Roche Ends Development of Experimental SMA Drug

Rare Daily Staff

Roche said it is discontinuing the development of olesoxime, which was in late-stage testing for spinal muscular atrophy, a rare progressive neuromuscular disease.

Roche purchased the drug in 2015 from Trophos.

In explaining the decision in a letter to the SMA patient community, the company said it had experienced many difficulties formulating the drug as a liquid and the need for an additional late-stage study to determine the most appropriate dose.

The company also acknowledged that the treatment options for SMA have changed dramatically in the last few years. With an effective treatment on the market, it said any new drug would face higher hurdles to win marketing approval and that impacted designs for clinical studies.

While the company initially had encouraging data at 12 months from its ongoing study of olesoxime, it said the most recent analysis at 18 months showed worsening motor function in patients.

“Unfortunately, despite all of our efforts and a strong desire to deliver olesoxime as a medicine to people with SMA, we have concluded that this is not going to be possible,” wrote Sangeeta Jethwa, head of patient partnership, rare diseases for Roche in the letter to the patient community. “Based on all of the available evidence and the continued difficulties described above, we have decided to stop further development of olesoxime.”

The company said it will keep the existing study open until all ongoing participants have an alternative treatment option confirmed.

June 1, 2018
Photo: Sangeeta Jethwa, head of patient partnership, rare diseases for Roche