UK Agency Grants Raxone Temporary Approval to Treat Certain DMD Patients

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Raxone (idebenone)  has been granted temporary scientific approval to be used to treat certain patients with Duchenne muscular dystrophy (DMD) in the United Kingdom (UK).

The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted Santhera Pharmaceuticals‘ drug candidate a positive Early Access to Medicines Scheme (EAMS) scientific opinion. The EAMS allows Raxone to be temporarily indicated for slowing the decline of respiratory function in patients with DMD not younger than 10 years of age who are currently not on glucocorticoids.  .

Raxone is  designed to improve energy production in the mitochondria (the cell’s powerhouse) of muscle cells.

Before initiating treatment, patients must have a decline of respiratory function confirmed by repeated measurements. Raxone can be used in patients who have previously received treatment with glucocorticoids, who are unable to tolerate glucocorticoids, or for whom this type of treatment is not recommended.

“We’re proud to receive the positive EAMS scientific opinion for Raxone in the UK and to have our lead compound designated as a promising innovative medicine, the first for a drug intended for the treatment of DMD,” Thomas Meier, PhD, Santhera’s chief executive officer, said in a press release. “This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options.”

Under the EAMS, patients with life-threatening conditions can have access to medicines that have not yet received a marketing authorization. Without replacing the normal licensing procedure for new medicines, the MHRA provides a scientific opinion and assesses the benefit/risk balance of the candidate treatment based on the available data. The temporary regulatory status can last for up to one year and can be renewed.

Raxone is currently under review for marketing authorization by the European Medicines Agency (EMA).

“This is excellent news for patients with respiratory decline in Duchenne muscular dystrophy,” Janet Bloor, chair of the Board of Trustees at Action Duchenne said in the release. “The need for new treatments in DMD is very great and the EAMS can help to accelerate access for patients. Action Duchenne was pleased to provide advice during the development of the EAMS program and we are delighted to see this first positive opinion in DMD.”

Santhera started recruiting patients in February 2017 for a Phase 3 SIDEROS clinical trial (NCT02814019), evaluating Raxone in slowing respiratory decline in DMD patients being treated with glucocorticoids.

The trial is designed to measure the efficacy of Raxone by the change in the forced vital capacity percent predicted (FVC % p), a measure of how much a person can exhale, and other secondary outcomes. Results for the SIDEROS trial are expected to be known in mid-to-late summer 2019.