Revolutionary Drug that Targets ‘Untreatable’ Cancers Gets FDA Approval

The FDA has approved a new drug that health experts say could be critical in giving people with drug-resistant cancers a chance to prolong their lives.

The treatment, known as larotrectinib or Vitrakvi, works a little bit differently from standard cancer treatments that are already on the market.

Normally, treatments target cancer cells and inhibit tumors by, essentially, attacking them. They are often tied to the region a cancer might be in, so for example the lung or thyroid. That works really well for a lot of different cancers, but for somewhat rarer tumors, not so much.

Among that class is a group of tumors known as TRK fusion cancer, which have particular genetic mutations driving them and can occur across many different cancers. They don’t respond well to conventional treatments and, whether occurring in adults or children, this kind of cancer is often classed as untreatable. These kinds of cancers can include thyroid and lung cancers, as well as infantile fibrosarcoma and more.

However with this new treatment, drugs giant Bayer and developer Loxo Oncology are hoping to change that. Larotrectinib works differently from other treatments, because it doesn’t matter where the tumor is in the body, it can target those tumors by targeting the genetic biomarkers that underpin the cancer cell growth. This new approach, part of an emerging class of treatments known as TRK inhibitor therapy, is the next step in moving away from chemotherapy treatments and into immunotherapy.

The FDA has now fast tracked  approval for this therapy for adults and children who have what’s known as an NTRK gene fusion without resistance, where surgery isn’t an option and where other treatment options are no longer possible.

“This new site-agnostic oncology therapy isn’t specific to a cancer arising in a particular body organ, such as breast or colon cancer,” FDA Commissioner Scott Gottlieb, M.D. said in a press release, “Its approval reflects advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine. We now have the ability to make sure that the right patients get the right treatment at the right time.”

Gottlieb also characterized this fast-track approval as a necessary one that, while under heavy restrictions, is particularly important for treating children who may be facing TRK fusion cancers, saying: “using our breakthrough therapy designation and accelerated approval processes, we support innovation in precision oncology drug development and the evolution of more targeted and effective treatments for cancer patients. This is especially true when it comes to pediatric cancers.”

FDA grants fast track approval if a drug has a demonstrated good safety record and if it is able to serve an as yet unfulfilled but pressing health need, which larotrectinib does.

The FDA has based its approval on a number of key trials involving 55 pediatric and adult patients who fit the treatment profile. Overall, patients treated with larotrectinib demonstrated a 75 percent response rate across several different types of tumors, meaning that the treatment halted the progression of their cancers.

Just as important is that the vast majority of that group saw their cancers halted for six months, while 39 percent saw progression halted for a year or more. Keeping in mind that without this intervention patients would likely have run out of treatment options, and the worth of larotrectinib is immediately apparent.

As with any treatment, we have to think about cost, but on this topic the supplier, Bayer, is one step ahead: it has pledged to try to make the drug as affordable as possible.

Keeping Costs Low for Patients

NTRK gene fusion cancers are incredibly costly for patients, and not just in terms of their overall health. Getting a diagnosis hinges on having a TRK fusion cancer test, which in itself can cost thousands of dollars. As with all cancer treatments, the price quickly adds up.

Bayer, however, has reportedly designed a number of strategies to ensure that out-of-pocket expenses for patients receiving the treatment remain low, so for insured patients the expense could be as little as $20 or less.

If there are complications with insurance, Bayer has pledged to look at ways to get the treatment to the patient regardless while the issue is worked out or connect patients with Bayer-approved charities who may take over the expense of copays. Bayer is also offering a money-back guarantee for patients who show no effects from the treatment.

Critics point out that the burden of the high price tag Bayer is putting on the treatment, even if not directly felt by patients, may still make the larotrectinib treatment cost-prohibitive. They also note that the exact details for the relief programs are yet to emerge. However, others have praised Bayer for taking proactive steps to ensure that people who need this treatment can get it, regardless of their insurance.

All eyes will be on how larotrectinib is priced and how easy it is for patients to access. In the meantime, the approval is no doubt a ray of hope for people living with cancers who, until now, may have had very few to no treatment options left.

Take Action

Ultimately, we shouldn’t have to rely on drug companies to self-police when it comes to fair drug prices. Join over 37,000 Care2 members, and sign this petition telling Congress that we want more transparency from drug companies and insurance companies when it comes to drug prices.

If you want to make a difference on an issue you find deeply troubling, you too can create a Care2 petition, and use this handy guide to get started. You’ll find Care2’s vibrant community of activists ready to step up and help you.


Related at Care2

Photo credit: Getty Images.


Joanna M
Joanna M1 months ago

If you are an adult in the US who needs help with their cancer-related bills, check out 1UpOnCancer - they are a great grassroots nonprofit! No one should have to worry about money while fighting cancer.

heather g
heather g2 months ago

immunotherapy seems a better option than chemotherapy treatments.

Julia R
Julia R2 months ago

I hope that many patients who had untreatable cancers now can be treated successfully with this new treatment and hopefully be cured! I hope that Bayer follows through with its promises to keep this treatment very affordable to many patients who need it!

Susanne R
Susanne R2 months ago

If it's true that Bayer is "taking proactive steps to ensure that people who need this treatment can get it, regardless of their insurance," I would think very highly of them. Unfortunately, the greed and indifference of the pharmaceutical industry has made me skeptical of any of their claims. I hope Bayer is very successful in further refining this drug and that they stay true to their word and make sure it's made available to all patients --not just the wealthiest among us.

Christine S
Christine Stewart2 months ago


Kathy K
Kathy K2 months ago


Shae Lee
Shae Lee2 months ago

Thanks for sharing.

Ruth S
Ruth S2 months ago


Ruth S
Ruth S2 months ago


Debbi W
Debbi W2 months ago

Immunotherapy is or can be wonderful, at least my experience with it is, and less expensive than chemo or radiation. It will be wonderful when there is an immunotherapy treatment for every cancer known.