We are excited to share with the Duchenne community, that Santhera Pharmaceutical’s drug Raxone, is the first Duchenne drug approved via the Early Access to Medicines Scheme (EAMS).

The drug’s purpose is to stop respiratory decline, in people living with Duchenne who are not on steroids

Santhera has announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted Raxone (idebenone) a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) for patients with respiratory function decline not taking glucocorticoids in Duchenne muscular dystrophy. 

The aim of the EAMS is to provide patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need.

The MHRA decision allows patients with Duchenne, who meet criteria defined under this scheme, to gain access to Raxone, an investigational medicinal product currently under review for Duchenne for Marketing Authorisation by the European Medicines Agency (EMA). Under the EAMS, and as shown in the public assessment report, Raxone is indicated for slowing the decline of respiratory function in patients with Duchenne from the age of 10 years who are currently not taking glucocorticoids. The decline of respiratory function must be confirmed by repeated measurements prior to initiation of treatment. Raxone can be used in patients previously treated with glucocorticoids or in patients in whom glucocorticoid treatment is not tolerated or is considered inadvisable.

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,” said Thomas Meier, PhD, CEO of Santhera. This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options.”

This is excellent news for patients with respiratory decline in Duchenne muscular dystrophy,” said Janet Bloor, Chair of the Board of Trustees at Action Duchenne. 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 programme and we are delighted to see this first positive opinion in DMD.

 

About the UK Early Access to Medicines Scheme (EAMS)

The UK’s industry-sponsored EAMS aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. Under the scheme, the MHRA provides a scientific opinion on the benefit/risk balance of the medicine, based on the data available when the EAMS submission was made. The opinion lasts for a year and can be renewed. The scheme is voluntary and the opinion from MHRA does not replace the normal licensing procedures for medicines.

 

As ever the best place in the UK to hear updates on Duchenne research is at the Action Duchenne International Conference. This year the event is 10-12 November at the Hilton Birmingham Metropole.