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Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company, announces 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).
In parallel to the MAA submission to the UK MHRA, Santhera is currently preparing an application to include vamorolone for the treatment of DMD in the Early Access to Medicines Scheme (EAMS) in the UK. 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.
“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,” said Shabir Hasham, MD, CMO of Santhera. “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.”
At the core of the MAA submission are positive data from the pivotal phase 2b VISION-DMD study which comprised a (1) 24-week period to demonstrate efficacy and safety of vamorolone (2 and 6 mg/kg/day) versus placebo and prednisone (0.75 mg/kg/day), followed by a (2) 24-week period to evaluate the maintenance of efficacy and collect additional longer-term safety and tolerability data. In addition, the filing includes data from three open-label studies in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months.
In the US, the Food and Drug Administration (FDA) has set October 26, 2023, as the Prescription Drug User Fee Act (PDUFA) target action date upon which approval of the new drug application (NDA) for vamorolone in DMD is expected. In the EU, a corresponding MAA has been validated and is under review by the European Medicines Agency (EMA) with an expected approval in late 2023. Subject to approvals, Santhera plans to launch vamorolone in both the US and the EU in Q4-2023.
Vamorolone has been granted Orphan Drug status in the US and in Europe for DMD and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD.
Vamorolone is an investigational drug candidate with a mode of action based on binding to the same receptor as corticosteroids but modifying its downstream activity and as such is considered a dissociative anti-inflammatory drug. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore vamorolone could emerge as an alternative to existing corticosteroids, the current standard of care in children and adolescent subjects with DMD. In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity. Vamorolone is an investigational medicine and is currently not approved for use by any health authority.
Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD.
Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need.
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