Roche SMA treatment scores positive trial results

Jun 18, 2018

Roche reported glowing results from a trial for a new treatment for spinal muscular atrophy (SMA).

SMA, a genetic disease that affects the motor nerve cells in the spine, can often be fatal in its most severe form, because it affects the patient’s ability to walk, eat and even breathe.

According to PTC Therapeutics, which is licensing the medication, 90 percent of babies with severe Type 1 SMA who were given the companies’ drug, risdiplam, showed an increase in neuromuscular progress six months after beginning treatment.

If Roche and PTC and win approval for the therapy, analysts predict it could become one of the most expensive in the industry. It would also intensify competition in the market for SMA medications, which is currently dominated by Biogen’s Spinraza (which sells for $750,000 the first year and $375,000 every year after that). Novartis is also developing a one-time gene therapy to treat SMA that analysts say could cost $1.25 million per patient.

If approved, Roche’s new treatment could have an edge in the market because it is taken orally — as opposed to the spine injections needed to take Spinraza about four times a year. 

Read the full Reuters report.