SMN2

The FDA announced the approval of Assembild to treat patients two years and older with spinal muscular atrophy who are currently receiving SMN2-targeted therapy.

Jim, what should we know about this new treatment option for patients with SMA?

SMA is a genetic condition in which patients lose the spinal muscle function due to a mutation in the SMN1 gene.

There are now three therapies for this condition.

One is a gene therapy that replaces that SMN1 gene, and the other two, significantly cheaper therapies, increase the activity of a backup gene known as SMN2 to increase the missing protein.

It's the first ever therapy designed to directly target the muscle loss that comes with SMA, rather than the underlying genetic defect itself.

It's meant to be used alongside the SMN2-targeting drugs that are already standard of care in patients two years and older.

In the pivotal trial, patients on Isenbeld plus their existing SMA therapy saw real improvement in motor skills over a year, while a comparison group on placebo actually got worse, and that difference was statistically meaningful.

There is one caveat worth mentioning.

We appreciate you listening.

On the regulatory and clinical side, the FDA approved Scholar Rock's ISMBYL'd apidogromab on Friday for adults and children age 2 and older already receiving an SMN2-targeted therapy.

The agency calls it the first spinal muscular atrophy treatment that directly targets muscle loss.

That gives Scholar Rock a way into care without replacing the therapy patients already take.