Horribly worded headline, but great news...

 

http://online.wsj.com/article/BT-CO-20110119-706326.html

Glaxo, Prosensa Starts Phase III Of Duchenne Medication Dystrophy

LONDON (Dow Jones)--GlaxoSmithKline PLC (GSK.LN) and Prosensa said Wednesday that the first patient has commenced treatment in the Phase III clinical study investigating GSK2402968 (968), in ambulant boys with Duchenne Muscular Dystrophy or DMD.

MAIN FACTS:

-Commencement of this study confirms previously announced plans to progress this asset into Phase III.

-This randomized, placebo controlled study will enroll 180 patients, from up to 18 countries, and is currently the most advanced ongoing study for this rare, severely debilitating, neuromuscular disease.

-The study is designed to assess the efficacy and safety of GSK968 6 mg/kg, once weekly, compared to placebo, for 48 weeks in ambulant boys over 5 years of age with DMD The primary efficacy endpoint is a measure of muscle function using the six minute walking distance test.

-Prosensa is a privately held biopharmaceutical company.

-GlaxoSmithKline shares at 1315 GMT up 8 pence, or 0.68%, at GBP1189.50 pence, valuing the company at GBP61.81 billion.

-By Razak Musah Baba, Dow Jones Newswires; 44-20-7842-9275; razak.baba@dowjones.com

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Does anyone know if the FDA finally approved testing here in the U.S.?????  The article didn't mention where phase III started, but I'm guessing it's in Europe....In Dec. I emailed Paula at Cinci and she replied they were still waiting FDA authorization..any updates appreciated!!!!!!!!!!!!!!!!! Karen
No, there has been no approval for US trials.

Karen flor said:
Does anyone know if the FDA finally approved testing here in the U.S.?????  The article didn't mention where phase III started, but I'm guessing it's in Europe....In Dec. I emailed Paula at Cinci and she replied they were still waiting FDA authorization..any updates appreciated!!!!!!!!!!!!!!!!! Karen
Unfortunately, FDA did not approve phase 3 start in the US.
Ofelia are you saying you heard they are "NOT" approving for US or they just have not YET?

Ofelia Marin said:
Unfortunately, FDA did not approve phase 3 start in the US.
Just haven't obtained approval yet for USA. 

JoAnne Hathcoat said:
Ofelia are you saying you heard they are "NOT" approving for US or they just have not YET?

Ofelia Marin said:
Unfortunately, FDA did not approve phase 3 start in the US.

I am not able to understand the reasons for not getting FDA approval.

 

Non ambulatory trials have been going on in Columbus, Ohio, for some time. Data from that trial must be there for FDA to review. Theoretically this data should be sufficient for approving phase II.

 

What I am missing is a FDA ombudsman. Almost every discussion on research come to a halt when it reaches FDA. Does anyone know about a FDA ombudsman for DMD. Probably Sharon may pick this up.

The non-ambulatory trial was only ONE injection.

Phase 3 is a long term trial (48 weekly injections). FDA requested additional data in animals in order to approve a LONG term trial. That data was collected and sent to the FDA a while ago. After review, FDA still did not approve the start oif the trials...I am assuming the toxicity level in that data was not convincing enough to make FDA allow the start of the trials (?).

 

I do not know if FDA requested additional data and/or if GSK has any intention to provide that anytime soon.



Tulika said:

I am not able to understand the reasons for not getting FDA approval.

 

Non ambulatory trials have been going on in Columbus, Ohio, for some time. Data from that trial must be there for FDA to review. Theoretically this data should be sufficient for approving phase II.

 

What I am missing is a FDA ombudsman. Almost every discussion on research come to a halt when it reaches FDA. Does anyone know about a FDA ombudsman for DMD. Probably Sharon may pick this up.

Does anyone know if Americans will be allow to participate in the phase III trials that just started???? I would be willing to relocate for a year, if my son could be a part of this study. Karen
You can email the sites and ask that question. Each site might follow different enrollment rules in terms of accepting US citizens. GSK will update/expand the list of sites soon.

Karen flor said:
Does anyone know if Americans will be allow to participate in the phase III trials that just started???? I would be willing to relocate for a year, if my son could be a part of this study. Karen
Back to the question from Cheryl.

Ofelia is it anywhere confirmed that FDA has rejected the application, or is it like that FDA has yet not responded?

There are 18 countries where the trial is supposed to happen and clinicaltrials.gov lists only a few. I wish that its a case of FDA taking more time as compared to rejection







Ofelia Marin said:

The non-ambulatory trial was only ONE injection.

Phase 3 is a long term trial (48 weekly injections). FDA requested additional data in animals in order to approve a LONG term trial. That data was collected and sent to the FDA a while ago. After review, FDA still did not approve the start oif the trials...I am assuming the toxicity level in that data was not convincing enough to make FDA allow the start of the trials (?).

 

I do not know if FDA requested additional data and/or if GSK has any intention to provide that anytime soon.



Tulika said:

I am not able to understand the reasons for not getting FDA approval.

 

Non ambulatory trials have been going on in Columbus, Ohio, for some time. Data from that trial must be there for FDA to review. Theoretically this data should be sufficient for approving phase II.

 

What I am missing is a FDA ombudsman. Almost every discussion on research come to a halt when it reaches FDA. Does anyone know about a FDA ombudsman for DMD. Probably Sharon may pick this up.

The data was submitted in November as announced by the GSK guy during ActionDuchenne's conference in London. FDA has to answer in 30 days after data submission to either request additional data/info or the IND is open and the trial is allowed to start. The 30 days have already passed and the long term trial is not allowed to start in the US. Other than that I do not have any info besides the following private email: 

"

There is currently one clinical study of GSK’968 ongoing in the US – a Phase I study in non-ambulant boys (Study DMD114118: http://clinicaltrials.gov/ct2/show/NCT01128855?term=GSK2402968&...)   

 

The FDA recently allowed the initiation of this early phase study that investigates the pharmacokinetics (PK), safety and tolerability of GSK’968, after a single subcutaneous administration at different dose levels.  In the US, long term studies (including the 48 week Phase III study, Study DMD114044) cannot progress.   GSK will continue to work closely with FDA to address their data needs and requirements. Once FDA’s requirements are met and FDA allows longer term studies to be conducted in the US, it is currently envisaged that US patients may  potentially be recruited into a separate study. 


 

In the meantime, I am unable to specifically address your question “Although my son is a US citizen, is there any way to get him on this trial?”  Responsibility for recruiting patients lies with the investigators undertaking the clinical study. Any questions regarding participating in the study should be addressed to the appropriate investigators once the study sites are posted on http://clinicaltrials.gov/ct2/show/NCT01254019?term=GSK2402968&... .  We anticipate a second wave of investigator sites will be posted shortly.  You can also find investigator site information on this link - http://www.gsk-clinicalstudyregister.com/protocol_comp_list.jsp?com...     

 

Results from each of the ongoing clinical studies will provide information regarding the efficacy and safety of GSK’968 and will form part of the data package we file with individual regulatory agencies (including FDA) when we apply for a license.

 

I wish to assure you that we are doing everything possible to develop GSK’968 as safely and as rapidly as we can, as a potential treatment for appropriate patients with DMD.

 

Best regards,

 

John

 

John E. Kraus, MD, PhD

Director, Medical Sciences

Neurosciences MDC

GlaxoSmithKline R&D"




Tulika said:

Back to the question from Cheryl.

Ofelia is it anywhere confirmed that FDA has rejected the application, or is it like that FDA has yet not responded?

There are 18 countries where the trial is supposed to happen and clinicaltrials.gov lists only a few. I wish that its a case of FDA taking more time as compared to rejection







Ofelia Marin said:

The non-ambulatory trial was only ONE injection.

Phase 3 is a long term trial (48 weekly injections). FDA requested additional data in animals in order to approve a LONG term trial. That data was collected and sent to the FDA a while ago. After review, FDA still did not approve the start oif the trials...I am assuming the toxicity level in that data was not convincing enough to make FDA allow the start of the trials (?).

 

I do not know if FDA requested additional data and/or if GSK has any intention to provide that anytime soon.



Tulika said:

I am not able to understand the reasons for not getting FDA approval.

 

Non ambulatory trials have been going on in Columbus, Ohio, for some time. Data from that trial must be there for FDA to review. Theoretically this data should be sufficient for approving phase II.

 

What I am missing is a FDA ombudsman. Almost every discussion on research come to a halt when it reaches FDA. Does anyone know about a FDA ombudsman for DMD. Probably Sharon may pick this up.

Does anyone happen to know what the toxicity concern is with this drug? I've heard bits and pieces but am unclear whether the toxicity they refer to has to do with the actual mechanics of exon skipping (the fact this would be toxic in a healthy body) or some side effect they are seeing with the drug? Wasn't it shown in the phase 2 study that the drug was well tolerated, or was some toxicity demonstrated in higher dosages in a separate test?


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