Santhera Pharmaceuticals Holding AG submitted an application with the European Medicines Agency seeking approval of its medicine Puldysa to treat respiratory dysfunction in patients with a rare muscle disorder.
The Switzerland-based company is seeking approval of Puldysa, or idebenone, to treat respiratory dysfunction in patients with Duchenne muscular dystrophy, or DMD, who are not using glucocorticoids, a class of steroid hormones. DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. Symptoms of the condition include difficulty in walking and breathing.
In January 2018, the EMA maintained its negative opinion on the company's drug Raxone, which has the same main component idebenone, to treat DMD.
The EMA's Committee for Medicinal Products for Human Use had invited Santhera to present additional data to further link the observed treatment effects on respiratory function outcomes to patient benefit. The company had said it intended to collect further evidence to strengthen the clinical data for Raxone before it refiled its application in Europe.
Raxone is approved in the EU, Norway, Iceland, Liechtenstein, Israel and Serbia for treating Leber's hereditary optic neuropathy — a type of inherited vision loss disorder.
Santhera said in a May 27 press release that its marketing authorization application for Puldysa was backed by data from several clinical trials.
Kristina Sjöblom Nygren, chief medical officer and head of development at Santhera, 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."
In addition, Santhera noted it plans to submit a new drug application with the U.S. Food and Drug Administration following the completion of an ongoing phase 3 trial, dubbed Sideros. The trial is evaluating the safety and efficacy of idebenone in delaying the loss of respiratory function in patients with DMD taking glucocorticoid steroids.
Idebenone has been granted orphan drug designation by European, U.S., Swiss and Australian authorities to treat DMD and has also received a U.S. FDA fast track designation, Santhera added.