Roche’s Evrysdi wins FDA approval in newborns with spinal muscular atrophy

Roche (SIX:RO, ROG; OTCQX:RHHBY) has announced that the FDA has approved a label extension to allow the use of Evrysdi (risdiplam) in babies younger than two months old with spinal muscular atrophy (SMA).

SMA is a genetic disease associated with progressive muscle wasting and weakness that affects roughly 1 in 11,000 babies.

The drug first won FDA approval in 2020 for adults and children at least two months old.

The label extension was based on interim efficacy and safety data from the RAINBOWFISH study in newborns.

That data demonstrated the majority of pre-symptomatic babies who received Evrysdi achieved developmental milestones related to sitting and standing. In addition, half of the babies were walking after 12 months of treatment.

“The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA before symptoms start to arise can help babies to achieve motor milestones,” said Dr. Richard Finkel, …

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3 notable types of innovative drugs from 2020

Chemical structure of the COVID-19 therapy remdesivir. Image is from Wikipedia.

Last year, FDA approved 53 drugs, leading the industry to describe 2020 as “a strong year for new drug therapy.”

There are several drugs that stand out, according to Todd Wills, the co-author of a study that analyzes how innovative drugs are based on their structure.

The drugs that follow are examples of notable innovative therapies.

[Related: Here’s what molecular shape can tell you about pharma innovation]

1. COVID-19 therapies

One of the prominent drugs that stands out as structurally novel is remdesivir from Gilead Sciences (NSDQ:GILD). The first COVID-19 treatment to win FDA approval, remdesivir (Veklury), was first developed as an Ebola treatment. But the broad-spectrum antiviral also showed promise against the SARS-CoV-2 virus.

Gilead recently announced that sales of remdesivir beat …

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