The efficacy of Elevidys in treating Duchenne muscular dystrophy (DMD) has shown promising results in clinical studies, but are the results enough? The $3 million price tag is considered prohibitive in all DMD communities, and unfortunately many families are trying to access treatment by organizing individual campaigns.
Today we will discuss with DMD WarrioR’s Turkish representative his views on Elevidys. We will share what our Turkish representative wrote to Sarepta and Roche for Elevidys and what responses he received, without comment. We think this interview will be of interest to all DMD patients and their families in the world. How well does Elevidys come in handy in treating DMD? Is it worth 3 million American dollars?
As we explore these questions, we hope to provide clarity and insight into the potential benefits and challenges associated with Elevidys. Our discussion will also touch on the broader implications for access to innovative treatments for DMD patients globally.
Our Questions About Elevidys and the Answers We Received:
Is Elevidys Worth 3 Million American Dollars?
My answer to this is: No, this price is too high.
How much will the normal lifespan of a child taking Elevidys increase?
No one knows the answer to this!
Why Do You Think Elevidys Gene Therapy Is Expensive?
First of all, let me state that I am also a DMD parent. Let’s say I somehow got the Elevidys gene therapy. How much longer will this treatment extend my child’s life? Sarepta needs to answer this question so I can understand whether it is worth 3 million US dollars or not.
Do You Have Any Other Concerns About Elevidys?
Of course there is. For example, how much do CK, AST, ALT levels decrease in children taking Elevidys? Sarepta should explain this in detail and transparently.
What did You Write to Sarepta and Roche?
As DMDWarrior’s Turkish Representative, I first sent a detailed e-mail to Sarepta. I asked when Roche would apply for a license in Turkiye. Sarepta got back to me on my fifth e-mail, I think. They said they would ask Roche about the issue and get back to us. In the meantime, we, as a team, tried to make an appointment with Roche Turkiye, but we were unsuccessful. We never received a response or explanation from Sarepta during this process.
What Did They Answer?
Nothing!
What is the Biggest Question on Your Mind?
Micro-dystrophin is about 30% of the size of unmutated dystrophin.
When we look at clinical trials, Elevidys enables the production of beetwen 30% and 40% micro dystrophin. But I don’t think this data is very meaningful. No one knows how effective this is. Well, according to all this data, is this treatment worth 3 million US dollars?
For example, if Elevidys was produced 100% normal dystrophin, perhaps a cost of $3 million would seem normal in the pharmaceutical industry, but Sarepta also states that Elevidys is not a cure-all.
Do you Have Any Questions You Would Like to Ask Sarepta Through This Interview?
Yes.
How long will this treatment keep my son alive?
Why is Elevidys Gene Therapy treatment $3 million?
What is happening to CK, AST and ALT levels after received Elevidys?
Why don’t they apply for licenses in all countries? [Read More]
Why don’t they respond to DMDWarrioR?
What do you think about the Chilean Ministry of Health expressing concerns about the effectiveness of Elevidys?
Sarepta needs to share all the data and clearly explain why this treatment costs $3 million.
I agree with the statements of the Chilean Minister of Health on this matter. [Read More]
Do you think Elevidys will get approval from the European Medicines Agency (EMA)?
Everyone wants this approval to be given by EMA. But I think the clinical results will not be considered sufficient. In fact, the most likely reason for rejection will be the exorbitant price. Moreover, there are serious studies in Europe, and EMA will consider the effectiveness along with the price when evaluating Elevidys. [Read More]
What Do You Think of Other Clinical Trials?
If you ask my personal opinion, most of them will be more effective and cheaper than Elevidys.
There are currently studies that produce higher levels of micro dystrophin. Some research companies even say they have succeeded in producing normal dystrophin.
What is Your Message to Those Who Follow You in Turkiye?
There are currently significant studies in Turkey, Russia, China, France, Canada and Japan.
News of these studies will soon start hitting the press.
Is There a Study on DMD in Turkiye?
Of course there is. There are very valuable researchers in Turkiye. Their works are progressing rapidly. We are following very closely and are in get in touch.
Maybe the surprise will come from Turkiye.
We would like to thank our Turkish friend for sharing his views. We hope that Elevidys and all other treatment options will reach Turkiye and be covered by the state’s reimbursement.
