The US Food and Drug Administration (FDA) has approved a gene therapy product that costs more than two million dollars (1.78 million euros) for a single dose. The drug Zolgensma is said to be used to treat the hereditary disease spinal muscular atrophy (muscle wasting) in infants and toddlers. The manufacturer Novartis announced that a single treatment should replace a more expensive lifelong therapy of the disease.
The FDA approved the use of the drug in children up to two years of age. US media reported it was the most expensive drug ever approved by the FDA. Zolgensma is initially available only in the US. Novartis also claims to seek approval in Europe and Japan.
Children who have the most severe form of spinal muscular atrophy often die before their second birthday. Those affected have difficulty breathing, swallowing and holding their heads. Novartis said a single dose of Zolgensma should replace a defective or missing gene. In clinical trials with the drug, patients had shown improvements that did not occur in the normal course of the disease. Among other things, they could have sat and talked.
