Sarepta Therapeutics announced the approval of Elevidys (delandistrogene moxeparvovec) by the U.S. Food and Drug Administration (FDA). Elevidys is the first gene therapy approved to treat Duchenne muscular dystrophy (DMD).
Elevidys is a one-time gene therapy designed to address the underlying genetic cause of Duchenne. The FDA granted accelerated approval for this indication based on observing Elevidys micro-dystrophin expression in patients who received the treatment. It should be noted that continued approval for this specific indication may be subject to further verification and confirmation of clinical benefits in subsequent trials.
However, it's important to mention that Elevidys is unsuitable for patients with any deletion in exon eight and/or exon 9 of the DMD gene. The approval of Elevidys was based on the increase in micro-dystrophin protein expression observed in skeletal muscle.
The decision to grant accelerated approval was based on the trial, which has two parts. In the first part, children with DMD were treated with either Elevidys or a placebo and followed for 48 weeks. Post that, children who received a placebo in the first part were treated with Elevidys and a placebo was administered to those who were treated with Elevidys. There was an improvement in micro-dystrophin, a surrogate endpoint expected to show long-term benefits.
It's important to note that children treated with Elevidys have experienced some adverse effects, including severe acute liver injury, immune-mediated myositis, and myocarditis. Other side effects were vomiting, nausea, increased liver function test results, fever (pyrexia), and low platelet count (thrombocytopenia).
Elevidys delivers a gene that produces Elevidys micro-dystrophin, a shortened protein (138 kD) compared to 427 kDa dystrophin protein. The gene therapy's ability to address the underlying genetic cause of the disease represents a significant step forward in combating this debilitating condition.
Elevidys received approval through the Accelerated Approval pathway, which Food and Drug Administration grants for therapies which treat severe or life-threatening diseases and have high unmet needs. The approval was given based on the surrogate endpoint, which is likely to show benefit in the long term. Accelerated approvals are aimed at getting early access to drugs.
In the trial, Elevidys has not demonstrated benefits in motor function. Sarepta Therapeutics is expected to complete the clinical trial to establish the benefit of gene therapy. Physical function and mobility are assessed in the trial, and FDA takes the final decision based on data availability.
Elevidys cost is $3.2 million per patient, which makes it one of the most expensive treatments.
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The United States Food and Drug Administration has approved Viltepso (viltolarsen) for treating patients suffering from Duchenne muscular dystrophy (DMD) who have a mutation in the DMD gene that is amenable to exon 53 skipping. FDA has reported that this is the second drug approved for patients suffering from this mutation.
Approximately 8% of the patients with DMD have a mutation that is amenable to exon 53 skipping.
The approval has been granted to NS Pharma.
The drug approval is based on the two clinical trials enrolling 32 male patients and had genetically confirmed DMD. Improvement in the dystrophin levels was reported in one of the trial which enrolled sixteen patients, dystrophin level improved from 0.6% to 5.9% at week 25.
The clinical benefit of the drug has not been established.
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The drug has received the accelerated approval, the company has to conduct a clinical trial confirming the efficacy of the product. The current study is planned to assessed the efficacy of Viltepso, if it can improve the time to stand in patients suffering from DMD and have the specific mutation.
The product has received Priority Review designation in addition to the accelerated approval.
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About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy, a common type of muscular dystrophy is a genetic disorder which causes the muscle deterioration and weakness. The disease is caused because of the mutation in the DMD gene which results in the absence of dystriophin, protein responsible for keeping the muscle intact. Symptoms are reported at the age of three to five years which further wosen with age. The disease usually affects the males and rarely affects females.
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