resistant malaria. This investment builds upon Novartis’s extensive track record in malaria control; since 1999, the company has delivered more than 1.1 billion Coartem treatment courses globally.
The dual regulatory achievements for Rhapsido and Coartem Baby underscore Novartis’s strategic focus on both innovative therapeutics for developed markets and essential medicines for global health priorities. This balanced approach allows the company to leverage its scientific expertise and manufacturing capabilities to address a wide spectrum of patient needs.
In a related development, Novartis recently announced an agreement to acquire the biotech business Excellergy, specializing in next-generation anti-immunoglobulin E (IgE) therapies, for up to $2 billion in upfront and milestone payments. This acquisition signals Novartis’s continued investment in immunology and its pursuit of novel treatment modalities for allergic and immune-mediated diseases, further broadening its portfolio and therapeutic reach. These recent regulatory wins and strategic acquisitions highlight a dynamic period for Novartis, demonstrating its ongoing drive to innovate and expand access to critical medicines worldwide.
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