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FDA approves Ninlaro (ixazomib)

by Beacon Staff on Fri Nov 20, 2015 12:52 pm

Breaking news - The FDA today approved ixazomib as a new treatment for multiple myeloma. The drug, which will be marketed under the brand name "Ninlaro," has been approved for use in combination with Revlimid and dexamethasone in multiple myeloma patients who have had one prior therapy.

For the Beacon news article about the approval, see

"Ninlaro (Ixazomib) Approved By FDA For Multiple Myeloma", The Myeloma Beacon, Nov 22, 2015

(Yes, the link to the Beacon news article was added to this posting once the article was published. We thought it might be useful, however, to have it in this thread.)

This FDA press release also has basic information about the approval:

"FDA Approves Ninlaro, New Oral Medication To Treat Multiple Myeloma" Nov 20, 2015.

Some key excerpts from the press release:

Today the U.S. Food and Drug Administration granted approval for Ninlaro (ixazomib) in combination with two other therapies to treat people with multiple myeloma who have received at least one prior therapy  ...

Ninlaro is a type of cancer drug called a proteasome inhibitor and works by blocking enzymes from multiple myeloma cells, hindering their ability to grow and survive. Ninlaro is the first oral proteasome inhibitor and is approved in combination with another FDA-approved treatment for multiple myeloma called Revlimid (lena­lido­mide) and dexamethasone (a type of corticosteroid).

The safety and efficacy of Ninlaro were demonstrated in an international, ran­dom­ized, double-blind clinical trial of 722 patients whose multiple myeloma came back after, or did not respond to, previous treatment. Study participants received either Ninlaro in combination with lenalidomide and dexamethasone or placebo plus lena­lido­mide and dexamethasone. Those taking Ninlaro lived longer without their disease worsening (average 20.6 months) compared to participants taking the other regimen (14.7 months).

The most common side effects of Ninlaro are diarrhea, constipation, low blood platelet count (thrombocytopenia), peripheral neuropathy (numbness and pain from nerve damage, usually in the hands and feet), nausea, peripheral edema (fluid under the skin causing swelling), vomiting and back pain.

Beacon Staff

Re: FDA approves Ninlaro (ixazomib)

by NStewart on Fri Nov 20, 2015 1:05 pm

Wow, 2 new myeloma drugs approved in one week! Yes, it is a proteasome inhibitor, as are Velcade and Kyprolis, but it is oral. I hope that people who may want to use it have really good prescription coverage, or are millionaires, since Revlimid is also oral and costs about $10,000 a month. I doubt that Ninlaro will be much less expensive or even more expensive.

But, the news is good. More options for all of us.

Nancy in Phila

NStewart
Name: Nancy Stewart
Who do you know with myeloma?: self
When were you/they diagnosed?: 3/08
Age at diagnosis: 60

Re: FDA approves Ninlaro (ixazomib)

by Beacon Staff on Fri Nov 20, 2015 1:25 pm

Thanks for your comments, Nancy.

Here is a separate press release from the FDA with more basic information about the Ninlaro approval; it seems to be aimed mainly at healthcare professionals.

Also, here is the FDA approved prescribing information for the drug:

NinlaroPrescribingInformation.pdf
(240.57 KiB) Downloaded 244 times


Let us know if there if you find anything noteworthy in the prescribing information.

Beacon Staff

Re: FDA approves Ninlaro (ixazomib)

by BeatMyeloma on Fri Nov 20, 2015 1:43 pm

Fantastic news! Wow, two in one week! Maybe they'll approve elotuzumab tomorrow and we can get three in. :)

BeatMyeloma
Name: BeatMyeloma
Who do you know with myeloma?: My mother
When were you/they diagnosed?: Jan. 2008
Age at diagnosis: 54

Re: FDA approves Ninlaro (ixazomib)

by Multibilly on Fri Nov 20, 2015 2:32 pm

This is amazing news. It's also good news for me since I have no desire to go regularly into a clinic for treatment. I hope it will be extended to newly diagnosed myeloma patients in the near future.

The peripheral neuropathy (PN) figures in the prescribing information seem to bear out the earlier indications that PN is reduced with ixazomib with respect to subq Velcade.

I also think it will be interesting to see how this will change the myeloma maintenance landscape in the coming years.

Multibilly
Name: Multibilly
Who do you know with myeloma?: Me
When were you/they diagnosed?: Smoldering, Nov, 2012

Re: FDA approves Ninlaro (ixazomib)

by Mike F on Fri Nov 20, 2015 3:40 pm

What a week! This is just freakin' awesome!

We're still not looking at the cure, but every new option is a step forward.

Mike F
Name: Mike F
Who do you know with myeloma?: Me
When were you/they diagnosed?: May 18, 2012
Age at diagnosis: 53

Re: FDA approves Ninlaro (ixazomib)

by Ellen Harris on Fri Nov 20, 2015 4:25 pm

This is, indeed, a banner week! I am currently on Velcade bi-weekly for maintenance. I wonder if it can be prescribed for maintenance. I will ask my oncologist next time I see him.

Let's hope this saves some lives!!!

Ellen Harris

Re: FDA approves Ninlaro (ixazomib)

by computerteacher on Fri Nov 20, 2015 4:37 pm

Hi Ellen!

I am on Velcade (bi-weekly) for maintenance also! I will be seeing my doctor next week, and will ask him if it could be a maintenance drug, and if he would recommend it for me. Velcade has been fine for me, so I don't want to rock the boat, but, it would be nice to know it this would be a good alternative.

I will make a toast with a turkey leg this Thanksgiving, in gratitude for the approvals.

computerteacher
Name: Computer Teacher
Who do you know with myeloma?: self
When were you/they diagnosed?: November/December 2014
Age at diagnosis: 58

Re: FDA approves Ninlaro (ixazomib)

by Beacon Staff on Fri Nov 20, 2015 7:22 pm

Takeda, the company that developed and will market Ninlaro (ixazomib), has issued its press release regarding the FDA approval of the drug. You can read it here:

"U.S. FDA Approves Takeda’s NINLARO® (Ixazomib), The First And Only Oral Proteasome Inhibitor To Treat Multiple Myeloma" Takeda Oncology press release, Nov 20, 2015

In the press release is a statement from Dr. Paul Richardson from Dana-Farber about the approval:
“With the approval of NINLARO, we can now offer patients a once-weekly oral proteasome inhibitor as part of a highly active triplet therapy,” said Paul Richardson, M.D., Clinical Program Leader and Director of Clinical Research, Jerome Lipper Multiple Myeloma Center Institute Physician at Dana-Farber Cancer Institute, and investigator for TOURMALINE-MM1, the pivotal Phase 3 trial on which today’s approval is based. “We, as investigators of the TOURMALINE-MM1 trial, felt it was vital to conduct a comprehensive ‘real world’ eval­u­a­tion of this combination that included some of the most common patient types in the relapsed/refractory multiple myeloma setting, such as older patients, patients with moderate renal impairment, light chain disease, and high risk cytogenetics. Further, we treated patients until disease progression to determine the sustainability of NINLARO in treating their relapsed/refractory disease. The TOURMALINE-MM1 data demonstrate convincingly that oral NINLARO-based triplet treatment is effective at extending progression-free survival, over and above the clinical benefit seen with lena­lido­mide and dexa­metha­sone, with a tolerable safety profile.”

The press release also has this information about the TOURMALINE-MM1 clinical trial that was the basis for the Ninlaro approval:
TOURMALINE-MM1 is an international, randomized, double-blind, placebo-controlled clinical trial of 722 patients, designed to evaluate NINLARO plus lena­lido­mide and dexa­metha­sone compared to placebo plus lena­lido­mide and dexa­metha­sone in adult patients with relapsed and/or refractory multiple myeloma. Results showed NINLARO is effective in extending Progression Free Survival (PFS) and has a manageable safety profile. The trial achieved its primary endpoint and demonstrated a clinically meaningful and statistically significant prolongation in PFS at this analysis, which showed that patients treated in the NINLARO arm lived without their disease worsening for a significantly longer time compared to patients in the control arm. Patients continue to be treated to progression in this trial and will be evaluated for long term outcomes.

In the TOURMALINE-MM1 trial, the most common adverse reactions (≥20%) in patients receiving NINLARO included diarrhea, constipation, thrombocytopenia, peripheral neuropathy, nausea, peripheral edema, vomiting and back pain. Serious adverse reactions reported in ≥2% patients included thrombocytopenia (2%) and diarrhea (2%).

Efficacy and safety data were reviewed by an Independent Data Monitoring Committee (IDMC), who recommended the study be continued in blinded fashion to allow further maturation of long term outcomes, including overall survival (OS) and long-term safety.

Beacon Staff

Re: FDA approves Ninlaro (ixazomib)

by goldmine848 on Fri Nov 20, 2015 8:33 pm

I saw a statement that mentioned ongoing clinical trials of ixazomib, including one utilizing it in the post-transplant maintenance setting.

goldmine848
Name: Andrew
When were you/they diagnosed?: June 2013
Age at diagnosis: 60

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