A new drug for treating a rare disease in infants and toddlers is priced at $425,000 per year over five years and is poised to become the nation's most expensive drug.
Novartis said it will price the one-time drug,The FDA approved Zolgensma for children under the age of two with spinal muscular atrophy, a rare inherited disease caused by a mutation in the survival motor neuron gene. This defect can disrupt production of a protein and cause muscle-controlling motor neurons to die.
Acting FDA Commissioner Ned Sharpless described the approval as a"milestone in the transformational power of gene and cell therapies" that have the potential to treat a range of diseases. and medical technology. It will also confront private and government insurers with challenge of covering the costs of what is poised to become the nation's most expensive drug.
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