MHRA approves ‘safer’ medicine alternative for rare disease

A new drug has been approved in the UK as a safer alternative to corticosteroids to treat Duchenne muscular dystrophy (DMD).

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Agamree® (vamorolone), as a safer alternative to corticosteroids, for Duchenne muscular dystrophy (DMD).

“In addition to its anti-inflammatory efficacy, both the EMA and the MHRA recognise the benefits of treatment with Agamree for bone health and growth,” Dr Shabir Hasham, Chief Medical Officer of Santhera Pharmaceuticals shared.

Following the MHRA’s UK approval, Agamree is therefore not only the first medicinal product fully approved in the EU and UK for Duchenne, but it is the first treatment that is approved for the condition in DMD in the EU, UK and the US collectively.

The medicine is indicated in patients who are four years of age and over, independent of the underlying mutation and ambulatory status.

“We are delighted that the first drug designed specifically for everyone with Duchenne has been approved in the UK,” Emily Reuben OBE, Chief Executive of Duchenne UK, and Alex Johnson OBE, Chief Executive of Joining Jack commented, following the MHRA’s decision.

Vamorolone’s mechanism of action

According to Santhera, vamorolone binds to the same receptor as glucocorticoids, but its downstream activity is modified. Previous research, including a 2022 paper published in JAMA Neurology, have highlighted the treatment’s potential to ‘dissociate’ efficacy from steroid safety concerns.

Clinical efficacy of Agamree for Duchenne muscular dystrophy

The European Commission (EC)’s approval was based on data from the VISION-DMD study and three other studies, which showed Agamree (vamorolone)’s reported safety benefits.

In the VISION-DMD study, on average, boys given vamorolone maintained growth comparative to those treated with placebo, the findings showed.

While individuals given prednisone, on average, experienced growth stunting. Data also showed that after 24-weeks, the patients who switched from prednisone to vamorolone were able to, on average, resume growing in height over the remaining time in the study.

In the study, unlike corticosteroids, vamorolone did not result in a reduction of bone metabolism, Santhera noted. Patients did not experience a significant reduction of bone mineralisation in the spine after 48 weeks.

“We are working towards making Agamree available to patients in the UK in the second half-year 2024, after NICE completes its pricing review. Initial European launch will be in Germany in Q1,” Dr Hasham confirmed.

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