Positive trial results for an oral drug to treat spinal muscular atrophy (SMA) are bringing Roche one step closer to breaking into the high-cost treatment area.
The company reported today that when compared to a placebo, its drug risdiplam has been shown to be effective in providing statistically significant symptom improvements in patients aged 2-25 with types 2 or 3 SMA.
Roche is gunning for a place in the SMA market alongside gene therapies such as Biogen’s Spinraza — which lists for $750,000 for the first treatment and $375,000 thereafter — and Novartis’s Zolgensma — which, at $2.1 million for a one-time dose, is the world’s most expensive drug.
In addition to efficacy, Roche has been touting the safety profile of risdiplam and the fact that it can be taking orally, rather than via injection.
Read the Reuters report.