Santhera Pharmaceuticals has updated the timeline for the ongoing assessment by the European Committee for Medicinal Products for Human Use (CHMP) of its Duchenne muscular dystrophy (DMD) drug, Raxone (idebenone).
In June 2016, the European Medicines Agency’s (EMA) CHMP validated a marketing authorization application (MAA) for Raxone as a treatment for DMD in patients with respiratory function decline who were not receiving glucocorticoid therapy.
The recent submission was filed as a type II variation of an existing MAA for Raxone. If approved, the new indication will include patients with DMD who are not currently taking glucocorticoids but who have been previously treated with them, and patients for whom glucocorticoid treatment is not a viable option.
“Santhera is in ongoing, constructive discussions with the CHMP, and we are now expecting to receive a request for supplementary information to further support the clinical relevance of our data. We are working closely with the CHMP to conclude the application process and anticipate an opinion in Q3 2017,” Thomas Meier, PhD and CEO of Santhera, said in a press release.
The outcomes of the Phase 3 DELOS study, titled “Treatment Effect of Idebenone on Inspiratory Function in Patients with Duchenne Muscular Dystrophy,” were published in Pediatric Pulmonology.
Raxone is an investigational drug designed to improve energy production in the mitochondria (which provides power to cells) of muscle cells.
Raxone is approved in Europe to treat visual impairment in adults and children, starting at age 12, with Leber’s hereditary optic neuropathy (LHON). In the United States, the U.S. Food and Drug Administration (FDA) has designated idebenone an orphan drug for the treatment of both LHON and DMD.
In related news, Santhera is currently recruiting patients for a new Phase 3 clinical trial evaluating Raxone in slowing respiratory decline in DMD patients who are receiving treatment with glucocorticoids.
The SIDEROS study (NCT02814019) will enroll 266 DMD patients who will either receive Raxone 900 mg daily or a placebo for 78 weeks.
Participants who complete the trial will be eligible to enroll in an extension study where all patients receive Raxone. Results are expected to be released during the second half of 2019. Duchenne patients or caregivers may contact Jodi Wolff for more information regarding enrollment.