Prosensa Announces 48-Week Data from a U.S. Phase II Placebo-Controlled Study of Drisapersen in 51 DMD Boys

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Clinically meaningful improvement from 24-week treatment period was maintained for 24 weeks after drisapersen administration ceased

Very encouraging! Who knows exon 53 skipping technology progress to what extent?

Exon 53 skipping is in phase 1 trial at Prosensa and pre-clinical at Sarepta. 

Thank you! I know that this drug is very expensive, howener, I still  want to know how much does it cost a year for treatment.If it is too expensive that I can not afford, as a non-American, is there any way to solve?


Jason Darienzo said:

Exon 53 skipping is in phase 1 trial at Prosensa and pre-clinical at Sarepta. 

I believe the cost a year will be $75,000 to $100,000.  I would write to Prosensa and Sarepta on when the drugs will get to China.  

My God! It's so expensive that few people can afford it in China !  There are about more than 150,000 patients who are counting on the U.S. studies, waiting the drugs coming out。The cost  will  push them  into the abyss of hopelessness again! How can I do to save my son,and who can help me?


Jason Darienzo said:

I believe the cost a year will be $75,000 to $100,000.  I would write to Prosensa and Sarepta on when the drugs will get to China.  

How you calculate this price for this  medicines. any offical information for the cost of medicines it arount 38lacs in indian currency very high we cannot think for purchase of such medicine . or our childeren will die just becoause of medicines

Without health insurance most Americans can not afford it, as well.  However, price can vary greatly country to country with different government subsidies and the company may have different prices for different markets.  Also, when a drug looses its patient protection (in US 7years) the price will be much lower. Before that there will be a way to rise money with charity organization.

 

  

I read about the price in one of Sarepta's conference calls a few years back. I will try to find it.

Interesting article:

 "The World's Most Expensive Drugs"

  http://www.forbes.com/2010/02/19/expensive-drugs-cost-business-heal...


 
VIJENDER BAJAJ said:

How you calculate this price for this  medicines. any offical information for the cost of medicines it arount 38lacs in indian currency very high we cannot think for purchase of such medicine . or our childeren will die just becoause of medicines

Anything on price is just speculation at this point.  It's not even approved.  The investment community has been speculating the drug will cost $400,000 a year, so like 24 million rupees.  This was based on other drugs for rare disease, and the agreement that GSK had with Prosensa for marketing rights to 4 approved drugs, which was over $600 million.

The company might not lose patent protection after 7 years - drug companies frequently are able to manipulate the system and maintain patents beyond that.  And orphan drugs have special protections.  There's also no guarantee any other companies will make the drug after the patent expires.  It's a limited market, others might not choose to invest in the manufacturing required.

When and if the drug is approved (I believe the exon 51 drugs will be at some point), it will be phenomenally expensive.  The Affordable Care Act protects patients' access to expensive drugs, because it outlawed annual and lifetime caps on benefits.  Without it, your plan - employer group or private - could institute a limit to control their costs, and you could be cut off from the drug.  This can and did happen to people with other expensive conditions prior to the law taking effect.   

 

 

I was using an old price estimate.  Wedbush Securities and thestreet.com say $350,000 to $400,000 a year.

Why don't we get it approved first. 

Laws can be changed and the backbone of exon skipping drugs goes well beyond DMD, so it's not going away.

 

Thank you Jason!  I wonder  whether the two companies have plans to bring drugs into  China,when? We are very worried!
 
Jason Darienzo said:

Without health insurance most Americans can not afford it, as well.  However, price can vary greatly country to country with different government subsidies and the company may have different prices for different markets.  Also, when a drug looses its patient protection (in US 7years) the price will be much lower. Before that there will be a way to rise money with charity organization.

 

  

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