FDA Approves Gene Therapy For Rare Muscle Disease In Children

The US Food and Drug Administration (FDA) has officially approved the first gene therapy treatment for child patients aged 4 to 5 of Duchenne muscular dystrophy. This debilitating disease causes exponential muscle weakness as the child ages, causing many to not be able to walk by the time they are teenagers. Most die before their 30s. This may change that, but it comes with a hefty price tag of $3.2 million per patient.

Gene Therapy:

The therapy, called Elevidys, is designed to be taken only once, and will be eligible to be given to 6% of people who suffer from Duchenne muscular dystrophy. Brian Abrahams, a Wall Street analyst, predicts that the drug would bring in up to $2.1 billion if it is priced at $2.8 million per person.

Though the price may seem outrageous, it is not very out of place in the context of other gene therapies, some of which have crossed the price threshold of $3 million, but it will still become the second most expensive of its kind. However, to the families of patients who can afford it, it will be absolutely worth it.

The treatment was approved on accelerated approval pathway, meaning clearance of medicines based on urgent need or evidence that they confer clinical benefits. The move was preceded by moving testimonials from several families who tried it experimentally, and recounted their positive experiences at an FDA advisory committee meeting.

Dr Peter Marks, director of the FDA Centre for Biological Evaluation and Research says:

“Today’s approval addresses 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.”

“The FDA remains committed to facilitating the development of innovative new therapies to reduce the impact of debilitating diseases and to improve outcomes and quality of life for those affected.”

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