Santhera Pharmaceuticals recently launched an expanded access program (EAP) to allow certain Duchenne muscular dystrophy (DMD) patients in the U.S. to gain access Raxone (idebenone).
Raxone is not approved to treat Duchenne MD by the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA).
The BreatheDMD EAP program will allow eligible patients who are age 10 or older and with diminishing respiratory function to access the investigational treatment at no cost. It will be made available through a network of research centers nationwide.
EAPs are protocols, established FDA guidance and permission, that make potential treatments available to people with serious or life-threatening conditions who have few or no adequate treatment alternatives.
“Several patient advocacy organizations in the U.S., including Parent Project Muscular Dystrophy (PPMD), Jett Foundation, CureDuchenne and the Muscular Dystrophy Association (MDA) have consistently mentioned that patients need access to additional treatment options, especially older patients in respiratory decline,” Thomas Meier, PhD, chief executive officer of Santhera, said in a press release. “Santhera is proud to be launching this EAP, allowing eligible patients to obtain access to investigational idebenone.”
Raxone has a complex regulatory past. An arm of the EMA, the Committee for Medicinal Products for Human Use (CHMP), has twice recommended against extending its approval to include Duchenne, most recently in January. And the FDA declined to grant Raxone accelerated approval as a DMD treatment in 2016. It is approved by both agencies to treat Leber’s hereditary optic neuropathy, a mitochondrial disease.
In a Phase 3 DELOS clinical trial (NCT01027884), Raxone was shown to slow the loss of respiratory function in treated patients. But the FDA and EMA have requested additional data supporting the finding.
Another Phase 3 trial(NCT02814019), SIDEROS, is now evaluating Raxone’s ability to prevent respiratory decline in DMD patients using glucocorticoids. The study is expected to conclude in mid-2019.
Santhera announced that DMD patients in the U.K had begun receiving Raxone under a similar early access scheme in September 2017.
That decision was applauded by Action Duchenne, an organization supporting research into Duchenne treatment.
“I am delighted that respiratory function is being considered by the regulatory agencies. This will hopefully pave the way for more potential treatments that may benefit the wider spectrum of DMD patients,” said Janet Bloor, Chair of Trustees at the organization.