Duchenne MD Patients with Respiratory Decline Gain Access to Raxone in US

The investigational treatment Raxone (idebenone) could become available to qualifying Duchenne muscular dystrophy (DMD) patients in the U.S. with respiratory decline following the start of an expanded access program (EAP), Clinigen and Santhera Pharmaceuticals announced.

BreatheDMD is an EAP authorized by the U.S. Food and Drug Administration (FDA). Expanded access refers to the use outside of a clinical trial of a medical product still in development for the treatment of a serious or life-threatening disease for which no current comparable or satisfactory treatment is available. The request must be initiated by the patient’s physician.

With BreatheDMD, eligible U.S. patients with DMD will have a mechanism to obtain free-of-charge Raxone through a network of research centers across the country. Details of participating centers will soon be posted here. The list of participating physicians is not yet available.

Eligibility criteria for BreatheDMD include a diagnosis of DMD, a minimum age of 10 years, the ability to swallow medication, and a lung function test showing values of Forced Vital Capacity (the amount of air exhaled after a deep breath) or Peak Expiratory Flow (which measures how fast the air flow in and out of the lungs is) less than or equal to 80 percent, but greater than 25 percent of the predicted value.

The safety and efficacy of Raxone in DMD patients with respiratory decline are currently being evaluated in clinical studies. Santhera is currently recruiting participants for a Phase 3 study (NCT02814019), called SIDEROS, of Raxone in DMD patients taking glucocorticoid steroids.

Patients are advised to discuss with their physician the possibility of participating in the BreatheDMD program and the risks potentially associated with the treatment candidate.

“There is a huge unmet clinical need for patients with DMD and we are pleased to be working in partnership with Santhera to help eligible patients in the U.S. gain access to this important medicine,” John Lagus, head of managed access at Clinigen, said in press release. “This partnership aligns with our mission of getting the right medicines to the right patient at the right time.”

Thomas Meier, PhD, CEO at Santhera, added, “Santhera’s mission remains to develop and bring new treatment options for patients with DMD. Feedback from the DMD patient community highlights the need for access to additional treatment options. Santhera is proud to be launching this EAP, allowing eligible patients to obtain access to investigational idebenone.”

Raxone is a synthetic analog of ubiquinone, a key antioxidant that also plays a role in the cellular production of energy. The FDA granted Raxone orphan drug status for the treatment of DMD. In July 2016, the FDA rejected Santera’s request for accelerated approval of Raxone as a DMD treatment, saying more data were needed.