Santhera submits marketing authorisation application to the UK MHRA for Valmorolone in Duchenne Muscular Dystrophy
On March 2, 2023 – Santhera Pharmaceuticals (SIX: SANN) made an announcement that it has submitted a marketing authorization application (MAA) to the UK Medicines and Healthcare products Regulatory Agency (MHRA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD).
Santhera is also preparing an application to include vamorolone for the treatment of DMD in the Early Access to Medicines Scheme (EAMS) in the UK. EAMS is a regulatory scheme in the UK that allows patients with life-threatening or seriously debilitating conditions to gain access to new medicines that do not yet have a marketing authorization, hence not yet been approved for general use.
Vamorolone is an investigational drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. It showed a good safety and tolerability profile in the studies as well as potential to ‘dissociate’ efficacy from steroid safety concerns. Therefore, vamorolone could emerge as an alternative to existing corticosteroids.
This is what Shabir Hasham, MD, CMO of Santhera has to say about the news:
“We are proud to have completed the third submission for vamorolone in DMD to a major regulatory agency within a few months. This underpins our determination to bring a new treatment to patients in need of effective and well-tolerated therapies. We look forward to working closely with the MHRA during the MAA and EAMS review process with the goal of quickly providing an emerging therapy to patients in the UK.”
To read the full press release please click here:
