{"id":486519,"date":"2024-01-15T13:03:15","date_gmt":"2024-01-15T18:03:15","guid":{"rendered":"https:\/\/platohealth.ai\/mhra-approves-safer-medicine-alternative-for-rare-disease\/"},"modified":"2024-01-15T16:18:36","modified_gmt":"2024-01-15T21:18:36","slug":"mhra-approves-safer-medicine-alternative-for-rare-disease","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/mhra-approves-safer-medicine-alternative-for-rare-disease\/","title":{"rendered":"MHRA approves \u2018safer\u2019 medicine alternative for rare disease","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"
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A new drug has been approved in the UK as a safer alternative to corticosteroids to treat Duchenne muscular dystrophy (DMD).<\/p>\n<\/div>\n

\n\"Vamorolone\"Vamorolone<\/div>\n

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved<\/a> Agamree\u00ae<\/sup> (vamorolone), as a safer alternative to corticosteroids, for Duchenne muscular dystrophy (DMD).<\/p>\n

\u201cIn addition to its anti-inflammatory efficacy, both the EMA and the MHRA recognise the benefits of treatment with Agamree for bone health and growth,\u201d Dr Shabir Hasham, Chief Medical Officer of Santhera Pharmaceuticals shared.<\/p>\n

Following the MHRA\u2019s 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.<\/p>\n

The medicine is indicated in patients who are four years of age and over, independent of the underlying mutation and ambulatory status.<\/p>\n

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the first drug designed specifically for everyone with Duchenne has been approved in the UK\u201d<\/p>\n<\/div>\n

\u201cWe are delighted that the first drug designed specifically for everyone with Duchenne has been approved in the UK,\u201d Emily Reuben OBE, Chief Executive of Duchenne UK, and Alex Johnson OBE, Chief Executive of Joining Jack commented, following the MHRA\u2019s decision.<\/p>\n

Vamorolone\u2019s mechanism of action<\/h2>\n

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<\/a>,<\/em> have highlighted the treatment\u2019s potential to \u2018dissociate\u2019 efficacy from steroid safety concerns.<\/p>\n

Clinical efficacy of Agamree for Duchenne muscular dystrophy<\/h2>\n

The European Commission (EC)\u2019s approval was based on data from the VISION-DMD study<\/a> and three other studies, which showed Agamree (vamorolone)\u2019s reported safety benefits<\/a>.<\/p>\n

In the VISION-DMD study, on average, boys given vamorolone maintained growth comparative to those treated with placebo, the findings showed.<\/p>\n

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.<\/p>\n

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In the [VISION-DMD] study, unlike corticosteroids, vamorolone did not result in a reduction of bone metabolism\u201d<\/p>\n<\/div>\n

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.<\/p>\n

\u201cWe 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,\u201d Dr Hasham confirmed.<\/p>\n