Swiss pharmaceutical company Santhera has announced that the Early Access to Medicines Scheme (EAMS) for Raxone (idebenone) study has accepted its first patients.
Those eligible to be enrolled in the study are Duchenne Muscular Dystrophy (DMD) patients with respiratory function decline not taking glucocorticoids.
DMD is the result of mutations in the dystrophin
gene. The mutation causes low levels of the dystrophin protein and, eventually, progressive degeneration and eventual death of the muscles. Patients typically begin to experience symptoms in between 4 and 5 years old, and affected individuals will likely be non-ambulatory by the time they hit their teenage years.
Raxone become the first drug approved under the EAMS when the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted it a positive scientific opinion in June. Patients with DMD who meet the specified criteria under the scheme can gain access to the drug thanks to the MHRA decision. Enrollment in the new study documents the first use of Raxone in patients and outside clinical trials.
"I am pleased to be able to offer Raxone to several of my patients in respiratory decline, as there are no other medical treatments available," said Dr. Dipansu Ghosh, a respiratory physician at a DMD center based in Leeds in a press release
Throughout the UK, 15 specialist DMD centers have received Raxone administration training under the EAMS requirements, and other additional sites are likely to do the same within the next few months.
"At Action Duchenne we were encouraged by the positive EAMS opinion, earlier this summer. Particularly, for young people living with Duchenne who have respiratory decline," said Janet Bloor, Chair of Trustees of Action Duchenne. "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.”
Raxone is currently indicated for delaying the decline of respiratory function in DMD patients ages 10 and up who are not currently taking glucocorticoids.
For more on new studies pertaining to the rare disease community, follow Rare Disease Report