Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD). But the company’s efforts to expand its use to a new indication is coming up short.
The Swiss drugmaker has halted development of Enspryng in Duchenne muscular dystrophy (DMD), according to a letter sent to patient organizations. In the notice, Roche said that it had stopped recruiting patients for the phase 2 Shield DMD trial, which is assessing Enspryng’s ability to improve bone muscle health.
The company will continue the study until the interim data collection point later this year. Roche said that it will present six-month bone mineral density data from the trial at a future medical conference.
“We routinely evaluate our portfolio to focus on delivering transformative medicines that address critical gaps in care,” Roche said in the letter. “There are times when our assessment results in difficult decisions that affect an ongoing clinical study. These decisions are never taken lightly and are made with thoughtful consideration, guided by a systematic evaluative framework.”
The company said that new information indicated that it could not meet regulatory requirements as well as recruitment and study completion timelines for the trial or for a potential phase 3 study in the indication and that there was no realistic path to approval. Roche added that fewer than 30 patients are enrolled in Shield DMD, with participants scattered across the U.S. and five European countries.
Roche specified that there is no safety issue at play in its decision and that ongoing investigational studies of the treatment are not affected.
Roche has had mixed results in its attempts to expand the label of Enspryng. Last year, the company reported success in a phase 3 trial of the treatment in myelin oligodendrocyte glycoprotein-associated disorder (MOG-AD), with filings planned this year with regulators in the U.S. and Europe. Additionally, Roche is gunning for U.S. filing for Enspryng in thyroid eye disease despite mixed outcomes from two phase 3 trials. And another phase 3 trial is ongoing for the drug in autoimmune encephalitis.
In a setback in 2024, Roche’s subsidiary Chugai reported that a study of Enspryng in generalized myasthenia gravis (gMG) met its primary endpoint but that the degree of benefit observed “did not reach our expectations.”
Enspryng, which was originally approved by the FDA in 2020, generated (PDF) sales of $364 million Swiss francs ($470 million) in 2025, with $267 million Swiss francs ($345 million) coming from outside of the U.S. Overall sales for the treatment were up 17% year over year.