I'm so excited about all the news about Exon 51.  It got me thinking that, oh yeah...  we're still in a trial too.  Ataluren.  What's up with that?  Is it time to get off the ataluren?  Do we just keep plugging along?  Would love to hear thoughts of others, families and providers.

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There's excellent news from PTC Therapeutics. They received the acceptance for review of their application for conditional approval in the European Union.

This is the e-mail they are sending out right now: 

------------------------------------------

Dear Friends,

 

As announced this morning, PTC is very pleased to have received the acceptance for review of our application for conditional approval in the EU. This is a historic moment for the Duchenne muscular dystrophy community and for PTC, as this is the first filing of a marketing authorization application for a new chemical entity for DMD. 

 

We would like to thank all the patients and families who have participated in our trials and have help guide our efforts over the last decade. The results of these clinical studies not only served as the foundation of the MAA, but provided PTC and the DMD community with valuable insights into the natural history of Duchenne muscular dystrophy and the potential impact that ataluren may have on disease progression.

 

In terms of our corporate strategy and next steps, PTC is moving forward not only with the EU MAA filing acceptance, but also with increased U.S. regulatory clarity. Based on our conversations with the FDA and EMA, we plan to enroll the first patients in our Phase 3 confirmatory trial as early as the first quarter of 2013. We have developed the design of this study in collaboration with our DMD investigators and patient organizations and with input from EMA and FDA – for which we are extremely grateful. The results of this confirmatory Phase 3 study will hopefully serve as the basis for full approval in the US and EU.

 

We expect to have additional information for patients and families on our website, www.ptcbio.com and www.clinicaltrials.gov in January. Parent Project Muscular Dystrophy is planning a webinar at 12 noon EST on January 17 so that we can provide additional information on the trial. PPMD will have details about the webinar as well as how to submit questions in advance on its website, www.parentprojectmd.org, within the week.

 

As you know, the path to approval for a treatment in a disorder that has never had a drug approval is not easy or straight-forward, but our corporate vision and sense of urgency have never wavered – even when faced with challenges. Instead, our energy and enthusiasm to advance ataluren as rapidly as possible continues to grow, and is reinforced by our robust foundation of preclinical and clinical data. We are supported by our collaborators and investors and are inspired by the courage of these amazing boys and young men, the dedication of their families and the commitment of clinicians, researchers, physical therapists, site coordinators, patient advocates and all members of DMD community.

 

I want to take a moment to thank the team at PTC, which has been relentless in advancing ataluren from the initial idea through all the multiple steps leading to this milestone. Each team member has worked tirelessly and shares a deep commitment to our goal of transforming the lives of patients.

 

Again, we thank you for all of your efforts and support! 

 

Warmest regards,

 

Stu

 

Yes, I also just got it. So excited and grateful.

Trinh
 
Bernardo A. Iriberri said:

There's excellent news from PTC Therapeutics. They received the acceptance for review of their application for conditional approval in the European Union.

This is the e-mail they are sending out right now: 

------------------------------------------

Dear Friends,

 

As announced this morning, PTC is very pleased to have received the acceptance for review of our application for conditional approval in the EU. This is a historic moment for the Duchenne muscular dystrophy community and for PTC, as this is the first filing of a marketing authorization application for a new chemical entity for DMD. 

 

We would like to thank all the patients and families who have participated in our trials and have help guide our efforts over the last decade. The results of these clinical studies not only served as the foundation of the MAA, but provided PTC and the DMD community with valuable insights into the natural history of Duchenne muscular dystrophy and the potential impact that ataluren may have on disease progression.

 

In terms of our corporate strategy and next steps, PTC is moving forward not only with the EU MAA filing acceptance, but also with increased U.S. regulatory clarity. Based on our conversations with the FDA and EMA, we plan to enroll the first patients in our Phase 3 confirmatory trial as early as the first quarter of 2013. We have developed the design of this study in collaboration with our DMD investigators and patient organizations and with input from EMA and FDA – for which we are extremely grateful. The results of this confirmatory Phase 3 study will hopefully serve as the basis for full approval in the US and EU.

 

We expect to have additional information for patients and families on our website, www.ptcbio.com and www.clinicaltrials.gov in January. Parent Project Muscular Dystrophy is planning a webinar at 12 noon EST on January 17 so that we can provide additional information on the trial. PPMD will have details about the webinar as well as how to submit questions in advance on its website, www.parentprojectmd.org, within the week.

 

As you know, the path to approval for a treatment in a disorder that has never had a drug approval is not easy or straight-forward, but our corporate vision and sense of urgency have never wavered – even when faced with challenges. Instead, our energy and enthusiasm to advance ataluren as rapidly as possible continues to grow, and is reinforced by our robust foundation of preclinical and clinical data. We are supported by our collaborators and investors and are inspired by the courage of these amazing boys and young men, the dedication of their families and the commitment of clinicians, researchers, physical therapists, site coordinators, patient advocates and all members of DMD community.

 

I want to take a moment to thank the team at PTC, which has been relentless in advancing ataluren from the initial idea through all the multiple steps leading to this milestone. Each team member has worked tirelessly and shares a deep commitment to our goal of transforming the lives of patients.

 

Again, we thank you for all of your efforts and support! 

 

Warmest regards,

 

Stu

 

Does it mean that Ataluren will be available for children suffering from DMD with nonsense mutations?

Dear Peter,

As I understand it is just accepted to review in EU. It takes 13 ~ 15 months for the review ; then if it is approved, it will be available.

Trinh

Has anyone on ataluren experienced elevated triglycerides and cholesterol? 

My boy just entered screentest for Ataluren trial, phase III

Hi Trinh.  My son was one of original boys, and is in the continuation study in the states.  No placebo.  It's been a long road for us.   Good Luck!

Hi all,  follow up here.  Kyle just had his semi annual check up at the University of Iowa Children's Hospital.  His neuromuscular pediatrician specializes in DMD.  Her word of the day after evaluating Kyle was FANTASTIC!!!  He is doing EXTREMELY well.  The tricky question is why???  He has been on deflazacort (steriod) since diagnosis (5 + years ago) and has been in the Ataluren study since 6 months after going on steriods. His exon problem is at exon 58 (which by the way I have yet to find anyone with the same).  The doctor does not know if it's because of the steriods, the ataluren, because perhaps there "could be" some read through at this exon, or if he is just plain doing well.  He is VERY short (about 10% under the growth chart) and we are contemplating going to a weekend only steriod dosage (yet to be 100% decided, but his doctor is in favor).

Hi Donna, that's great.  Adam use to impress his docs too.  He walked until he was 15 years old.  Then he broke his femur, after falling out of his chair (wasn't wearing his seatbelt).  He takes deflazacort, only 15 mg/day, eats well, and has been on ataluren.  He stopped taking it, after spinal fusion surgery 2 months ago, and we are debating whether to go back on.   Adam wasn't happy with his shorter stature either, and so we opted for growth hormone therapy.  He gained some with that, and is more average height now.  After sx, he's even taller in his chair.  Adam's stop is at Exon 26.  We had wondered too, if this was just his natural history.....

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