Though it's not for DMD the key point to note is that approval is given for kids who are not responding to steroids. The company is obligated to continue clinical trials while those left with no option can get the stem cells even while trials are ongoing.

This is how terminal diseases should be dealt with. Not taking decades while generation of kids are fodder for the FDA process.


http://www.osiris.com/pdf/2012-05-17%20Health%20Canada%20Release.pdf

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Hi  Tulika,
Thanks for posting this.Hope, very soon you will post an article with tag like this:'stem cell therapy for DMD'. Interesting point is that, this drug is an intravenous formulation of mesenchymal stem cells (MSCs), which are derived from the bone marrow of healthy adult donors.what I remember in an earlier post you started a discussion with this MSCs for treatment of DMD.So, treatment is in our way, only I pray for SPEED !

The article is confusing.

 

It states "First approved treament" and yet it also states "Prochymal is currently available in several countries, including the United States, under an Expanded Access Program"

 

If it is already commercially available in US, why would Canada be "the first" ?

 

EAP is "investigational drug outside of a clinical trial by patients with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress"

This is not rights for commercial marketing.

Anyways the message is there is more than a linear Ph1 to Ph4 for getting the drug to the needy. Amazing that as a community we dont find a single drug to us on EAP.

Ah...ok I get it. Or more to the point, I have some weekend reading to learn what EAP is and how it works.

New Zealand also approves use of Prochymal, 2nd country to allow a stem cell treatment while clinical trials are ongoing.

Hoping for similar proactive decision making for Duchenne too.

http://finance.yahoo.com/news/zealand-approves-osiris-stem-cell-205...

COLUMBIA, Md. (AP) -- Osiris Therapeutics Inc. said Friday thatregulators in New Zealand approved its stem cell therapyProchymal.

Prochymal is a treatment for graft vs. host disease, a side effect of bone marrow transplants in which transplanted bone marrow cells attack the body of the patient. Prochymal is approved for use in children who are not being helped by steroid therapy.

Prochymal has now been approved in two countries. Canadian regulators awarded the product a preliminary approval on May 17, meaning Osiris can market the drug but has to run additional trials to gain full approval. The Food and Drug Administration has not approved Prochymal, but adult and child patients can get the treatment under certain conditions.

Shares of Osiris Therapeutics jumped $1.11, or 13.8 percent, to $9.18 on Friday. The stock is up 65 percent since May 18.

It sounds like Canada has granted their equivalent of subpart H to this drug. US patients are possibly getting compassionate use? I wonder if the "certain conditions" is the eminent death that they seem to want to push everyone to before getting access...

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