Santhera Presents Clinical Data of Catena® in Duchenne Muscular Dystrophy at the 2011 Congress of the World Muscle Society

 

Liestal,  Switzerland, October 19,  2011 - Santhera  Pharmaceuticals (SIX: SANN)
announced  today  that  it  will  present  data from the 2-year open-label study
(DELPHI-E  study)  evaluating  Catena®  for  the  treatment of Duchenne Muscular
Dystrophy  [1],  one  of  the  most  common  and  devastating  types of muscular
dystrophy.  The findings indicate that Catena® slowed the decline in respiratory
function  compared to  the expected  natural history  in patients  with Duchenne
Muscular  Dystrophy. Data from this study are in agreement with and confirm data
from a randomized controlled study (DELPHI study) of 12 months duration [2]. The
poster  presentation will  be given  at the  16th International Congress  of the
World Muscle Society in Almancil, Portugal on October 21, 2011.

 

http://www.cisionwire.com/santhera-pharmaceuticals-holding-ag-ext/r...

 

 Sharon is attending WMS 2011. PRAYING TO GOD to hear many good news on Duchenne from WMS 2011.

 

Raktim

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and yet we need to wait YEARS until the trials are completed, then more YEARS until approval. ridiculous!

Although the FDA isn't fast enough we are able to acquire medications our children need.  Deflazacort is the best example and I imagine idebenone might just work a similar way.   The FDA can't stop us from providing for our sons.  I don't have the impression that stopping us is their goal anyway. 

 

With regard to, and especially with Duchenne, coloring outside the lines is called for.   

Santhera's is approved in Canada for FA but heard that it is very expensive, 20k or so/year????

cheryl cliff said:

Although the FDA isn't fast enough we are able to acquire medications our children need.  Deflazacort is the best example and I imagine idebenone might just work a similar way.   The FDA can't stop us from providing for our sons.  I don't have the impression that stopping us is their goal anyway. 

 

With regard to, and especially with Duchenne, coloring outside the lines is called for.   

Catena is simply idebenone by another name. No need to wait on trials or regulators on this one. Docs are already more willing to recommend up to 900mg/day based largely on these results.

when and where will be this data presented on 21st Oct be available? thanks.

Hi Amrit, 

Sharon asked me to pass along the abstract she found for the poster presentation.. Hope this helps!

 

Results from a two-year open label intervention study with idebenone (Catena®) in the Duchenne muscular dystrophy

G.M Buyse, D. Thijs, N. Goemans, M. van den Hauwe, H. Wei, C. Rummey, T Mier, and L Mertens

 

A 12-month double-blind, randomized, placebo-controlled phase II study in 21 patients with DMD (DELPHI Study) has indicated that idebenone (Catena®) may improve cardiac and respiratory function in 8-16 year old patients with DMD. Patients completing the DELPHI study were eligible to enroll into the open label extension study (DELPHI-e) of 24 months duration to further investigate the long-term safety and efficacy of idebenone.  Patients were enrolled into DELPHI-e and received Catena at 450 mg/d (patients 45 kg body weight) or 900 mg/d (patients > 45 kg body weight). Efficacy was assessed as change from baseline to month 6, 12, 18 and 24 for respiratory function tests (including peak expiratory flow (PEF) , forced vital capacity (FVC), maximum mouth pressures).  Cardiac function tests were conducted by echocardiography and Tissue Doppler Myocardial Imaging, particularly by assessment of the peak systolic radial strain of the left ventricular inferolateral wall, the region of the hear that is the earliest and most severely affected in DMD.  Nineteen patients at age 15.1+2.6 years (mean SD) were enrolled and completed the 24 months study period of DELPHI-E. Eleven patients were taking concomitant glucocorticoids.  Catena was safe and generally well tolerated.  Baseline respiratory function data for the entire study cohort were: FVC: 2.20+0.851, FVC percent predicted: 68.8+25.6%, PEF: 269.4+93.61/min, PEF percent predicted: 66.1+22.3%. The change over time in respiratory as well as cardiac function data will be presented. Data from this long-term open label study will inform on the usefulness of different respiratory and cardiac efficacy outcome measures for intervention trials in DMD. Thus study also will provide additional data to assess the safety and therapeutic potential of idebenone (Catena®) in patients with DMD. Study sponsored by Santhera Pharmaceuticals.

 

Do we know if the chemistry is the same? I am always concerned about what's in the different brands. I give my son the Kirkman one, just thought that it cannot be worse than giving him CoQ10 since Coq10 is also produces but tens of different labs. In fact I believe idebenone is superior to CoQ10, just not sure if the brand we use is "best".

David said:

Catena is simply idebenone by another name. No need to wait on trials or regulators on this one. Docs are already more willing to recommend up to 900mg/day based largely on these results.

Ofelia,

I have been thinking about the same lately, I believe that the brand that is making a difference is the one that "Joshua´s mom" I think she is the one that posted long time ago how Joshua is improving with Idebenone.

I´ll check past posts to see if the brand is mentioned.

Ofelia Marin said:

Do we know if the chemistry is the same? I am always concerned about what's in the different brands. I give my son the Kirkman one, just thought that it cannot be worse than giving him CoQ10 since Coq10 is also produces but tens of different labs. In fact I believe idebenone is superior to CoQ10, just not sure if the brand we use is "best".

David said:

Catena is simply idebenone by another name. No need to wait on trials or regulators on this one. Docs are already more willing to recommend up to 900mg/day based largely on these results.

 

 

Hi Eliane,

 

Joshua is on Deflazacort as well.  I order them from Kirkman.

 

Now I know why we are ordering from kirkman.

Thanks Stephanie

Stephanie Matthes said:

Hi Amrit, 

Sharon asked me to pass along the abstract she found for the poster presentation.. Hope this helps!

 

Results from a two-year open label intervention study with idebenone (Catena®) in the Duchenne muscular dystrophy

 

G.M Buyse, D. Thijs, N. Goemans, M. van den Hauwe, H. Wei, C. Rummey, T Mier, and L Mertens

 

A 12-month double-blind, randomized, placebo-controlled phase II study in 21 patients with DMD (DELPHI Study) has indicated that idebenone (Catena®) may improve cardiac and respiratory function in 8-16 year old patients with DMD. Patients completing the DELPHI study were eligible to enroll into the open label extension study (DELPHI-e) of 24 months duration to further investigate the long-term safety and efficacy of idebenone.  Patients were enrolled into DELPHI-e and received Catena at 450 mg/d (patients 45 kg body weight) or 900 mg/d (patients > 45 kg body weight). Efficacy was assessed as change from baseline to month 6, 12, 18 and 24 for respiratory function tests (including peak expiratory flow (PEF) , forced vital capacity (FVC), maximum mouth pressures).  Cardiac function tests were conducted by echocardiography and Tissue Doppler Myocardial Imaging, particularly by assessment of the peak systolic radial strain of the left ventricular inferolateral wall, the region of the hear that is the earliest and most severely affected in DMD.  Nineteen patients at age 15.1+2.6 years (mean SD) were enrolled and completed the 24 months study period of DELPHI-E. Eleven patients were taking concomitant glucocorticoids.  Catena was safe and generally well tolerated.  Baseline respiratory function data for the entire study cohort were: FVC: 2.20+0.851, FVC percent predicted: 68.8+25.6%, PEF: 269.4+93.61/min, PEF percent predicted: 66.1+22.3%. The change over time in respiratory as well as cardiac function data will be presented. Data from this long-term open label study will inform on the usefulness of different respiratory and cardiac efficacy outcome measures for intervention trials in DMD. Thus study also will provide additional data to assess the safety and therapeutic potential of idebenone (Catena®) in patients with DMD. Study sponsored by Santhera Pharmaceuticals.

 

The results were supposed to be presented in Portugal on the 21-st of October. Has anybody seen them?

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