United States: In a recent development that has been made by the manufacturers of the gene therapy, the US Food and Drug Administration has approved it as a possible treatment meant for the most patients of muscular dystrophy disease and those with a definite gene mutation.
More about the news
Last year, the drug Elevidys from the company Sarepta Therapeutics was approved for the treatment of only children between the ages of 4 and 5 with Duchenne muscular dystrophy, one of the inherited muscular dystrophies and muscular dystrophies with a proven mutation of the DMD gene related to muscle strength.
FDA approved traditional approval of Elevidys for patients of both genders, four years of age and older, who are confirmed to be eligible with a mutation in the DMD gene, and the accelerated approval for the patients who cannot walk or are non-ambulatory, who are with the same age of 4 and above and with a mutation in the DMD gene.
However the expert committee also says there isn’t enough information about safety to recommend it for use in children under four years.
Elevidys, administered as a one-time intravenous infusion, costs USD 3.2 million per patient and is considered one of the costliest drugs ever developed, as CNN Health reported.
While admittedly steep, such a price is not unprecedented in the context of other one-off gene therapy treatments that have been topped USD 3m-4m per patient in recent years.
What more have the experts said?
Elevvidys was earlier granted the approval by FDA under the merged accelerated approval mechanism that paved the way for medicines for badly needed diseases based on the likelihood that such a medicine would be beneficial.
The drug has been carefully watched since that approval, and last October, Sarepta Therapeutics, the company that produces the therapy, released the results of a confirmatory trial conducted on the drug, stating that the therapy failed the initial test, which was a measure of mobility of kids but succeeded in several other parameters that are usually considered secondary.
According to Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, the approval addressed “an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individual’s health over time,” as CNN Health reported.
More about the approved therapy
This was the first time that such therapy of this kind – a one-time treatment that supplies a functional copy of a gene that is missing and causes disease – would be approved through the accelerated approval system.
This followed emotional stories from families whose loved ones gained benefits testified at an FDA advisory committee meeting.
Duchenne muscular dystrophy manifests itself through muscle deterioration that leads to the inability to walk in teenagers, and most of them do not survive until the age of thirty or so.
It mostly impacts males due to its pattern of genetic inheritance, of which an incidence of 1 in 3,300 boys is estimated.