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It’s not because it’s ‘Rare’ that we care

Posted by
Chris Clark, Patient Value Unit - Neurology
UCB is a company with a commitment to patient value at our core. We're structured to discover, and develop new medicines and innovative approaches to care quickly and efficiently, taking into account unique perspectives, experiences and requirements of the people living with severe and chronic conditions.

By embracing this approach, we aim to connect targeted patient populations to cutting-edge science and, in doing so, deliver improved, differentiated experiences to people with the highest unmet needs.

One of our newest medicines in development, rozanolixizumab, is a novel biologic therapy being investigated for the treatment of multiple IgG (immunoglobulin G) autoantibody-mediated diseases. Currently available treatments are able to partially stabilize IgG autoantibody-mediated diseases. However, they can be invasive, burdensome, costly, and can negatively impact a person’s quality of life.

The development of rozanolixizumab exemplifies our approach to researching new medicines: We’re listening to and learning from people with first hand experiences of IgG autoantibody-mediated diseases and applying this knowledge to address some of the significant unmet needs they face.

We are leveraging and growing our capabilities to deliver value for specific patient populations new to UCB – for example, people with Myasthenia Gravis (MG) and Idiopathic Thrombocytopenic Purpura (ITP). And, while MG and ITP have IgG autoantibody-mediation in common, they are quite distinct conditions each with unique patient experiences. We are striving to understand the unique factors which differentiate individual patient experiences for people with these conditions and tailoring our solutions to solve for these unique needs.

The FDA has recently awarded rozanolixizumab orphan drug status for MG – building on a similar orphan drug designation granted to rozanolixizumab in ITP by the FDA and  European Medicines Agency in 2018. These decisions by regulators reinforce our strategy to bring new and innovative medicines which address well defined patient populations. Importantly, through these designations, the regulatory authorities recognize the potential of UCB’s unique science driven approach, and the impact our research and development activities could have for these very specific patient groups.

Development of rozanolixizumab dovetails perfectly with our overarching company strategy. We are committed to creating patient preference by responding to the specific needs of increasingly well-defined patient populations suffering from IgG-mediated disease, whether their numbers are small or large.

Many of these patient populations are supported by organizations which collectively will be marking International Rare Disease day on 28th February. Their activities will further highlight the importance of improving our knowledge and understanding of these conditions, the significant unmet need which unfortunately exists for many people, as well as the importance of our continuing to progress R&D efforts.

Currently, we’re proud to be actively engaging with patient and advocacy organizations around the world: Bolstering our knowledge, centering our activities on differentiating individual experiences, and ensuring the views of people who could one day benefit from rozanolixizumab are taken into account as early as possible. We are opening up new ground where previously our specific experience was limited - creating a reference point for these targeted patient groups at UCB and building authentic relationships that will be foundational in our aspiration to be leaders for the populations we serve.

We believe that our science, combined with our commitment to understanding the real unmet needs of patients, provides us with an opportunity to go far beyond current treatments. By redefining treatment goals and patient experiences, we have a unique and exciting opportunity to make a real difference.

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