NICE Approved Ripretinib for Advanced GISTs: A New Fourth-Line Treatment Option

NICE Approved Ripretinib for Advanced GISTs: A New Fourth-Line Treatment Option

On March 31, 2026, Sarcoma UK and GIST Cancer UK announced that the National Institute for Health and Care Excellence (NICE) has approved ripretinib (Qinlock®) for use within NHS England. This decision introduces a new treatment option for patients with advanced gastrointestinal stromal tumors (GISTs) who have exhausted three or more prior kinase inhibitor therapies.

Ripretinib will provide a vital fourth-line treatment option for GIST patients after they have exhausted the currently available three lines of therapy (imatinib, sunitinib and regorafenib).

Clinical evidence highlighted in the announcement indicates that ripretinib can both extend life and improve quality of life, with reduced side effects compared to existing therapies.

Epidemiology of Gastrointestinal Stromal Tumors

According to the American Cancer Society, gastrointestinal stromal tumors (GIST) are rare and were only recognized as a distinct tumor type in the late 1990s. Approximately 6,000 new cases are diagnosed each year in the United States.

GISTs can arise anywhere along the gastrointestinal tract but are most commonly found in the stomach (about 60%) and the small intestine (around 35%). Less frequently, they occur in the esophagus, colon, or rectum, and in rare cases, they may develop outside the gastrointestinal tract within the abdomen.

These tumors affect men and women equally and are most often diagnosed in individuals over the age of 50. Although uncommon in people younger than 40, they can occur at any age.

In some patients, GIST is associated with inherited genetic alterations. Mutations in KIT or PDGFRA genes can lead to familial GIST syndrome, while other genetic changes, including SDH or NF1, may also increase the risk of developing these tumors.

GIST

Read more about Gastrointestinal Stromal Tumors on OncoDaily.

What is Ripretinib and How does it work?

Ripretinib, developed by Deciphera Pharmaceuticals, is a targeted cancer therapy used in patients with advanced gastrointestinal stromal tumors (GIST), particularly after progression on multiple prior treatments. It belongs to a class of drugs known as tyrosine kinase inhibitors (TKIs), which block signaling pathways that cancer cells rely on for growth and survival.

In GIST, tumor growth is primarily driven by abnormal activation of KIT and PDGFRA proteins. Ripretinib is designed to broadly inhibit these kinases, including secondary mutations that may develop resistance to earlier therapies, thereby helping to control disease progression in later lines of treatment.

In simple terms, ripretinib works by:

  • Blocking abnormal growth signals in cancer cells
  • Targeting KIT and PDGFRA signaling pathways
  • Inhibiting multiple mutations, including resistant forms
  • Slowing down or stopping tumor progression

By acting on these pathways, ripretinib provides an important treatment option for patients who have already received standard therapies.

A Decision Supported by Clinical Evidence and Patient Voice

Both Sarcoma UK and GIST Cancer UK have actively participated in the NICE appraisal process, submitting written evidence about patient experiences and supporting the case for approval throughout.

Expert Perspective

Richard Davidson, Chief Executive of Sarcoma UK, stated:

“We are delighted that NICE has made this positive recommendation, which is excellent news for GIST patients in England who have had very limited treatment options after progressing through the three currently approved therapies. Ripretinib offers real hope for extended survival with fewer side-effects. After exhausting existing treatments, these patients may have just weeks to live. This treatment can provide both extended survival and improved quality of life for patients with a rare cancer like GIST.”

Richard davidson

Richard Davidson, Chief Executive of Sarcoma UK

He further added:

“Sarcoma UK’s 2020 National Sarcoma Survey found that 95% of GIST patients reported that their diagnosis and treatment negatively affected their mental health and emotional wellbeing. A fourth-line treatment option will provide not just physical benefits but crucial psychological support in knowing that further options exist.”

Nic Puntis, Chair of GIST Cancer, commented:

“People with GISTs experience significant challenges with existing treatments, including severe side-effects that often require dose reductions, treatment resistance that develops over time, and disease progression. Knowing there is now an approved fourth-line treatment available in England and Wales will help give people with a GIST diagnosis more hope than having to rely solely on the limited treatments that have existed until now.”

“This is fantastic news for the whole GIST Community, and I would like to express my gratitude to the patients and medical professionals who contributed to the NHS Appraisal, which finally approved Ripretinib. GIST Cancer UK supports and represents a community of patients, some of whom were relying on this positive outcome in order that they might enjoy a fulfilling life for longer.”

A Community Experience

The announcement also highlights the experience of Cathy Hampshire, Vice-Chair of GIST Cancer UK, who has been living with GIST for five years. She underwent major abdominal surgery in October 2021 to remove a large tumour. About two years later, the cancer returned and spread to her liver and other areas of her bowel.

Cathy has since tried all three currently available treatments. She reacted badly to imatinib, the standard first-line therapy, and experienced the same difficulties with sunitinib. She is now on regorafenib, the last treatment option currently available, which she has been taking for two-and-a-half years.

She described the uncertainty associated with this stage of treatment:

“Regorafenib has a limited window of effectiveness. At every scan and every consultation, I brace myself for the moment I’m told the drug has stopped working, that the tumours are growing again and that there is no further treatment options left.”

Following the NICE decision, she added:

“Today’s NICE decision changes that picture entirely. I was literally dancing around the room when I heard. When you’re on the third of the three available treatments and been told there are no more options, you feel like the system has given up on you. After a two-year battle, NICE has recognised the importance of making Ripretinib available. For people like me across England and Wales, this opens up a new treatment pathway and with it, renewed hope. I feel as though a huge weight has lifted from my shoulders. For the first time in a long while, there is light at the end of the tunnel.”

Expanding Access to Treatment

Ripretinib was previously approved for medical use in the United States and Australia in 2020, in the European Union in 2021, and by the Scottish Medicines Consortium in August 2025 for use within NHS Scotland.

With the NICE decision, patients in NHS England now gain access to this fourth-line treatment option.

The full article is available on Sarcoma UK.