I've been following Beacon postings closely since my nephew was diagnosed with multiple myeloma in April. Thanks so much to the Beacon Staff and to those who take time to share their experiences! Very helpful for all of us.
My nephew is now preparing for stem cell transplant in a few weeks. I am curious to know if anyone was prescribed Granix (tbo-filgrastim) instead of Neupogen (filgrastim) injections to stimulate the release of stem cells into the blood. I have read that Granix is not FDA approved for stem cell mobilization in the U.S. but the transplant center wants him to use that – not Neupogen. Is that acceptable?
Since Granix is being provided through the transplant center, it is not clear that insurance approved this non-FDA approved (cheaper) alternative or whether it is a substitute of what insurance approved.
Also, how much of this medicine is required for stem cell mobilization? My nephew is being prescribed almost 20 mcg/kg instead of the recommended 10 mcg/kg for Neupogen. He will give himself 3 injections (!) at the same time each day for 4 days before first day of harvest. He will also be injecting Mozobil before the first harvest attempt. There is no indication his stem cells will be difficult to mobilize so this appears to be preemptive. While he took Revlimid, it was for 4 cycles and ended 3 weeks before the scheduled harvest. I understand the center probably wants to minimize the chance of a failed mobilization, but there is no clear failure risk.
I feel helpless since these decisions appear to be institution-determined and difficult to change. Perhaps they are to allow the center to process large numbers of patients quickly through the stem cell harvest process. This just adds to the strain on our already stressed out family as we prepare for transplant. It's difficult to question these decisions for fear that it might affect the quality of needed care. The oncologist himself is very good - an excellent myeloma specialist! Although this stage of treatment is dominated by the transplant center.
Any thoughts and guidance would be very much appreciated. Is this nothing to worry about? Just go along?
Forums
Re: Neupogen or Granix for stem cell mobilization?
Hello Faith:
I am not familiar with Granix. I did quickly look it up, however, and it looks like it's under clinical trial right now. I did not do a deep dive into the biochemistry of it, but basically it sounds like a close derivative of filgrastim (Neupogen). If I am correct that this is a clinical trial setting, then your nephew should have a full disclosure, and his participation should be completely voluntary. This is somewhat different than the way you characterized it. I am guessing that there might be a communications gap somewhere, but if it's a clinical trial situation, then your nephew should understand (if he does not already) that his participation would be completely voluntary.
Stem cell mobilization and collection has become a standard part of the ASCT process, that is typically executed as a relatively minor part of the overall process (compared to the later phases of the ASCT). Still, it's critically important, the filgrastim does potentially cause bone pain, and the process could take several days. If it's made more effective, that would be good (faster with lower side effects). I have also read about new mobilization drugs under development that actually have a measurable anti-myeloma effect, which would be very good, and could potentially contribute to better responses. I recall that Neupogen does not have any anti-myeloma effect in and of itself.
Generally, if you find a clinical trial that your are comfortable with, then participation does contribute to the improvement of the state of the art. However, no one should ever be pressured or pushed into a clinical trial that they are not comfortable with. I hope this helps in your understanding of the situation.
I am not familiar with Granix. I did quickly look it up, however, and it looks like it's under clinical trial right now. I did not do a deep dive into the biochemistry of it, but basically it sounds like a close derivative of filgrastim (Neupogen). If I am correct that this is a clinical trial setting, then your nephew should have a full disclosure, and his participation should be completely voluntary. This is somewhat different than the way you characterized it. I am guessing that there might be a communications gap somewhere, but if it's a clinical trial situation, then your nephew should understand (if he does not already) that his participation would be completely voluntary.
Stem cell mobilization and collection has become a standard part of the ASCT process, that is typically executed as a relatively minor part of the overall process (compared to the later phases of the ASCT). Still, it's critically important, the filgrastim does potentially cause bone pain, and the process could take several days. If it's made more effective, that would be good (faster with lower side effects). I have also read about new mobilization drugs under development that actually have a measurable anti-myeloma effect, which would be very good, and could potentially contribute to better responses. I recall that Neupogen does not have any anti-myeloma effect in and of itself.
Generally, if you find a clinical trial that your are comfortable with, then participation does contribute to the improvement of the state of the art. However, no one should ever be pressured or pushed into a clinical trial that they are not comfortable with. I hope this helps in your understanding of the situation.
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JPC - Name: JPC
Re: Neupogen or Granix for stem cell mobilization?
My mom just used Granix last week for her stem cell harvest at Northwestern Memorial Hospital in Chicago in preparation for her ASCT next week. She was able to harvest 25 million stem cells in less than 3 full days without Mozibil and with very minimal side effects in terms of bone pain. Granix was described to us as a new-generation Neupogen by our transplant team.
Re: Neupogen or Granix for stem cell mobilization?
JPC and Cubs Fan – Thank you!
Cubs Fan - Good your mom just had a successful harvest. Did she have chemo mobilization or just Granix? I believe Northwestern often does chemo mobilization. Also, do you know how much Granix she took per kg? Or how many injections per day?
JPC - I know my nephew is not in a clinical trial. Since NIH is only now doing a comparison study of Granix and Neupogen combined with Mozobil, I worry my nephew is being given experimental treatment outside a clinical trial. It may work, but is it worth the chance? Maybe the doubling of the dose is less significant than using Granix before its FDA approval for that purpose?
Will let you know how this goes. Stem cell harvest is a week away. Thanks.
Cubs Fan - Good your mom just had a successful harvest. Did she have chemo mobilization or just Granix? I believe Northwestern often does chemo mobilization. Also, do you know how much Granix she took per kg? Or how many injections per day?
JPC - I know my nephew is not in a clinical trial. Since NIH is only now doing a comparison study of Granix and Neupogen combined with Mozobil, I worry my nephew is being given experimental treatment outside a clinical trial. It may work, but is it worth the chance? Maybe the doubling of the dose is less significant than using Granix before its FDA approval for that purpose?
Will let you know how this goes. Stem cell harvest is a week away. Thanks.
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Faith
Re: Neupogen or Granix for stem cell mobilization?
Hi Faith: Thank you for the response. I am just curious as to how a drug not yet approved by the FDA can be given in the US off of clinical trial. I will have to look into that. From the report of Cub's fan, it sounds good so far. Good luck to your nephew. JPC
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JPC - Name: JPC
Re: Neupogen or Granix for stem cell mobilization?
Granix was FDA approved back in 2012.
http://www.drugs.com/history/granix.html
Once a drug is FDA approved, doctors in the USA generally have the flexibility to use it as they see fit.
http://www.drugs.com/history/granix.html
Once a drug is FDA approved, doctors in the USA generally have the flexibility to use it as they see fit.
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Multibilly - Name: Multibilly
- Who do you know with myeloma?: Me
- When were you/they diagnosed?: Smoldering, Nov, 2012
Re: Neupogen or Granix for stem cell mobilization?
Hello Multibilly,
Thank you. I so much appreciate your immense knowledge on these issues, which you have freely shared.
It appears Granix was approved for use to support those doing chemo to address neutropenia, but specifically not approved for other purposes. The prescribing information does not include use for stem cell mobilization. Here is the link to the ongoing inferiority study.
https://clinicaltrials.gov/ct2/show/NCT02098109
It appears that Granix is biosimilar but not identical to Neupogen. It will probably get full FDA approval but it seems not to have it yet for stem cell mobilization. All a bit confusing.
Thank you. I so much appreciate your immense knowledge on these issues, which you have freely shared.
It appears Granix was approved for use to support those doing chemo to address neutropenia, but specifically not approved for other purposes. The prescribing information does not include use for stem cell mobilization. Here is the link to the ongoing inferiority study.
https://clinicaltrials.gov/ct2/show/NCT02098109
It appears that Granix is biosimilar but not identical to Neupogen. It will probably get full FDA approval but it seems not to have it yet for stem cell mobilization. All a bit confusing.
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Faith
Re: Neupogen or Granix for stem cell mobilization?
As others have just mentioned, Granix IS approved by the FDA. More importantly, Granix is essentially a generic equivalent of Neupogen, or what is known as a "biosimilar".
In most countries where Granix is sold, it is explicitly described as a biosimilar. That is not the case in the U.S. because there was no regulatory framework for biosimilar approvals when Granix was submitted for FDA review. So Teva, the company that sells Granix, had to get it approved the traditional way, which means it had to be approved for a single initial indication, which happens to be something other than stem cell mobilisation.
Had Granix been approved in the U.S. as a biosimilar, as in other countries, the U.S. prescribing information for Granix would be able to list exactly the same approved uses that Neupogen has.
The European prescribing information for Granix, where the drug is marketed under the Tevagrastim brand name, recommends a dosing of 10 mcg/kg/day when the drug is used for stem cell mobilisation. That is exactly the same dosing recommended for Neupogen in its U.S. prescribing information. Here is the European prescribing information ("Summary of Product Characteristics") for Granix / Tevagrastim, It lists stem cell mobilisation for transplantation as one of the indications, and also gives the dosing as 10 mcg/kg/day.
I suspect the higher dosing of Granix being recommended by Faith's nephew's doctors is due to their conservatism – as evidenced by their use of Mozobil as well – rather than anything to do with Granix and its efficacy.
In most countries where Granix is sold, it is explicitly described as a biosimilar. That is not the case in the U.S. because there was no regulatory framework for biosimilar approvals when Granix was submitted for FDA review. So Teva, the company that sells Granix, had to get it approved the traditional way, which means it had to be approved for a single initial indication, which happens to be something other than stem cell mobilisation.
Had Granix been approved in the U.S. as a biosimilar, as in other countries, the U.S. prescribing information for Granix would be able to list exactly the same approved uses that Neupogen has.
The European prescribing information for Granix, where the drug is marketed under the Tevagrastim brand name, recommends a dosing of 10 mcg/kg/day when the drug is used for stem cell mobilisation. That is exactly the same dosing recommended for Neupogen in its U.S. prescribing information. Here is the European prescribing information ("Summary of Product Characteristics") for Granix / Tevagrastim, It lists stem cell mobilisation for transplantation as one of the indications, and also gives the dosing as 10 mcg/kg/day.
I suspect the higher dosing of Granix being recommended by Faith's nephew's doctors is due to their conservatism – as evidenced by their use of Mozobil as well – rather than anything to do with Granix and its efficacy.
Re: Neupogen or Granix for stem cell mobilization?
As usual, Ian makes some great points and I tend to agree with his assumptions.
Faith: Putting aside the exact question of the use of Granix in this situation, doctors in the USA can prescribe a drug that has been FDA approved for one use for use in other "non-approved" applications. This is called "off-label" prescribing.
I for one am very grateful for living in a country where this is possible (it's not generally allowed in many countries, including Europe). A now-classic example of the off-label prescribing in multiple myeloma cases is with the use of Revlimid. While Revlimid was only just FDA-approved this year for newly diagnosed multiple myeloma patients, it had been routinely prescribed for newly diagnosed multiple myeloma patients by many US doctors for quite a number of years...and many patients are the better for it.
Detractors of this practice would argue that drugs should not generally be prescribed off-label and should instead only be used in a clinical trial setting so that the drug's impact can be better characterized for the benefit of the entire medical arena. But, when push comes to shove, I would gladly welcome the opportunity for a knowledgeable multiple myeloma specialist to be able to save my butt by being able to prescribe a drug off-label and outside the very restrictive environment of a clinical trial. But that's just my personal two cents.
Faith: Putting aside the exact question of the use of Granix in this situation, doctors in the USA can prescribe a drug that has been FDA approved for one use for use in other "non-approved" applications. This is called "off-label" prescribing.
I for one am very grateful for living in a country where this is possible (it's not generally allowed in many countries, including Europe). A now-classic example of the off-label prescribing in multiple myeloma cases is with the use of Revlimid. While Revlimid was only just FDA-approved this year for newly diagnosed multiple myeloma patients, it had been routinely prescribed for newly diagnosed multiple myeloma patients by many US doctors for quite a number of years...and many patients are the better for it.
Detractors of this practice would argue that drugs should not generally be prescribed off-label and should instead only be used in a clinical trial setting so that the drug's impact can be better characterized for the benefit of the entire medical arena. But, when push comes to shove, I would gladly welcome the opportunity for a knowledgeable multiple myeloma specialist to be able to save my butt by being able to prescribe a drug off-label and outside the very restrictive environment of a clinical trial. But that's just my personal two cents.
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Multibilly - Name: Multibilly
- Who do you know with myeloma?: Me
- When were you/they diagnosed?: Smoldering, Nov, 2012
Re: Neupogen or Granix for stem cell mobilization?
If Granix is a 'biosimilar', it has probably been suggested because it would be less costly than Neupogen. I don't know the actual costs of the two drugs though, so that is a guess on my part. 'Biosimilar' drugs are interesting. A drug that has 'biological' activity cannot be synthesized directly the same as is a drug that is just a chemical. Those can become 'generic' drugs. Usually the generic drugs are produced to be marketed once a patent runs out on the original drug, usually in about 20 years.
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Nancy Shamanna - Name: Nancy Shamanna
- Who do you know with myeloma?: Self and others too
- When were you/they diagnosed?: July 2009
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