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Genentech Granted FDA Breakthrough Therapy Designation for Actemra® for Systemic Sclerosis.

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Genentech Granted FDA Breakthrough Therapy Designation for Actemra® for Systemic Sclerosis.

 

On June 10, 2015, Genentech announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation status to Actemra® (tocilizumab) for the treatment of systemic sclerosis (also known as scleroderma), a rare and potentially life-threatening disease with no FDA-approved therapy. ISN News Release, 06/10/2015. (Also see: Scleroderma Treatments and Clinical Trials: Actemra)

 

This item was posted in the ISN Newsroom. Please check the newsroom daily for updates on scleroderma and other related articles.


Jo Frowde

ISN Assistant Webmaster

SD World Webmaster

ISN Sclero Forums Manager

ISN News Manager

ISN Hotline Support Specialist

International Scleroderma Network (ISN)

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This is huge news.  Does anyone know if this means Actemra is approved for Scleroderma or if it is just going to be tested more?  Is there anyone here that is already taking it? 

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Hi Grey,

 

Please see FDA Breakthrough Therapy Designation.

 

I'm not an expert in this, and I may be wrong (I often am!) so we seeking more clarification.  As I understand it, the FDA designation will help expedite the review process a bit.

 

In the U.S., Actemra is already approved for moderate to severe Rheumatoid Arthritis, in people who have had an inadequate response to a DMARD. So it would appear to be available, but only as an off-label use for scleroderma, which means that it most likely would not be covered by most insurance plans because it would be experimental use until it has full FDA approval. Therefore, at the moment, it would be primarily available for scleroderma through clinical trials. (See Scleroderma Treatments and Clinical Trials: Actemra)

 

It's my understanding that it costs roughly $1,600 per vial for tocilizumab in the U.S. It is generally prescribed for once a month self-injection for RA, and patient assistance and co-pay programs are available for RA patients. I am *assuming* that the discounts would probably not be available for scleroderma until (or if) it eventually clears the final hurdles of FDA approval for scleroderma.  See Actemra.com and contact them for further information.

 

Actemra is also known as RoActemra in Europe, where it is approved for even early-stage rheumatoid arthritis.


Warm Hugs,

 

Shelley Ensz

Founder and President

International Scleroderma Network (ISN)

Hotline and Donations: 1-800-564-7099

 

The most important thing in the world to know about scleroderma is sclero.org.

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Thanks Shelley.  I know a few members on another site are currently on Actemra for lupus, which has not been approved and have had insurance cover it, so I'm remaining hopeful.  I was a little upset to see that the clinical trial won't be done until 2018, which can be an eternity for those in need now.  

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