US regulators have approved a once-daily pill shown to double survival time for people with advanced pancreatic cancer, intensifying calls for the promising treatment to be made available in Britain as soon as possible.
Daraxonrasib is being described as one of the biggest steps forward in the fight against the devastating disease for decades.
The US Food and Drug Administration (FDA) cleared the first-of-its-kind therapy yesterday after a large trial found patients given the drug lived for an average of 13.2 months, compared with 6.7 months among those receiving standard chemotherapy.
The medicine, which will be marketed in the US under the brand name Rasonque, is intended for patients with metastatic pancreatic cancer — where the cancer has spread to other parts of the body — who have already been treated with chemotherapy.
It has not yet been approved for use in Britain.
Pancreatic Cancer UK last night urged UK regulators to move swiftly so NHS patients can access the treatment.
Diana Jupp, the charity’s chief executive, said: ‘This exciting new drug is one of the most significant breakthroughs for pancreatic cancer in decades.
‘Its approval for patients in America is a source of real hope for people with pancreatic cancer and their loved ones worldwide.
A daily pill that doubles survival time for patients with advanced pancreatic cancer has been given the green light by US regulators
‘This new treatment has been proven to double survival for advanced pancreatic cancer, and its assessment was fast-tracked to reflect the vital need for new life-extending treatments.’
Around 11,500 people are diagnosed with pancreatic cancer in the UK each year and approximately 10,200 die from the disease.
It is Britain’s fifth biggest cause of cancer death, despite accounting for only around three per cent of new cancer cases.
Fewer than six in 100 patients are still alive five years after diagnosis, making it one of the deadliest common cancers.
Ms Jupp said: ‘Pancreatic cancer is uniquely devastating, half of people with the disease die within just three months of diagnosis. That is simply not long enough to make precious memories.’
Pancreatic cancer is particularly difficult to treat because it frequently causes few obvious symptoms until it has already spread.
Early warning signs can include pain in the upper abdomen or back, unexplained weight loss, indigestion, changes to bowel habits and jaundice – yellowing of the skin or whites of the eyes.
By the time the disease is advanced, surgery – currently the only treatment with the potential to cure pancreatic cancer – is usually no longer possible.
Daraxonrasib offers a new way of attacking the disease.
Around 90 per cent of pancreatic cancers are driven by mutations in a gene called KRAS.
The gene normally helps control how cells grow and divide. But when it mutates, it can become permanently switched on, sending signals that cause cancer cells to multiply uncontrollably.
For decades, scientists struggled to develop medicines capable of blocking KRAS, leading it to be described as ‘undruggable’.
Daraxonrasib works by blocking the abnormal signals produced by KRAS and related proteins, slowing the growth and spread of cancer cells.
The FDA’s decision followed a trial involving 500 patients, with an average age of 66, from North America, Europe and Asia who had metastatic pancreatic cancer and had previously received treatment.
Just under half received daraxonrasib while the remaining patients were given standard chemotherapy.
As well as living longer, patients taking daraxonrasib also went for longer before their cancer worsened – a median of 7.2 months compared with 3.6 months among those receiving chemotherapy.
Tumours shrank in 30 per cent of patients taking the drug, compared with 11 per cent of those given chemotherapy.
Daraxonrasib also caused fewer serious side effects than chemotherapy, with patients mainly reporting rash, diarrhoea, fatigue and nausea.
The drug is not a cure and patients in the trial eventually saw their cancer progress.
But experts say the results are remarkable for a disease where patients with advanced cancer have historically had few effective treatment options.
Clinical trials of daraxonrasib and other drugs targeting RAS are already under way in the UK.
Pancreatic Cancer UK is now calling on the Medicines and Healthcare products Regulatory Agency (MHRA), NICE and other UK assessment bodies to accelerate their assessment of the treatment.
Ms Jupp said: ‘It is imperative that regulators here in the UK now respond with the same urgency.
‘We must see the MHRA, NICE and assessment bodies across the UK enact fast-track approval processes for daraxonrasib.
‘People with pancreatic cancer have been overlooked and left behind for too long. We must do everything possible to ensure the most promising new treatments are available on the NHS as soon as possible.’
