Scholar Rock Does Its Homework For Successful SMA Launch

On the back of a stellar showing in Phase III, apitegromab is going to be filed in the US and Europe in the coming weeks for spinal muscular atrophy and CEO Jay Backstrom believes it will be a $2bn blockbuster.

Spinal muscular atrophy
• Source: Shutterstock

Scholar Rock has laid out regulatory and commercialization plans for its closely-watched spinal muscular atrophy (SMA) candidate, apitegromab, at the J.P. Morgan Healthcare conference in San Francisco and is eyeing a US launch by the end of the year.

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