The first DMD gene therapy SRP-9001 may cost 4 million US dollars
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The first DMD gene therapy SRP-9001 may cost 4 million US dollars
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The first DMD gene therapy SRP-9001 may cost 4 million US dollars.
SRP-901 (delandistrogene moxeparvovec), developed by Sarepta Therapeutics, will easily be the main talking point at the Muscular Dystrophy Association of America (MDA) 2023 Clinical & Scientific Meeting, March 19-22, 2023.
Despite the late-stage regulatory issues, company executives and patient advocates at the Neuromuscular Diseases conference here predict that the FDA will still approve the first drug against Dushi on May 29, 2023. Gene therapy for muscular dystrophy (DMD).
MDA President and CEO Dr. Donald S. Wood said he was unsure why the federal agency recently announced it would convene a meeting of independent experts to review Sarepta’s application .
“They see something wrong in the data. I don’t know what, but I’m sure it’s not a safety issue. It’s probably an efficacy issue. If it was safety, they wouldn’t even consider the initial ruling in May.”
Dr. Wood hopes the FDA will approve the treatment this year. “Newly diagnosed patients are in a bad situation. They hear about gene therapy and the family will want to jump in right away. They will be very upset when they learn the fastest appointment may be a year away.”
Dr Sharon Hesterlee, chief research officer at MDA, said it was difficult to speculate on what might happen. “If the FDA didn’t have any questions about the drug — if they loved it or hated it, we wouldn’t have an advisory committee,” she said. “But that doesn’t mean it won’t be approved. At the advisory committee meeting we heard ) until it goes, we won’t know. But they say it won’t delay a decision.”
Paul Melmeyer, the MDA’s vice president for public policy and advocacy, said he remains “excited and eager” to see the drug approved in the coming months, as originally envisioned. “We never considered it an issue to bring in outside experts to really ensure the safety and efficacy of a treatment. By doing that, the community also had an opportunity to engage and express their views.”
Diana Castro, M.D., a pediatric neurologist at Children’s Health in Dallas, said she doubts the new treatment will retail for less than $4 million, nearly as much as Novartis’ onasemnogene abeparvovec xioi (Zolgensma®) for spinal muscular atrophy (SMA). ) twice the price of $2.1 million.
Cost isn’t the only hurdle, however, said Dr. Castro, who opened her own neurology and neuromuscular care center in suburban Denton during the MDA meeting.
“About 14% of DMD patients may be antibody positive and therefore ineligible for Sarepta gene therapy,” she said. “Many of the boys I treat have a chance if they are approved for use in children ages 4 to 7. Even if they are approved, insurance Nor will we be allowed to start before the end of the year.”
Dr. Barry J. Byrne of the University of Florida’s Powell Gene Therapy Center (PGTC) in Gainesville, who is also the MDA’s chief medical advisor, predicts that SRP-9001 will cost at least $3.5 million. This, combined with a larger potential patient pool, could mean a significant increase in the overall cost to the healthcare system.
“You have to think about the total cost to the provider ecosystem. There are 10 times as many DMD patients as there are SMA patients,” Dr. Byrne said.
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What is DMD gene therapy?
DMD (Duchenne muscular dystrophy) gene therapy is a type of medical treatment that aims to correct or replace the mutated DMD gene that causes Duchenne muscular dystrophy.
Duchenne muscular dystrophy is a genetic disorder that affects muscle function and leads to progressive muscle weakness and wasting.
It is caused by mutations in the DMD gene, which provides instructions for making a protein called dystrophin that is essential for muscle function.
DMD gene therapy involves introducing a functional copy of the DMD gene into the patient’s cells using a vector, which is usually a harmless virus that has been modified to carry the new gene.
Once inside the cells, the new gene can produce dystrophin, which may improve muscle function and slow down the progression of the disease.
There are several different approaches to DMD gene therapy, including gene replacement therapy, exon skipping therapy, and CRISPR/Cas9 gene editing.
Some of these approaches are still experimental and are being evaluated in clinical trials, while others have already been approved for use in some countries.
However, more research is needed to determine the long-term safety and effectiveness of DMD gene therapy.
What factors caused DMD ?
The mutations that cause DMD are usually inherited in an X-linked recessive pattern, which means that the mutated gene is located on the X chromosome.
Since males have only one X chromosome, they are more likely to be affected by DMD than females. Females can also be carriers of the mutated gene and may pass it on to their children.
Most cases of DMD are caused by large deletions or duplications of genetic material within the DMD gene.
These mutations can disrupt the normal function of the gene and prevent the production of dystrophin.
In rare cases, DMD can also be caused by point mutations or small insertions or deletions within the DMD gene.
These mutations can also disrupt the normal function of the gene and prevent the production of dystrophin.
Overall, the specific factors that cause DMD are genetic mutations in the DMD gene that result in a deficiency or absence of dystrophin, which leads to muscle weakness and wasting.
The first DMD gene therapy SRP-9001 may cost 4 million US dollars
(source:internet, reference only)
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