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MSD’s WELIREG▼(belzutifan) made available for the treatment of eligible adults with von Hippel-Lindau (VHL) disease in Scotland

October 9, 2023 – Pharmaceutical – MSD, Merck, oncology

Scotland is the first European country to accept belzutifan as a treatment for adult patients with von Hippel-Lindau (VHL) disease who require therapy for VHL-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas or pancreatic neuroendocrine tumours (pNETs), and for whom localised procedures are unsuitable or undesirable.
In a phase II study, belzutifan demonstrated clinically meaningful antitumor activity with overall response rates of at least 64%, 44% and 91% in RCC, CNS and pNETs, respectively.
For patients, belzutifan could potentially reduce the need for risky, life-changing surgeries and help prevent them from developing tumour-related complications.

9 October 2023 — London, UK — MSD announced today that the Scottish Medicines Consortium (SMC) has accepted the use of WELIREG(belzutifan) within NHS Scotland as a treatment for adult patients with von Hippel-Lindau (VHL) disease requiring therapy for VHL-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas or pancreatic neuroendocrine tumours (pNETs), and for whom localised procedures are unsuitable or undesirable.

VHL disease is a rare hereditary disorder that occurs in approximately 1 in 27,300–39,000 live births. It affects patients of all ages, causing benign and malignant tumours and cysts to grow in different parts of the body. Tumours associated with this condition include RCC (in approximately 70% of patients), CNS and retinal hemangioblastomas, pNETs, adrenal gland tumours (phaeochromocytomas) and endolymphatic sac tumours.

Depending on the site of tumours, patients can suffer from debilitating constant pain, loss of balance and motor skills, loss of vision, breathlessness, coughing, headaches, confusion and severe nausea and fatigue.Loss of organ function can occur due to surgery which may also lead patients to experiencing life-altering complications including, but not limited to, requiring dialysis, insulin dependent diabetes and impaired digestive function.

Although survival has improved over time and life expectancy is now estimated to be 60–67 years, RCC and CNS tumour-related complications remain a common cause of VHL-related death and the interventions to manage different tumour types can be associated with significant impact on quality of life.This highlights the high unmet need for systemic medical treatment options that can control the disease and preserve quality of life by sparing patients from surgery or other localised procedures that may compromise organ function.

The SMC has accepted this treatment based on the available evidence and output from the Patient and Clinician Engagement (PACE) process with patient group representatives and clinical specialists, and after application of the appropriate SMC modifiers. This makes Scotland the first European country to accept belzutifan to treat VHL.

Graham Lovitt, chair of VHL UK/Ireland said: “VHL UK/Ireland is delighted to hear the wonderful news from the SMC today that certain qualifying VHL patients in Scotland will be the first in Europe to access belzutifan, fulfilling a huge unmet need for these VHL patients who until now, have had no medical treatment options and have had to rely on invasive surgery. We would like to thank the SMC for its clear guidance and its rapid and patient-focused appraisal process; the excellent contributing clinicians; and Action Kidney Cancer for their expert insights and support. We now wait with anticipation, for the outcome of the ongoing NICE appraisal, with hope for VHL patients in England and the rest of the UK.”

Professor Eamonn Maher, a clinical geneticist and expert in VHL disease, welcomed the decision to approve belzutifan. “It is excellent news that medical treatment is now an option to treat tumours in VHL disease in patients from Scotland and that they will be the first in Europe to have the opportunity to benefit from this drug, that was developed on the basis of fundamental scientific research discoveries, and which provides an exemplar of translational research and precision medicine.”

Mr Alex Laird, consultant Urological surgeon, Western General Hospital, Edinburgh said: “I’m delighted to be able to offer this treatment for appropriate patients with VHL disease. Belzutifan is an exciting treatment option for patients with VHL disease which could reduce the need for surgery in appropriate cases. This could significantly improve quality of life and outcome of patients.”

David Long, head of Oncology at MSD UK said: “We are delighted with SMC’s decision to accept belzutifan for use with eligible VHL patients. VHL can be a complex and, at times, devastating genetic disease, in which patients can also find themselves acting as carers for their loved ones who share their diagnosis. Until now, these patients have had few treatment options, typically limited to invasive procedures. This decision provides a welcome option for patients, reducing the need for and impact of numerous surgeries. We are committed to supporting NICE with their ongoing appraisal to ensure that patients from the rest of the UK are able to access this treatment option.”

Clinical trial data

The phase II LITESPARK-004 clinical trial, upon which this approval was based, involved 61 patients. The trial was designed to primarily assess the efficacy and safety of belzutifan in patients with VHL-associated RCC. Key data from the trial demonstrated that for patients with VHL-associated RCC tumours (n=61):

Overall, belzutifan demonstrated clinically meaningful antitumor activity after a median follow-up of over 3 years (37.8 months) with an overall response rate (ORR) of 64% (95% CI, 50.6–75.8).
The median time to response (TTR; n = 61) was 11.1 months (range 2.7–30.5).
The median time to surgery (TTS) could not be estimated, with only 7 patients requiring VHL-RCC-related surgery.
In patients with other VHL-associated non-RCC tumours, 50 patients with measurable CNS hemangioblastomas had an ORR of 44% and 22 patients with measurable pNETs had an ORR of 91%.

Common adverse events included anaemia, fatigue, headache, dizziness, nausea, dyspnoea, myalgia, constipation and arthralgia. 44% of patients experienced adverse events (AEs) of grade 3 or higher.

About MSD

At MSD, known as Merck & Co, in the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.msd-uk.com