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FDA Approves World's Costliest Drug For SMA

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Spinal muscular atrophy or SMA is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement.

The most common form of SMA is caused by a defect in the SMN1 gene that leads to deficiency of a motor neuron protein called SMN, which is necessary for normal motor neuron function.

The FDA approved Zolgensma, developed by Novartis (NVS), on May 24, 2019, for the treatment of spinal muscular atrophy (SMA) in children less than two years of age.

Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy that targets the cause of SMA by delivering a copy of human SMN gene into the target motor neuron cells.

Novartis priced the drug at $2.125 million, making it the most expensive medicine in the world. Analysts estimate peak annual sales of between $1.8 billion and $2.6 billion for the drug.

Zolgensma will compete with Biogen's Spinraza (nusinersen), which was approved in December 2016, for the treatment of SMA in pediatric and adult patients.

Zolgensma, the first and only gene therapy approved by the FDA, is a one-time treatment whereas Spinraza has to be administerd multiple times. Spinraza, priced at $750,000 for the first year, and $350,000 afterwards, brought in revenue of $1.724 billion in 2018.

Novartis expects to get approval for Zolgensma in Europian Union and Japan this year.

Zolgensma has a boxed warning for acute liver injury.

Now, let's take a look at the new drugs that won FDA approval in May.

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