Caplacizumab-YHDP

CSU is a chronic skin disease driven partly by type 2 inflammation, leading to severe itching and hives. Credit: Pormezz / Shutterstock.com. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the approval of Sanofi and Regeneron’s Dupixent (dupilumab) in the European Union (EU) for paediatric patients with chronic spontaneous urticaria (CSU). The recommendation applies to children aged two to 11 years experiencing moderate-to-severe CSU who do not respond adequately to histamine-1 antihistamines and are naïve to anti-immunoglobulin E (IgE) therapy. It is based on results from the LIBERTY-CUPID clinical programme, including two Phase III trials (Study A and Study C) involving children aged six to 11 years, and the single-arm CUPIDKids Phase III study for children aged two to 11 years with CSU. A final decision is anticipated in the coming months. Dupixent is already approved for use in certain adults and adolescents with CSU in regions including the EU, Japan, and the US. In the US, a supplemental biologics licence application is under review to extend approval of Dupixent for children aged two to 11 years, with a decision from the US Food and Drug Administration (FDA) anticipated by April 2026. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence CSU is a chronic skin disease driven partly by type 2 inflammation, leading to severe itching and hives. Dupixent is a human monoclonal antibody targeting interleukin-4 and interleukin-13 pathways without serving as an immunosuppressant. It is being jointly developed by Sanofi and Regeneron and studied across over 60 clinical trials involving more than 12,000 patients with type 2 inflammation-driven diseases. Ongoing Phase III studies are evaluating further potential indications. In December 2025, China’s National Medical Products Administration (NMPA) granted approval for Sanofi’s rare haematologic disease treatments, Qfitlia (fitusiran) and Cablivi (caplacizumab), for patients with haemophilia and acquired thrombotic thrombocytopenic purpura (aTTP), respectively.

What are VHH antibodies?VHH antibodies, singledomain antibodies (sdAbs) or nanobodies, are tiny, highly stable antibody fragments. They are gaining attention, because they combine powerful biological activity with unique structural advantages that traditional antibodies dont have. Theyre small, stable, and highly engineerable, which makes them ideal for therapeutics, diagnostics, and even industrial applications. However, whilst they offer strong advantages for biological development, they also introduce complexities for development and manufacturing.The discovery of the distinctive structure of heavy chain-only antibodies in species belonging to the Camelidae family in 1993 created interest in their variable antigen binding domain (VHH). The first FDA approved VHH therapeutic; Caplacizumab (Cablivi) opened the door to many more programs. That interest has continued to build with five VHHbased therapeutics, gaining global regulatory approval by the end of 2025, and more than 49 VHH sequences had advanced into clinical development.In addition to indicators of growing clinical confidence and investment in next generation biologics, the global VHH nanobody market is growing, with CAGR estimates around 15%. This growth is driven by their promise for use in applications such as oncology, immunology, and diagnostic imaging.VHH antibodies have been shown to accelerate progress from concept to clinic when supported by the right development infrastructure. This infrastructure is key, as they differ fundamentally from conventional monoclonal antibodies, and so they bring distinct challenges in discovery, humanization, expression, and analytical characterization. However, there are differences in structure that give VHH antibodies their potential. Whats so different about VHH structure?VHHs antibodies differ from traditional antibodies in several critical ways:Small size - VHH antibodies are extremely small (1215 kDa), allowing them to access hidden epitopes that conventional antibodies cant. Their compact, hydrophilic structure enables improved tissue penetration, and they can even cross biological barriers such as the bloodbrain barrier.Simple but stable structure - VHHs consist of a single variable domain, making folding straightforward. They exhibit exceptional thermal and chemical stability, enabling use across challenging formulation conditions, including respiratory and digestive environments. This inherent stability also supports robust, efficient manufacturability.Flexible engineering - The lack of a light chain gives VHHs exceptional design flexibility for multispecific formats. Their unique paratope geometry creates an adaptable scaffold, enabling modular use as building blocks for complex therapeutics. VHHs can be readily fused with effector domains, including enzymes, toxins, and Fc regions, to create highly functional, customizable molecules.Low-cost manufacturing - Unlike conventional monoclonal antibodies that require mammalian expression, VHHs can be produced in simpler systems such as E. coli, yeast, and plants. This flexibility significantly shortens production timelines and can reduce overall process development costs.What are the challenges for VHH development and manufacturing?As this is an emerging technology, there are limited established CMC pathways compared to IgG monoclonals. Full-length monoclonal antibodies benefit from decades of GMP experience and well-defined regulations. This lack of clarity can prolong the development time for VHH antibodies. Discovery challenges can often occur in library generation and diversity, as although library-based sdAb discovery technology is well-developed, some limitations remain, such as sequence diversity loss, difficulties in conducting functional assays, and the considerable time required.Process development and manufacturing challenges can include issues around platform fit and expressions systems, due to the differences in the host system used. Scaling the process is often challenging as the small size of the VHH antibodies increases their risk of loss during filtration and ultrafiltration. In addition, tailored analytical methods may be required. Where next for VHH antibodies?VHH (singledomain) antibodies already have strong scientific momentum, but commercial success depends on:Discovery quality - VHH success begins with producing highquality binders that match or surpass fulllength antibodiesEngineering robustness - wellengineered VHHs can outperform IgGs on size, tissue penetration, and manufacturability, with careful designManufacturability - VHHs promise lower COGS, but only if manufacturing is optimizedRegulatory clarity clear regulatory pathways and comparability frameworks will be essentialVHH antibodies: theyre not just small; they are modular engineering platforms. The only limit is our imagination! '