NICE publishes draft final guidance recommending Aspaveli™▼ (pegcetacoplan) in adults with paroxysmal nocturnal haemoglobinuria in England & Wales

NICE publishes draft final guidance recommending Aspaveli™▼ (pegcetacoplan) in adults with paroxysmal nocturnal haemoglobinuria in England & Wales 

Sobi UK has today announced that the National Institute for Health and Care Excellence (NICE) has issued the Final Appraisal Decision (FAD) recommending the use of Aspaveli™ (pegcetacoplan) in adults with paroxysmal nocturnal haemoglobinuria (PNH) in England and Wales. The Final Appraisal Document (FAD), published today following a Fast Track Appraisal by NICE, recommends Aspaveli as an option for treating adults with PNH who are anaemic after treatment with a C5 inhibitor for at least three months, once licensed in the UK. Aspaveli is currently undergoing assessment with the Medicines and Healthcare products Regulatory Agency (MHRA) for a marketing authorisation in the UK.

• PNH is a rare, chronic, life-threatening blood disorder, affecting approximately 900 people in England^[i]
• Characterised by persistently low haemoglobin, PNH can result in frequent blood transfusions and debilitating symptoms such as severe fatigue (96% of cases[ii]) and anaemia (88% of cases[iii]). If left untreated, it has a 10-year mortality rate of 29%[iv], with 40-67% of deaths caused by thrombosis[v]
• Despite improvements in haemolytic activity with C5 inhibitor treatment, to date focus has been on disease survival. Consequently patients can still experience a substantial life-limiting and debilitating burden of illness^[vi], with approximately 80% of C5-treated patients exhibiting fatigue and 72% of C5-treated patients remaining anaemic.^6,[vii] Around a quarter (20 – 36%) also remain transfusion-dependent^7,[viii]^,[ix]^,[x]
• Living with the symptoms of PNH, such as fatigue and anaemia, can have a significant impact on quality of life.⁶  Over a third of patients (37.5%) report PNH as causing impairment in normal daily activities and over a quarter (26.7%) report PNH as causing impairment at work[xi]

“Pegcetacoplan is a valuable step forwards in treatment for patients with PNH.  Despite recent treatment advances, some patients with PNH who are being treated with the current standard of care experience low haemoglobin levels and extravascular haemolysis, causing anaemia and fatigue.  This adversely affects their quality of life and can lead to blood transfusion requirement” said Dr Morag Griffin, Consultant Haematologist at Leeds Teaching Hospitals NHS Trust. “Pegcetacoplan has demonstrated clinically meaningful improvements in key markers of disease in patients with PNH, and therefore presents an important new therapeutic option.”

“Pegcetacoplan, in clinical trials has proven to improve anaemia and manage ongoing haemolysis for patients with PNH.  The subcutaneous self-administered infusion of pegcetaoplan also provides patients with alternative modality of treatment, addressing an unmet need in PNH for those with limited venous access.” said Dr Richard Kelly, Consultant Haematologist at Leeds Teaching Hospitals NHS Trust.

“This decision is an important milestone for people living with PNH in England and Wales, who we hope will now be among the first in Europe to be able to access this treatment” said Michael Oliver, General Manager at Sobi UK & RoI. “Until now, real world evidence studies indicate that many patients receiving treatment continue to experience debilitating symptoms, such as anaemia-related fatigue, which significantly impact their quality of life and result in them having to adjust to a ‘new normal’.  This recommendation paves the way for an alternative treatment option which can help tackle this unmet need.”

The draft final guidance follows results from the head-to-head phase 3 PEGASUS study, which evaluated the efficacy and safety of pegcetacoplan compared to eculizumab at 16 weeks in adults with PNH who had persistent anaemia despite treatment with eculizumab. In addition to finding an improvement in haemoglobin levels, the trial also found a significant decrease in transfusion requirements.[xii] Additionally, there was a clinically meaningful improvement in FACIT fatigue scores and sustained clinically meaningful global health status/QoL scores. ¹³ The results were published in The New England Journal of Medicine in March 2021.¹³

About the PEGASUS study
PEGASUS (APL2-302; NCT03500549) was a multi-centre, randomised, head-to-head phase 3 study in 80 adults with paroxysmal nocturnal haemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of Aspaveli® (pegcetacoplan) compared to eculizumab.

Aspaveli®/Empaveli™ (pegcetacoplan)

Aspaveli®/Empaveli™ (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Empaveli is approved in the United States for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for Aspaveli for the treatment of adults with PNH who are anaemic after treatment with a C5 inhibitor for at least three months. The positive opinion from the CHMP is now referred to the European Commission for an approval decision. The therapy is also under investigation for several other rare diseases across haematology, nephrology, and neurology.

Aspaveli® is currently undergoing assessment with the Medicines & Healthcare products Regulatory Agency (MHRA) for a marketing authorisation in the UK. Aspaveli™ has also been submitted for health technology assessment in Scotland.

About the Sobi and Apellis Collaboration

Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-US commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive US commercialisation rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA).

About Sobi™

At Sobi, we are transforming the lives of people affected by rare diseases. As a specialised biopharmaceutical company, we provide sustainable access to innovative therapies in the areas of haematology, immunology and specialty care. We bring something rare to rare diseases – a rare expertise and a strength in access that allows us to be a partner in care for those with high unmet needs. Sobi headquarters are located in Stockholm, Sweden. You can find more information about Sobi in the UK and the Republic of  Ireland at www.sobi-uk.co.uk

For more information, please contact

Madeleine Harris Smith, Patient Advocacy and Communications Manager
+44 (0) 7443 191 726
madeleine.harrissmith@sobi.com