Home Forums Melanoma Diagnosis: Stage IV Merck Announces Breakthrough Therapy Designation for Lambrol

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    Interesting news this morning around Merck’s pd1 (ps – why do they always name drugs such odd things… lambrolizumab? )

    Catherine – this sounds like good news but what exactly does it mean to be given breakthrough therapy designation?





    Thanks for posting this; that’s really interesting! I’m no expert, but here’s the FDA’s FAQ about Breakthrough Therapy. It’s a new designation just created last year and looks to me to be the fastest track possible for approval. My one (selfish) concern is that they aren’t planning to cut short the Phase I trial that I am hoping my dad will qualify for in June.


    From a different article


    What sets the breakthrough designation apart from other expedited drug development mechanisms-all of which have been in place at the FDA for at least 20 years-is the requirement of early clinical data demonstrating an unprecedented effect (see ‘Drug development in the fast lane’). Fast-track designation, for example, can be granted off the back of promising preclinical data; accelerated approval status has more to do with surrogate trial endpoints. And although companies with fast-tracked drugs will receive earlier and more frequent communication from the FDA, they won’t get the ‘all hands on deck’ approach that is promised by the breakthrough designation. For breakthrough therapies, senior FDA managers and reviewers are expected to work closely with drug sponsors to design collaborative, multidisciplinary development plans that hasten timelines to approval and minimize the number of patients exposed to less efficacious treatments or placebos.

    “This is really meant to signal from the agency that if you have a drug that shows a really unprecedented activity early on, they want to work with you to find the best course forward, rather than have you go it alone,” says Jeff Allen, executive director of Friends of Cancer Research, a think tank and advocacy organization based in Washington, DC, that has actively championed the new breakthrough pathway.

    Ultimately, Ivy expects patients to benefit as much as, if not more than, drug sponsors. Currently, individuals who are desperately seeking unapproved treatments but cannot enroll in a clinical trial must petition the FDA for a ‘special exception use’, yet drug companies are not always so keen to provide investigational medicines outside the confines of a controlled study. The breakthrough designation should increase drug access earlier on. “In the end,” Ivy says, “I think the driver, goal and motivation for breakthrough therapies is to make treatments more widely accessible to patients who don’t have other options.”

    Catherine Poole

    I’ve written to my contacts to find out as well. And we do have a patient advocate at the FDA from MIF and I will ask him as well. Looks like fast track to me and that’s a good thing!


    Yes thanks guys!

    Sounds like fast track to me also which is great news! Especially since lots of folks were saying it would take a long time to get this approved. I mean, I’m lucky enough that I’m already in a trial for it… but this will help so many more!

    Thanks for the info!



    This is a very interesting development, and I’ll do what I can to find out more about it.

    As mentioned by Tamils, the “Breakthrough Therapy” designation is very new at the FDA, less than a year, so I’d imagine it’s not completely clear what the practical ramifications are (i.e., for us stage 4 patients). In particular, will this mean easier access to the drug promptly (via expanded access or compassionate use applications – I haven’t seen those 2 mentioned anywhere yet), as well as truly faster approval overall? 2 months ago, my oncologist said he thought anti-PD1 was still at least 2 years away from approval (the BMS version phase III trial is due to be completed November 2014).

    I am an FDA patient representative (but that hardly means I’m especially up on this), so I will do some calling etc tomorrow and post anything useful I learn. Just so you know, I’ve been involved in a lot of letter-writing to drug officials in the past couple of years trying to get anti-PD1 pushed along towards approval, but have ended up feeling as if my efforts (in concert with a number of others) has had little demonstrable effect (maybe I made some people uncomfortable – possibly a good thing). Also, as a caution, just be aware that other established FDA programs, such as “accelerated” development, are now routinely given to almost anything that looks likely to succeed, so that’s an example of what “watering down” does. My guess is that, if they’ll be forthcoming, Merck officials are more likely to know what the future holds in the development of this very promising drug than folks at the FDA.


    Catherine Poole

    Merck has been very aggressive in this market and has so many trials going in just about every conceivable location. My contact could tell me nothing more than they hope for accelerated approval asap. For right now the trials are being amended to not require chemo as one arm of the trial, fairly soon and in some locations. They are the only trial that has cross over too if you do get stuck on the chemo arm. So fingers crossed this makes it to the prescription label soon.


    This sounds like it could be great news. So much uncertainty…as we know, GSK’s BRAF/MEK drugs, which should be approved beginning of June (fingers crossed!) were submitted as monotherapies, so I’m very curious as to how insurance companies will treat combotherapy prescriptions.

    I’ve been trying to get into a PD1 trial…for 2 years…first there were exclusions for prior therapies (radiation, Yervoy, Remicade, etc.) then when I once again had the measurable disease necessary, I got the BRAF+ diagnosis, so I’ve been on Zel since.

    I’m anxiously awaiting word of approval timeframes in light of today’s news, and I hope these drugs’ approvals mean lots of NED reports for all of us!

    Best to all,



    Does this leapfrog the Merk version ahead of BMS’s Nivolumab in terms of approval? Does it say anything about the effectiveness of one versus the other?


    Catherine – very curious about the trial being amended to eliminate the chemo arm since I am in the process of trying to get into this now. My understanding was first scans in 12 wks and then you could crossover if you have progression, but what happens if the trial is amended while you’re on it? Any idea? The thought of chemo doesn’t thrill me, but I’m willing to risk it.

    Also, why just some locations?

    Thank you!

    Catherine Poole

    To answer some questions to the best of my knowledge, yes, this may push Merck ahead of BMS for approval of their PD1. They’ve been very aggressive in accrual and trials. As for the amendment, I do not know where that will take place, but it won’t happen once you are enrolled in a trial. The odds are very good of getting the PD1, 3-1 over chemo. In the Merck trial you can cross over if there is progression. I will let you know more as soon as I get more information. In the meantime, I would talk directly to the study centers to find out more as well.


    Thanks Catherine!


    This is really, really exciting.




    Just a follow-up – the FDA folks I contacted really have nothing to add to the materials on “Breakthrough Therapy” already posted on the link from Tamils. It’s really in the hands of Merck what their next step is in terms of development and application. From what I can see, their application for this status has already allowed closer developmental contact with the FDA (and may indeed have allowed them to leap-frog BMS’ version). My guess is that this means very little immediately in terms of expanded use or compassionate use applications. It may well mean that Merck’s drug gets approval faster down the line…If I find out anything more, I’ll certainly pass it on. And the numbers presented thusfar certainly suggest a better response rate than the BMS anti-PD1 (but they’re small). So the excitement with yesterday’s releases are probably mostly for the business prospects for Merck. As one contact said (Celeste), what’s presented in June at ASCO is probably going to be a lot more informative.

    Also in case you missed it, there’s a front page article in today’s NY Times about the horrendous costs ($100,000 per year) for new cancer drugs and the protests that some oncologists, particularly at Memorial Sloan Kettering, have initiated. I think they can only be applauded – it’s outrageous and more to the point, unsustainable, and disqualifies most people from treatment (I’ve had Ipi treatment, and that cost somebody $125K). In the article, the oncologists were quoted as saying these prices are unethical. I would also add that I’ve heard oncologists say it was unethical to withhold drugs from expanded use when it’s clear at early stages of development that they’re clearly superior to current “standard therapy.” Who of us would not do whatever we could to get anti-PD1 treatments now, rather than waiting 2 years for it to clear a phase III trial???? How many of us will be here then???


    Catherine while you are finding things out could you specifically ask about making access to anti PD1 for Ocular melanoma.

    Certainly in Europe we are excluded from all antin PD1 trials. There is no logic in this as Weber has found that in OM as opposed to skin melanoma imflammatory infiltrate of metastases is a bad sign He has found that the T cells in that imflammatory infiltrate are exhausted(resting or something not sure of exact term) and he states on one of your webinairs that giving anti PD1 to OM pateints before tumour harvesting and culturing the T cells in a medium containing anti PD1 helps reverse the process and makes cloning more sucessful.

    I would love to do anti PD 1 . My onocologist would love to do a trial but the drug companys are not forth coming. Anyone know of a trial ( I have had ipilimumab twice) that I could travel to?


    My sentiments exactly, Jonathan!!! It’s very frustrating to hear of success such as anti PD-1 and know you would do anything to take part, but exclusions don’t allow you to do so. Those that are running out of options fighting this deadly disease and are willing to do whatever it takes, will more than likely not be around in two years.

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