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US FDA approves world's most expensive drug Zolgensma one-time treatment for SMA

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Swiss drugmaker Novartis has received US approval for its spinal muscular atrophy gene therapy Zolgensma® (onasemnogene abeparvovec-xioiT) for the treatment of pediatric patients less than 2years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene). The one time treatment drug is priced at a record $2.125m. Novartis executives have defended the price, saying a one-time treatment is more valuable than expensive long-term treatments that cost several hundred thousand dollars a year. Zolgensma® is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene...
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ICNA

US FDA approves Nusinersen - The first treatment for SMA


The U.S. Food and Drug Administration (FDA) approved Biogen's SPINRAZA™ (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA (nusinersen) ​ is the first and only treatment approved in the U.S. for SMA. In ENDEAR, a pivotal controlled clinical study, infantile-onset SMA patients treated with nusinersen achieved and sustained clinically meaningful improvement in motor function compared to untreated study participants. In ad...
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