Santhera Receives UK Approval for AGAMREE
Santhera Pharmaceuticals have announced that AGAMREE (vamorolone) has been approved in the United Kingdom (UK) for the treatment of Duchenne muscular dystrophy (DMD) in patients 4 years of age and older, independent of the underlying mutation and ambulatory status.
The UK’s MHRA, adopting the view of the European Medicines Agency (EMA), acknowledged clinically important safety benefits of AGAMREE with regards to maintaining normal bone metabolism, density and growth compared to standard of care corticosteroids, alongside similar efficacy.
“We are delighted to have secured a third approval for AGAMREE to treat Duchenne from a major regulatory agency, after the U.S. FDA and the EU EMA, within a couple months,” said Shabir Hasham, MD, Chief Medical Officer of Santhera. “In addition to its anti-inflammatory efficacy, both the EMA and the MHRA recognize the benefits of treatment with AGAMREE for bone health and growth, underlining the favorable safety and tolerability profile of this novel medicine compared to conventional corticosteroids. 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.”
“We are delighted that the first drug designed specifically for everyone with Duchenne has been approved in the UK,” said Emily Reuben OBE, Chief Executive of Duchenne UK, and Alex Johnson OBE, Chief Executive of Joining Jack, who are the co-founders of Duchenne UK. “When our sons were diagnosed with Duchenne, we were told that steroids, the standard medication for children with it, would keep them independently mobile for longer. But with harmful side effects. We didn’t think this was good enough, and invested in finding better treatments. Duchenne UK and our partner charities, Joining Jack and the Duchenne Research Fund, funded the early-stage clinical research to test vamorolone in patients, when no-one else would. For it now to be available in the UK to treat DMD is proof that we can find better treatments for Duchenne and change things for Duchenne.”