Santhera Pharmaceuticals has filed a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the approval of Puldysa (idebenone) for the treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD).
The Swiss pharma company is looking for a conditional marketing authorization (CMA) for idebenone for DMD patients who are not using glucocorticoids for the treatment of their respiratory dysfunction.
Santhera’s application is supported by the findings of a phase 2 trial, called DELPHI, the long-term DELPHI-Extension study, the phase 3 DELOS study and the SYROS study, which was recently completed.
The MAA also includes long-term data collected from patients who completed the DELOS study and continued to be subjected to idebenone treatment for up to six years.
Santhera chief medical officer and development head Kristina Sjöblom Nygren said: “The new data included in this regulatory submission confirm clinically relevant patient benefits and long-term therapeutic efficacy with idebenone in patients with DMD. Thereby, we have closed earlier data gaps and respond to requirements from the regulatory authorities.”
The Swiss pharma company also has plans to file a new drug application (NDA) for idebenone with the US Food and Drug Administration (FDA) after completing an ongoing phase 3 trial called SIDEROS.
The late-stage double-blind, placebo-controlled trial is evaluating the efficacy and safety of the investigational drug in delaying the loss of respiratory function in DMD patients, who have been on treatment with glucocorticoid steroids over a period of 18 months.
The SIDEROS trial will enrol a total of 266 patients and will be held at nearly 60 clinical trial sites across Europe, the US and Israel. Patient recruitment for the late-stage trial is expected to be wrapped in the fourth quarter of 2019.
Idebenone was granted orphan drug designation for the treatment of DMD by European, US, Swiss and Australian regulators. In the US, the drug has a fast track designation.
A synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1), idebenone is said to be capable of stimulating mitochondrial electron transport, supplementing cellular energy levels, and reducing and scavenging reactive oxygen species (ROS).