Serving Sickle Cell Disease Patients in Europe: Nigel Nicholls, GBT

Newly appointed in his role as General Manager (UK) at Global Blood Therapeutics (GBT), a biopharma company focused on meeting the needs of underserved Sickle Cell Disease (SCD) patients, Nigel Nicholls has a strong record of achievement in the clinical and commercial development of orphan drugs. He began his career at KMPG, before moving into pharma with a focus on rare diseases. His previous roles include Area Director (UK/Ireland) for BioMarin, Country Manager (UK/Ireland) for Jerini AG and Business Unit Head at Alliance Pharma.

Nicholls is committed to addressing gaps in awareness and challenges patients face in achieving access to new treatments and technologies in the EU. His recent achievements include spearheading BioMarin’s Childhood Dementia campaign in collaboration with patient organizations (winning a 2020 Communique award) and driving corporate leadership with KOLs, patient organizations and industry bodies.

Fighting for the rights of those facing disparities in care hits close to home for Nicholls, whose son is living with a rare disease. This personal connection drives his commitment to working on behalf of patients and families affected by rare diseases.

Speaking to Pharmaceutical Executive, Nicholls provides an overview of his role as a General Manager for GBT, the company’s vision for Europe and the UK, and the health inequity faced by the SCD community.

Pharm Exec: Tell us about your new role as UK General Manager at GBT, and your passion and understanding of SCD.

Nigel Nicholls: As a father of a son living with a rare disease, I’ve always been drawn to roles where I can make a difference to underserved patient communities, which is why I was attracted to the opportunity to lead GBT in the UK. GBT is a biopharmaceutical company dedicated to discovering, developing, and delivering life-changing treatments for people living with grievous blood disorders, starting with SCD, a rare genetic condition affecting approximately 15,000 people in the UK.

Although the fundamental cause of SCD has been understood for decades, therapeutic innovation and access to care have significantly lagged. In recent years, however, industry has picked up its efforts and GBT, in particular, has been working to advance its pipeline to bring hope to the community that has long been put out of focus for both industry and society more widely. For me this is an opportunity to create change. GBT focuses on developing and delivering medicines that could be life-changing for the predominantly African and Caribbean communities who are disproportionately affected by SCD. What’s different about GBT is that we were not built around a molecule or medicine, we were built around the urgency to address an unmet need in SCD. I am passionate about this purpose.

What inspires you about GBT and how can GBT make its voice heard in Europe?

The SCD community deserves innovative treatments that address the underlying cause of this debilitating disease.

SCD has a devastating impact on the lives of those it affects and their families, including serious and life-threatening complications that can lead to organ damage and early death. Yet, despite this, there remains a lack of urgency to treat SCD, and healthcare disparities based on race persist.

Since it was founded 10 years ago, GBT set out to address the urgent needs of the community, driven by this historical injustice and lack of understanding and attention given to SCD. GBT wants to shine a light on those living with SCD, to highlight the key policies that can benefit them and to start an important conversation with stakeholders to address the inequity faced by these communities in the UK and in Europe.

GBT has the largest of all SCD pipelines. Its lead medicine, voxelotor, is a first-in-class oral, once-daily therapy that directly inhibits haemoglobin S polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes haemolytic anemia (low haemoglobin due to red blood cell destruction), which impairs adequate oxygen delivery to the tissues and organs in the body. Voxelotor is approved in the United States under the trade name of Oxybryta® for the treatment of SCD in patients ages 12 years and older. GBT is seeking regulatory approval from the European Medicines Agency (EMA) for voxelotor in the treatment of haemolytic anemia in SCD patients ages 12 years and older.

Tasked with building GBT in the UK, what is the status of your efforts to address health disparities and access challenges for these patients?

SCD patients need and deserve innovative therapies and high-quality care, and GBT is committed to supporting patient access. In the UK, the Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a Promising Innovative Medicine (PIM) designation for voxelotor for the potential treatment of hemolytic anemia in adult and adolescent patients 12 years of age and older with SCD.

This is important because PIM designations are given to promising treatments that are likely to offer a major advantage for patients and are an early indication that the treatment is a promising candidate for the Early Access to Medicines Scheme (EAMS). Voxelotor is the first SCD treatment to receive the PIM designation and this is a significant milestone in our efforts to potentially make this therapy available in the UK.

Prior to potential marketing authorization, GBT initiated an early access program for voxelotor in Europe and other regions outside the United States, which enables physicians to use early access regulatory and legal pathways to request voxelotor for the treatment of hemolytic anemia in eligible patients with SCD who do not have access to the medicine as part of a clinical trial.

These are important steps forward on our journey, and we remain committed to developing novel treatments with the hope of transforming the lives of those living with SCD.

What are your hopes for the future with regard to improving treatment outcomes for patients with SCD?

Beyond voxelotor, GBT has a robust pipeline and is seeking to address SCD from multiple approaches. GBT is advancing next-generation investigational therapies for SCD to transform the treatment of this devastating lifelong disease – both through internal R&D and via collaborations with academic centers and industry experts in the field throughout the globe. Our SCD development pipeline is focused on the development of innovative small molecules and biologics aimed at addressing multiple pathologies of the disease including hemoglobin polymerization and vascular inflammation.

At GBT, we are committed to fostering workplace development, diversity, and inclusion at our company and throughout the biotech industry. This is an important part of our future and our ability to impact SCD patients’ lives is the strength of our company culture. As General Manager of GBT in the UK, empowering my team with our GBT vision, we are determined to shine a light on those living with SCD and address the inequalities faced by this community.

How are you partnering with the patient community and what is the shared vision that you bring in terms of leading for the future?

For many years, GBT has been acutely focused on the SCD patient community. GBT is collaborating with the SCD community, including healthcare professionals, patient advocacy groups and community-based organizations to increase awareness of SCD and how it affects patients.We are working to change the treatment paradigm in this disease, while helping the sickle cell community advocate for quality care. At GBT, we are focused not just on developing innovative treatments for SCD but on changing how people with the disease are treated and cared for. GBT’s deep commitment to partnering with, and advocating for the sickle cell community, has been our guiding light since the beginning.

In the UK, recent high-profile examples of failings in care for people with sickle cell disease have led to growing awareness of the challenges sickle cell patients still too often face in receiving appropriate care. It is clear from testimony from healthcare professionals, sickle cell patients and their families over many years that these recent high-profile cases are not isolated incidents. In the UK, the inquiry by the All-Party Parliamentary Group (APPG) on Sickle Cell and Thalassemia will examine the care sickle cell patients receive, seek to identify why care for sickle cell patients is too often sub-standard, and look to ensure that recent tragic cases lead to national change to ensure that such failings are not repeated in the future.

Ultimately, we are all working together to ensure these outcomes deliver meaningful benefit for the SCD community which deserves society’s attention, industry’s commitment to innovation, and most importantly, access to the very best care. It really is all about elevating the patient voice and initiating important conversations so that the inequalities faced by the SCD communities in the UK and Europe are addressed.

Further reading

Profile of Ted W. Love, CEO of Global Blood Therapeutics

Education Mission: Oxbryta