UK Agency Renews Early Access Program for Raxone to Treat Duchenne MD

People with Duchenne muscular dystrophy (DMD) in the U.K. will continue to have access to Raxone (idebenone) to treat their disease, after the Medicines and Healthcare products Regulatory Agency (MHRA) renewed its Early Access to Medicines Scheme (EAMS) with the therapy’s developer, Santhera Pharmaceuticals.

Raxone has been available in the U.K through EAMS based on a positive scientific opinion issued by MHRA in June 2017. It is not a commercially accessible, approved treatment.

This renewal extends Raxone’s availability for another year for DMD patients ages 10 and older, who are in respiratory decline and not taking glucocorticoids. Further details are available here.

More than 40 patients are currently enrolled in EAMS at specialized DMD centers across the U.K.

“We are very pleased about the renewal of the EAMS for Raxone which allows patients with DMD who have progressed to the stage of respiratory decline and have no alternative therapeutic options available, to receive treatment,” Thomas Meier, PhD, CEO at Santhera, said in a press release.

“The MHRA renewal comes as a sign of continued recognition of both the high unmet medical need of DMD patients and the positive benefit-risk of Raxone in this population,” Meier added.

“We have waited many years to realize the opportunity for early access to potential new treatments,” said Janet Bloor, chair of the U.K. charity Action Duchenne and mother of a young man with Duchenne.

Bloor underscored Santhera’s work in addressing the underlying problems with respiratory failure. “Time is something we don’t have with Duchenne and I welcome the renewal of the EAMS by the MHRA after effective consultation with the patient community,” Bloor added.

Raxone is available to patients who previously received treatment with glucocorticoids, or in whom glucocorticoids are not tolerated or are inadvisable.

EAMS gives patients access to treatments for life threatening or seriously debilitating conditions with unmet medical need before a therapy’s regulatory approval. The MHRA assesses the medication’s benefit-risk balance, based on available data. If approved, early access lasts for one year and may be renewed. Medications under EAMS are still subjected to the standard licensing procedures.

Raxone is available to eligible DMD patients in the U.S. through Santhera’s expanded access program (EAP).

Previous requests for Raxone’s approval as a DMD treatment have been rejected in both Europe and the U.S, whose regulatory agencies found data given in support of these applications less than adequate. Santhera is now conducting a new Phase 3 clinical trial, called  SIDEROS (NCT02814019), to evaluate Raxone’s ability to prevent respiratory decline in DMD patients not using glucocorticoid steroids.

Patients are still being recruited at sites across the U.S., Europe and Israel; information is available here. Results are expected in late 2019.

Treatment with Raxone is approved in the EU, Norway, Iceland, Liechtenstein and Israel for Leber hereditary optic neuropathy, an inherited form of vision loss.