Santhera has suffered several regulatory setbacks around Raxone (idebenone) in Duchenne muscular dystrophy (DMD). In the absence of official approvals, the company is offering the drug for patients in the U.S. through an expanded access program operated by Clinigen.
Under the program dubbed BreatheDMD, eligible U.S. patients with DMD can obtain access to investigational Raxone upon physician request, through a network of research centers.
Santhera is currently on a mission to prove and persuade regulators that Raxone can slow the decline of respiratory function in DMD patients on or without glucocorticoids, which are the only available treatment that can slow the loss of muscle function. But it hasn’t had much luck in that regard.
Although the phase 3 DELOS study met its primary endpoint, showing the drug’s ability in maintaining respiratory muscle function in DMD patients, the EMA’s Committee for Medicinal Products for Human Use rejected a request for a label expansion in 2017. Santhera’s appeal was also struck down last month.
The company’s endeavor in the U.S. was also dealt with a blow in July 2016, when the FDA turned down Santhera’s application for accelerated approval of Raxone in DMD. The agency said it will look at the drug again with data from another phase 3 called SIDEROS, which is now recruiting DMD patients who are on glucocorticoid steroids.
“[W]e now have clarity that successful completion of the SIDEROS trial will provide the necessary data to support NDA filing for Raxone in all DMD patients irrespective of the glucocorticoid use status,” said Santhera CEO Thomas Meier at the time.
The study plans to enroll 266 patients, with results expected in late 2019.