On 14 September 2017, the CHMP of the European Medicines Agency (EMA) adopted a negative opinion, recommending the refusal of a change to the marketing authorisation for the medicinal product Raxone. The change concerned the addition of a new use of Raxone in patients with Duchenne muscular dystrophy to slow their gradual loss of breathing ability. (previous article link here). Raxone® is thought to help improve production of energy by restoring mitochondrial function, thereby preventing cellular damage and loss of muscular function observed in young people living with Duchenne.
Santhera requested a re-examination of the initial opinion. After considering the grounds for this request, the CHMP re-examined the opinion, and confirmed the refusal of the change to the marketing authorisation. After re-examination, the Committee was still of the opinion that the strength of the data on the benefits of Raxone in patients with Duchenne muscular dystrophy was limited. The CHMP therefore maintained its opinion that the benefits of Raxone® did not outweigh its risks in this indication and recommended that the change to the marketing authorisation be refused. Further information around the proceedings of the CHMP meeting can be accessed here.
The CHMP has invited Santhera to present additional data to further link the observed treatment effects on respiratory function outcomes to patient benefit. Santhera intends to collect further evidence to strengthen the clinical data package for Raxone® in preparation of a refiling of a Marketing Authorization Application (MAA) in Europe. Santhera will continue working with regulatory authorities to try bring this treatment option to young people living with Duchenne. Their press release can be accessed here.
Thomas Meier, PhD, CEO of Santhera said: “We are very grateful to the DMD community, patients, their caregivers and treating doctors, for their relentless support. Santhera remains fully committed to realizing the potential of Raxone in treating patients with DMD through its clinical trial program and disease awareness efforts in close cooperation with the medical community and patient organizations.”
Furthermore, Santhera confirmed that this update had no effect on ongoing clinical trials and those enrolled in the Early Access to Medicines Scheme here in the UK further information can be found here (insert article).
Action Duchenne is in contact with Santhera and will be updating the community as further developments arise. In the meantime, if you have any other questions or queries with regards to the above or in any other areas of research and clinical development, please feel free to contact Neil Bennett, Director of Research: neil@actionduchenne.org
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