Today we completed the Phase 2 trial running at Nijmegen with the intermittent regime. The trail continues since the last participant was on boarded in Aug 2011. Doc tells that results should be published by late this year. No other information available except for the few readings we took along the year.

We lost quiet some mts in the 6MWT over the year. The way my kid has slowed down; I am pretty sure that we were on placebo. Over that there were nothing but a few instances of bacteria and WBC in the urine samples. 

However we start on the extension study in a month from now. The gap is needed to ensure that data for extension is isolated from the original study. A new baseline for the extension study, and we start with weekly shots. This time its for sure that we will gets the meds. NO MORE PLACEBO.

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So this drug was approved BEFORE extensions were completed. Very similar to us, they had a 48-week Phase 3 trial and now the 96-week extension is ongoing BUT the drug was approved. I think that this proves my previous point.

http://investors.vrtx.com/releasedetail.cfm?ReleaseID=644158

About KALYDECO

KALYDECO is the first treatment to target the underlying cause of CF. The Phase 3 studies evaluated KALYDECO in people with CF ages 6 and older who had at least one copy of the G551D mutation. PERSIST, a Phase 3, open-label, 96-week extension study, is underway to evaluate the long-term safety and durability of treatment with KALYDECO. This ongoing study enrolled people who completed 48 weeks of treatment in either Phase 3 study (placebo and KALYDECO treatment groups) and met other eligibility criteria. KALYDECO will be taken as one 150-mg tablet twice daily (every 12 hours).

Vertex retains worldwide rights to develop and commercialize KALYDECO. In October 2011, Vertex submitted a marketing authorization application to the European Medicines Agency (EMA) for KALYDECO and has received agreement from the EMA for accelerated assessment in Europe. The EMA regulatory review is ongoing.

 

Acting three months before its scheduled decision date, the Food and Drug Administration yesterday approved a drug developed by Vertex Pharmaceuticals Inc. to treat some patients suffering from cystic fibrosis, a rare and often fatal genetic disease.

Kalydeco, which initially had an FDA decision date of April 18, targets the cause of the disease in roughly 4 percent of the US patient population of 30,000. Those patients have a genetic mutation in a particular protein that sits at the surface of cells and regulates the flow of water and salt. The Vertex drug binds to the protein and works to restores its normal function, helping patients make mucus that lets them absorb and digest food and gain weight.

It's great to see any drug get approval so fast, especially for a rare condition;....why can't AVI and Prosensa follow the same path for DMD? I don't know?

 

I believe AVI's plans are to pursue that path if the phase 2 data is good. They will unblind in Q2 and just completed dosing this week (which is much faster than GSK's unblinding timeline).

I have no idea what GSK’s plans are, but they might do it. This CF drug was approved after Phase 3. They had 200+ patients in the 2 (2 age groups) phase 3 trials. So after phase 3, the patients from the trials are in 96-week the extension on the drug. They analyzed and submitted data for review to the FDA and it was approved. No one loses here, the patients in extension still get the drug for free for 96 weeks, the other patients DO NOT have to wait until extensions end, they pay the $294K to get the drug…

It is possible that GSK will do this. However, GSK is only in a 6-month phase 2 trial (testing 3 mg and 6 mg doses) in the US so that is a problem, and from what I heard it is not even clear that all the boys in this US trial will be included in any extension after the 6 months. But they can still fill for approval in EU, Canada and all other countries with phase 3 trials after phase 3 ends.

My previous point was that approval doesn’t need to wait for extension trials. If some parents are concerned about long term safety and efficacy they are welcome to wait until the extension trials end, before they put their kids on the treatment. I would take my chance and put my son on the drug w/o waiting 2 additional years, if proven safe and effective after the Phase 3 trial. This creates a good precedent I think and shows that FDA does approve drugs for orphan diseases in shorter time and without waiting additional years for extensions.

 

I would agree with you Ofelia......I would take the jump rather than wait another two years!!....Is there any way that charities such as PPMD and Action Duchenne etc can follow up on this?

 

I also would take the jump. There are plenty of us who would i think.

Yes PPMD, how can we push this cart to go faster?

A.



damien lynch said:

 

I would agree with you Ofelia......I would take the jump rather than wait another two years!!....Is there any way that charities such as PPMD and Action Duchenne etc can follow up on this?

 

I just want to thank every single mother, father, brother, sister, aunt, uncle, and especailly these BRAVE boys who are going through this obviously difficult process to save my son's life (or in the least help his QUALITY of life).  My Wyatt is just 3 weeks shy of 4 years old, he's too young to be apart of these trials, but again I have to say, I owe so much gratitude to these selfless children and their parents for what they are doing.  And yes of course we all want our children on the drug, but even these darling angels that are on the placebo are doing their part too. I just hope and pray that all of our sweet boys get this drug soon.  However, we have to start somewhere and I know that exon 51 is only a small part of our community, but once it gets tested, approved, etc, then the lives of countless families will change forever.  The day of diagnosis I never thought this day would come.  People told me to be positive and things were changing quickly, but until we are all giving our babies these drugs, quickly isn't quick enough.   Bless you trial families, I truly feel like you are angels on earth. 

So, we have been pushing for exactly this type of regulatory reform--a drug that meets safety standards in phase II should be allowed a "conditional approval" during which time it could be sold and the phase III study could take place. This allows wider access and it would allow the company to have some revenue during that period, although it's not clear that insurance companies would be willing to pay at this point.  Our Board has spent some time on this and has developed a regulatory policy that will form the basis of our advocacy agenda and will provide language for any legislative changes.  That policy will be disseminated widely very soon (including to industry and the FDA)--I'll come back to this discussion and post the link as soon as it is.

 

Anyone who cares about this issue and is able please consider joining our Advocacy Summit in Washington in two weeks--our platform is focused solidly on the FDA approval process. Even if you can't attend the meeting due to costs, work, etc., make sure that Ryan has you on the mailing list to receive advocacy updates. With the reauthorization of PDUFA next year there may be opportunities to influence you representatives on this issue via phone calls and email (for more on PDUFA and why this legislation is relevant to this discussion, see the transcript of the PPMD Webinar at http://www.youtube.com/watch?feature=player_embedded&v=SNcBR_h_kpk).

 

Sharon

 



damien lynch said:

 

I would agree with you Ofelia......I would take the jump rather than wait another two years!!....Is there any way that charities such as PPMD and Action Duchenne etc can follow up on this?

 

 This sounds very positive Sharon...thanks for the information..

 


I only hope that it will affect the approval Ataluren.


Sharon Hesterlee said:

So, we have been pushing for exactly this type of regulatory reform--a drug that meets safety standards in phase II should be allowed a "conditional approval" during which time it could be sold and the phase III study could take place. This allows wider access and it would allow the company to have some revenue during that period, although it's not clear that insurance companies would be willing to pay at this point.  Our Board has spent some time on this and has developed a regulatory policy that will form the basis of our advocacy agenda and will provide language for any legislative changes.  That policy will be disseminated widely very soon (including to industry and the FDA)--I'll come back to this discussion and post the link as soon as it is.

 

Anyone who cares about this issue and is able please consider joining our Advocacy Summit in Washington in two weeks--our platform is focused solidly on the FDA approval process. Even if you can't attend the meeting due to costs, work, etc., make sure that Ryan has you on the mailing list to receive advocacy updates. With the reauthorization of PDUFA next year there may be opportunities to influence you representatives on this issue via phone calls and email (for more on PDUFA and why this legislation is relevant to this discussion, see the transcript of the PPMD Webinar at http://www.youtube.com/watch?feature=player_embedded&v=SNcBR_h_kpk).

 

Sharon

 



damien lynch said:

 

I would agree with you Ofelia......I would take the jump rather than wait another two years!!....Is there any way that charities such as PPMD and Action Duchenne etc can follow up on this?

 

Thanks Sharon.

Do you know what I can do from Canada?

Andrea

Sharon Hesterlee said:

So, we have been pushing for exactly this type of regulatory reform--a drug that meets safety standards in phase II should be allowed a "conditional approval" during which time it could be sold and the phase III study could take place. This allows wider access and it would allow the company to have some revenue during that period, although it's not clear that insurance companies would be willing to pay at this point.  Our Board has spent some time on this and has developed a regulatory policy that will form the basis of our advocacy agenda and will provide language for any legislative changes.  That policy will be disseminated widely very soon (including to industry and the FDA)--I'll come back to this discussion and post the link as soon as it is.

 

Anyone who cares about this issue and is able please consider joining our Advocacy Summit in Washington in two weeks--our platform is focused solidly on the FDA approval process. Even if you can't attend the meeting due to costs, work, etc., make sure that Ryan has you on the mailing list to receive advocacy updates. With the reauthorization of PDUFA next year there may be opportunities to influence you representatives on this issue via phone calls and email (for more on PDUFA and why this legislation is relevant to this discussion, see the transcript of the PPMD Webinar at http://www.youtube.com/watch?feature=player_embedded&v=SNcBR_h_kpk).

 

Sharon

 



damien lynch said:

 

I would agree with you Ofelia......I would take the jump rather than wait another two years!!....Is there any way that charities such as PPMD and Action Duchenne etc can follow up on this?

 

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