Pharma Strategy Blog

Commentary on Pharma & Biotech Oncology / Hematology New Product Development

Pfizer’s Crizotinib (Xalkori) approved in ALK-positive lung cancer!

The FDA just announced that they have approved Pfizer’s crizotinib (Xalkori):

The U.S. Food and Drug Administration today approved Xalkori (crizotinib) to treat certain patients with late-stage (locally advanced or metastatic), non-small cell lung cancers (NSCLC) who express the abnormal anaplastic lymphoma kinase (ALK) gene.

Xalkori is being approved with a companion diagnostic test that will help determine if a patient has the abnormal ALK gene, a first-of-a-kind genetic test called the Vysis ALK Break Apart FISH Probe Kit. It is the second such targeted therapy approved by the FDA this year.

Source FDA

This is wonderful news for those unfortunately affected by this debilitating disease and those yet to be diagnosed with the aberration who will be able to be treated with a new highly specific and targeted drug.

ALK aberrations typically occur in the order of 4-7% of NSCLC patients, depending on sources. No doubt the companion FISH diagnostic test from Abbott will make it easier to screen and identity patients. In turn this will help determine which patients with lung cancer are eligible for treatment.

Pfizer began the rolling NDA submission in January and completed it in May, giving a PDUFA date around November 17th. This rapid approval in approx. three months continues the 2-3 month trend seen with cabazitaxel (Jevtana) and abiraterone (Zytiga) in castration-resistant prostate cancer (CRPC), and vemurafenib (Zelboraf) in metastatic melanoma.

The Xalkori story has been nothing short of amazing and represents another major advance for targeted therapy in a clearly identified subset of patients. There are several patient stories that I’ve come across on the internet, most are heart warming – take a look at this snippet I have curated from ‘feel good’ anecdotes from a caregiver this month alone:

View “Does crizotinib work in ALK+ lung cancer?” on Storify

It’s amazing to follow their story of courage and grace under pressure; it is also very hard to have a bad hair day when the very fragility of human life stares at you in the face. It could be any of us under 50, even non-smokers.

The response rates to crizotinib have been incredible, as witnessed by Dr Jack West’s story about one of his patients at Swedish:

{Update 1: Dr West tells me that the young gentleman he referred to in his TED story has now been on crizotinib 2+ years and is doing well enough to coach soccer!}

In the final PI, the overall response rates for Xalkori in two single arm studies (n=136 and 119) in patients who had mostly received prior systemic therapy. They differed in that:

“In Study A, ALK-positive NSCLC was identified using the Vysis ALK Break-Apart FISH Probe Kit. In Study B, ALK-positive NSCLC was identified using a number of local clinical trial assays.”

The ORR was 50% and 61% for each respectively. This is pretty impressive, in my opinion. According to the PI, the adverse event profile is quite tolerable:

“The most common adverse reactions (≥25%) across both studies were vision disorder, nausea, diarrhea, vomiting, edema, and constipation.
Grade 3-4 adverse reactions in at least 4% of patients in both studies included ALT increased and neutropenia.”

These are fairly normal and commonplace for cancer therapy, although there are potential vision disturbances that may need to be watched (from the PI):

“Vision disorders including visual impairment, photopsia, vision blurred, vitreous floaters, photophobia, and diplopia were reported in 159 (62%) patients in clinical trials.
These events generally started within two weeks of drug administration.
Ophthalmological evaluation should be considered, particularly if patients experience photopsia or experience new or increased vitreous floaters.
Severe or worsening vitreous floaters and/or photopsia could also be signs of a retinal hole or pending retinal detachment.”

The good news is that Xalkori is now available – according to the Pfizer press release:

XALKORI is available immediately through a number of specialty pharmacies.
Patients prescribed XALKORI can call 1-877-744-5675 for assistance accessing the medication.

For more information about the FDA-approved ALK test, call (855) TEST-ALK (837-8255).

The big question many will be asking, though, is what’s the price?

Answer: Price: $9,600/month, putting it in line with similar pricing to Roche’s Zelboraf in metastatic melanoma.

{Update 2: Abbott has also received approved for the ALK test although no information on the cost of the test was provided}.


13 Responses to “My take on the crizotinib Xalkori FDA approval”

  1. Richard Meyer

    Another cancer drug that costs over $100,000 and this is for an oral treatment.  If pharma companies continue to develop more expensive cancer drugs that just extend life without quality of life than we are surely headed for spiraling healthcare costs

    • maverickny

      Fair point, Rich, but by all accounts I’ve heard patients on this drug have been doing very well in the trials with fewer side effects than the chemo they had previously, so I think that would qualify as improved quality of life.  Several PIs I spoke to also noted that many of their patients are living over 2 years on crizotinib, unthinkable before that as the ALK aberration normally portends a poorer prognosis.

      I was disappointed with the price tag, don’t get me wrong but once the CMS approved Dendreon’s Provenge $93K cost for 4 months of extra survival, there was only one way prices were going.

    • Rick G

      I have been taking XALKORI for just 3 weeks so I cannot attest to its effectiveness, though my doctor is very confident it will do a great job.  The side effects are less severe, overall, than what I was experiencing with chemo, though it’s obvious to me that the human body’s digestive system was not designed to absorb these sorts of chemicals.

      As for cost, XALKORI is expensive, but a great bargain compared to chemo.  My previous chemo (Alimta + Avastin) was costing $27,000 every 3 weeks, whereas the XALKORI costs about $9,600 every month.  So, the XALKORI is about 1/4 the cost of chemo. 

      • maverickny

        Hi Rick,

        Very pleased indeed to hear that you are on Xalkori at a lower cost than you had to pay previously! Really do hope that it has a great response for you. Please do drop back as you feel able and let us know how you are getting on, we’d love to hear of your progress. I gather that there are other patients in your situation discussing Xalkori on the Cancer GRACE forum run by Dr Jack West in Seattle (

      • Violetta V

        Rick – the nausea side effect disappears after a while. Also, ask your doctor about taking it with a little bit of food. Also anti-nausea medications help. My mother was in clinical trial of this drug in Sloan-Kettering and this is what her doctor told her: they first said not to eat one-two hours before/after (I am not sure if it was just a research protocol or if they still recommend it), but they told to make this exception if the nausea doesn’t go away. In her case, it was only an issue for the first week or so. After that she had no side effects.

  2. Kathleen

    Well, quality of life is pretty subjective isn’t it?  But I agree about cost entirely. However, they’ll give the drug away in most cases. 

    • maverickny

      It will be interesting to see how patients who get slapped with a 20% co-pay feel, Kathleen – that’s nearly $2K/month for therapy.  No doubt there will be a patient assistance program for those who cannot afford it though.

  3. Susanlgamble

    I have been on Xalkori for just over 3 weeks now. I had been through 4 rounds of traditional chemo which only stopped cancer progression during treatment and about 30 days after. Now after 3 weeks of treatment I am driving again, have stopped daily vomiting, have energy, have regained my sense of taste and appetite, and have gone off daily narcotics for pain relief from spread of cancer to my bones. I have not had any scans yet, but I have to believe al this relief is from the Xalkori.

    • maverickny

      Susan, that’s really wonderful to hear, am delighted to learn that you are feeling better and seeing some functional improvement in your daily life.  This is what we in the industry all work towards – making a difference to the lives of patients like yourself.  Please do stop back and let us know of your progress in the future. 

  4. Xy2008080808


    I am from China, and here we don’t have this medicine.
    But one of my friends with lung cancer need this medicine, actually she is so young (40 years old).

    Could you tell me whether this medicine can prescirbed by the family doctor or not?
    If she can have the therapy by this medicine, she would like to go to USA for it.

    Thank you so much!


  5. cialis

    I’m rooting for you too! I’ll be thinking of you next Wednesday. I have good vibes about you finding your second miracle. Xalkori will be just chapter 1 in your book with lots more amazing stories to be told.

  6. Glc

    I’ve been taking Xalkori for about 2 months since being diagnosed with stage 4 lung cancer in February, no conventional chemo or radiation prior to starting Pfizer’s pill. Other than occasional diarhea, and a slightly diminshed energy level and I tire more easily, so I’m getting close to 9 or 10 hours of sleep a night. Other than this, if i didn’t know i had cancer, I wouldn’t know i had cancer.
    Saw an X-Ray after three weeks of treatment, and even in this crude resolution, diminished cancer was evident. Obviously evident, I’m pleased to say, although understand I’m a new cancer patient, untrained in analyzing x-rays. I can, however, tell black from white!
    Looking forward to seeing a CT scan result in June for verification and a more accurate assessment.
    Also saw on NBC news where Xaklori wiped out a child’s lymphoma in 28 days- results so amazing they made national TV news.
    God bless al the aner researchers, for this patient is getting 95% of previous quality of life, heightened since I’m OT spending it 60 hours a week at work since i woke up to what is important in my life- because i feel I’ve been granted a second one with this treatment.   

Comments are closed.

error: Content is protected !!