Pancreatic cancer’s once daily pill gets US FDA nod: What it means for therapy and India?

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For people with advanced pancreatic cancer, a new treatment could mean more time to live, as it represents a breakthrough in an area scientists have struggled with for decades. The US Food and Drug Administration approved daraxonrasib (brand name Rasonque), an oral pill taken once a day, for certain patients whose cancer has spread and stopped responding to earlier treatment.It is the first approved pancreatic cancer treatment aimed at suppressing the RAS protein, one of the key drivers of the disease. In a major trial, patients taking the drug lived a median of 13.2 months, compared to 6.7 months with chemotherapy. The hazard ratio for death was about 0.40, indicating a substantial reduction in the risk of death during the study period.But there is an important distinction between doubling median survival and doubling an individual’s life expectancy. The trial result is a population median: some patients will live considerably longer, while others will not respond or may have to stop treatment because of toxicity.Why the drug approval matters so muchPancreatic cancer is one of the most challenging types of cancer to treat. A large proportion of patients are diagnosed only after the cancer has spread, limiting the possibility of surgery. The tumour itself is also biologically difficult to treat, with a dense surrounding microenvironment — the stroma — that has been resistant to many treatment strategies.As Professor Anant Ramaswamy, specialist in gastrointestinal cancers and Geriatric Oncology at Tata Memorial Centre, Mumbai, noted in our earlier explainer, advanced pancreatic cancer has historically offered only incremental improvements despite decades of research. Against that background, a treatment that produced a median survival of 13.2 months after earlier therapy — compared with 6.7 months for chemotherapy — represents a meaningful change in the treatment landscape.Also Read | A new pill can double survival time for deadliest cancer: An expert explains why this is a gamechangerWhat does the pill actually do?The drug targets RAS, a family of proteins that act like a signalling switch inside cells. As cells mutate in cancers, they impact RAS, leaving this signalling system stuck in an abnormal growth-promoting state. KRAS, one member of the RAS family, fuels pancreatic cancer.Daraxonrasib belongs to a new generation of medicines designed to switch off RAS proteins. The FDA describes it as targeting multiple forms of RAS, a key driver of tumour growth. For decades, RAS was regarded as one of cancer biology’s most difficult targets. Researchers could identify the problem but struggled to block the protein effectively without risking toxicity. Daraxonrasib shows that the target can, in fact, be attacked successfully in patients.Story continues below this adThe trial met its primary and key secondary endpoints, including overall survival and progression-free survival. Patient-reported quality-of-life measures also favoured daraxonrasib.Which patient should get it?Daraxonrasib is not currently approved as an across-the-board first treatment for pancreatic cancer. The approved population is adults with metastatic pancreatic adenocarcinoma who have received at least one previous systemic treatment, or are not candidates for multiagent systemic therapy.So, a newly diagnosed patient with potentially operable pancreatic cancer does not simply replace surgery or chemotherapy with this pill. The old therapies — surgery, chemotherapy and radiotherapy where appropriate — remain important. Targeted therapy is being added to the arsenal.Trials are continuing to examine whether daraxonrasib can move into earlier lines of treatment and potentially into combination approaches. Revolution Medicines, the manufacturer, is also developing other RAS-targeting drugs and studying the class in cancers beyond pancreatic cancer.Story continues below this adThe pill is not all mildThe convenience of a tablet should not be confused with the absence of side effects. The FDA lists rash, diarrhoea, inflammation/ulceration of the mouth (stomatitis), nausea, fatigue, vomiting, abdominal pain, swelling (edema), decreased appetite and haemorrhage among the common adverse effects.Earlier clinical data also showed that dose reductions were sometimes necessary. Doctors will have to assess whether, for an individual patient, the balance between tumour control, survival, side effects and quality of life is better with daraxonrasib than with available alternatives.What about the price?The reported US wholesale acquisition cost is $39,800 for a 30-day supply at the recommended daily dose (roughly ₹38 lakh at current exchange rates). That translates to roughly $477,600 a year (roughly ₹4.5 crore a year) before insurance discounts, rebates or other adjustments. But the list price is not the same thing as what an insured patient necessarily pays. Actual out-of-pocket costs depend on insurance coverage, deductibles, co-pay or co-insurance arrangements and assistance programmes.For India, there is an additional issue: US FDA approval does not automatically make it an approved or routinely available medicine in India. Regulatory approval, importation, local marketing arrangements and reimbursement would all affect eventual Indian access. That makes accessibility one of the next big questions for Indian oncologists and patients.Story continues below this adWhat does this then mean for India?For Indian patients, the FDA decision is best viewed as a major scientific development rather than an immediate guarantee of access.There is, however, a larger reason for optimism. RAS mutations are found across several major cancers. Daraxonrasib is being investigated not only in pancreatic cancer but also in other RAS-driven malignancies, including lung and colorectal cancers. The pancreatic-cancer approval therefore validates an entire therapeutic strategy rather than a single drug. The drug is “one more option” rather than a cure for a stubborn kind of cancer.