Anal cancer: UK’s first immunotherapy treatment for eligible anal cancer patients approved for licensing and reimbursement

Incyte Biosciences UK, today announced that the Medicines and Healthcare products Regulatory Agency (MHRA) and National Institute for Health and Care Excellence (NICE) have approved the license and reimbursement of Zynyz® ▼ (retifanlimab) in combination with carboplatin and paclitaxel for the first-line treatment of inoperable, locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC).

The approval and reimbursement were supported by the first positive Phase 3 trial of a systemic therapy completed in this advanced setting and represents the first immunotherapy treatment licensed for the treatment of anal cancer in the UK.

This milestone also comes at a time of renewed national focus for improving outcomes for people living with rare cancers, with the Government’s National Cancer Plan for England published in February along with the appointment of a National Clinical Lead for Rare Cancers.[v] This demonstrates the commitment to ensure that rare cancers receive greater parity within cancer policy and improved access to care and treatment.

The MHRA regulatory review and NICE health technology assessment progressed in parallel. This coordinated regulatory approach supported timely decision-making on both the medicine’s license and reimbursement for recommended NHS use, helping accelerate patient access to this first and only immunotherapy option for eligible patients in an area that has seen few treatment advances.

Squamous cell carcinoma of the anal canal (SCAC) is a cancer that starts in the squamous cells that line the anal canal, the end portion of the large bowel. Squamous cell carcinomas are the most common type of anal cancer, accounting for more than 80% of all anal cancers.[vi] Around 1,700 people are diagnosed with anal cancer each year in the UK, and of those roughly 6 in 10 cases occur in women.[vii] Whilst anal cancer is rare, its impact is significant and incidence is rising – with cases in the UK projected to increase by around 13% between now and 2038-2040.[viii]

Living with anal cancer can have a significant physical and psychosocial impact on the daily lives of patients. Lack of awareness and misunderstanding of risk factors associated with this cancer can lead some anal cancer patients to feel socially isolated, embarrassed or unwilling to seek support. Additionally, patients undergoing radiotherapy, which many anal cancer patients receive as part of their treatment, can experience a high burden of treatment-related side effects which can continue to impact them long after their treatment is complete.

Michael Anderson, CEO and Co-Founder of Bottom Line, a support group for people living with anal cancer in the UK, said: “The anal cancer community has been waiting a very long time to see meaningful innovation in the treatment of advanced disease and the standard of care has remained unchanged for years. This milestone decision brings much-needed hope to the patient community and is a welcome development for healthcare professionals and clinicians who have long sought additional treatment options.”

“As incidences of anal cancer are rising, this represents an important step forward for the community and all those affected. We now look forward to seeing patients across the UK benefit from timely and equitable access.”

Dr Sheela Rao, Consultant Medical Oncologist and Chief Investigator of the POD1UM-303/InterAACT2 trial at The Royal Marsden NHS Foundation Trust, said: “The POD1UM-303/InterAACT2 study was the largest Phase 3 trial to evaluate an immunotherapy in advanced squamous cell anal carcinoma, and the first to report positive results, including reducing risk of death by 25% which is hugely significant. The positive approval and license of retifanlimab in combination with chemotherapy therefore represents an important milestone for the anal cancer community.”

“In a disease setting where treatment options have long been limited, I am pleased to see this treatment become available through the NHS and proud that the work of patients, investigators and study teams involved in the trial has contributed to bringing this advance to clinical practice.”

David Winterflood, Chief Executive Officer of the Anal Cancer Foundation UK, said: “Patients with anal cancer often face a troubling lack of public awareness and understanding when it comes to risk factors, symptoms and their overall cancer journey. This approval. marks a step forward for advanced SCAC treatment, brings attention to a long-overlooked condition with limited treatment options and offers patients an option to treat their disease.”

Pete Williams, General Manager, Incyte Biosciences UK & Ireland, said: “We are delighted that retifanlimab has been approved for eligible adults with advanced anal cancer in England, providing an important new treatment option for a patient community that has historically had limited therapeutic advances. This approval is a significant milestone, not only for patients and clinicians, but also for everyone who has contributed to advancing research in this rare and challenging disease.”

“The decision reflects the benefits of close collaboration between Incyte, the MHRA, NICE and NHS England, helping to accelerate patient access to innovation. At Incyte, we remain committed to investing in science, research and development to bring meaningful new treatment options to patients, particularly in areas of high unmet need where progress has been difficult to achieve. We now look forward to ensuring eligible patients can benefit from access to retifanlimab as quickly as possible.”

The approval is based on data from the Phase 3 POD1UM-303/InterAACT2 trial (NCT04472429), evaluating retifanlimab or placebo in combination with platinum-based chemotherapy (carboplatin and paclitaxel) in more than 300 adult patients with metastatic or inoperable locally recurrent SCAC not previously treated with systemic chemotherapy.[ix] Results from the trial demonstrated that adding retifanlimab to standard-of-care chemotherapy cut the risk of death by 25% (median overall survival 32.8 months versus 22.2 months; hazard ratio 0.75, 95% confidence interval 0.55 to 1.01), a striking result in a disease where five-year survival for metastatic patients has historically sat around 30%.