FDA Approves First Targeted Therapy for Metastatic Pancreatic Cancer
FDA Approves First in Class Targeted Therapy for Metastatic Pancreatic Cancer
The U.S. Food and Drug Administration has approved Rasonque (daraxonrasib), a first-in-class RAS inhibitor for metastatic pancreatic adenocarcinoma, the most common form of pancreatic cancer. The once-daily tablet targets multiple RAS protein forms, a key driver of tumor growth. In a trial of 500 adults, Rasonque improved median overall survival to 13.2 months versus 6.7 months for standard chemotherapy. Approved 6.5 months ahead of schedule, it offers a new option for patients who have received prior therapy or are not candidates for multiagent systemic therapy.
This drug showed unprecedented results in an area of high unmet need.
- epistasis
Metastatic pancreatic cancer is the first indication for this class of RAS-inhibitor, but it certainly won't be the last, and it seems certain that there will be many many more cancers where this drug gets approved. There are mutations to activate KRAS in a substantial fraction of cancers across many organs, and but it's been such a hard protein to target with a drug that even decades ago I was told it was "undruggable."
What's going on with this new drug is a somewhat novel mechanism: instead of gumming up the enzymatic action of the protein, it acts as a glue between KRAS and a common "helper" protein, that then mucks up the RAS signalling. This sort of molecular glue mechanism has had a few examples built up over the past decade, but this is a really knock-it-out-of-the-park drug. Really amazing, and because of it lots of people are focusing lots of effort on how to discover more molecular glues.
- jmcharnes
My sister was diagnosed with pancreatic cancer in October (48 years old) and died in June from it.
I'm glad to see a drug like this developed for people suffering from pancreatic cancer. I don't wish what my sister went through on anyone.
Damn if I don't wish it had come a little sooner.
- dmschulman
My dad was diagnosed with pancreatic cancer in December 2019 and thankfully caught wind of it in its early stages due to an eagle-eyed nurse going over some routine blood work and noting his liver numbers were off, he should talk to the doctor.
You can imagine how hard it was to be sick in the early days of COVID, much less have to go to the hospital on a regular basis to receive chemotherapy for hours on end. Our family was lucky that he responded well to treatment at least and we had a few extra years with him before he passed away in late 2023.
Cancer is a terrible thing but pancreatic cancer is especially horrible due to the difficulty in detection when you're in otherwise good health. The fact that intense medical research into retrovirals has yielded something so significant as the RAS-inhibitor is nothing short of miraculous.
- hentrep
Beyond the data, this approval is notable for the speed from FDA acceptance of their new drug application (NDA) to FDA approval - just over a month. Compare this to the typical 8-12 month timelines we've historically seen for priority and standard reviews, respectively. This was enabled by the FDA's CNPV Pilot Program [0]
Previous discussion following the data presentation at ASCO: https://news.ycombinator.com/item?id=48517199
[0] https://www.fda.gov/industry/commissioners-national-priority...
- mstank
My mom was diagnosed with borderline stage-4 pancreatic cancer. Fortunately she had an aggressive surgeon willing to do a Whipple procedure, and survived almost 4 years after a 4-6 month prognosis.
We were incredibly lucky to even have that time with her. Pancreatic is one of the worst cancers someone can get — both for survival and quality of life.
I’m very happy to see that we’re finally making meaningful progress on treatment.