The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) have accepted Biogen’s applications for a higher dose regimen of spinal muscular atrophy (SMA) treatment Spinraza ...

Spinal muscular atrophy (SMA) therapy Spinraza has been a key earner for Biogen in recent years, but with sales now heading into reverse, the company is looking to extend its franchise.

There was no treatment until Doctor Adrian Krainer with Cold Spring Harbor lab on Long Island led the way to creation of Spinraza, a first FDA-approved treatment for Spinal Muscular Atrophy.

In 2016, Spinraza became the first approved treatment for SMA, with exceptional results in infants with severe SMA – so exceptional, it was considered unethical to continue enrolling children ...

However while Spinraza had a very strong launch in the first half of 2017, its US sales faltered in Q3 to $197.6 million, barely above the $194.8 million achieve in the previous quarter.

The two other SMA stalwarts on the market, Biogen’s oligonucleotide Spinraza and Novartis’ gene therapy Zolgensma, are delivered intrathecally (into the cerebrospinal fluid around the spinal ...