Vamorolone receives approval by the FDA in the US and positive opinion from CHMP in the EU for use in the treatment of Duchenne muscular dystrophy.
- The US Food and Drug Administration (FDA) approved vamorolone on October 26, 2023 for the treatment of DMD in children and adults aged two years and older. Santhera’s licensing partner for North America, Catalyst Pharmaceuticals, plans to launch vamorolone in the US in Q1-2024. Catalyst Pharmaceuticals has not announced any plans for Canada.
- The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for vamorolone for the treatment of Duchenne muscular dystrophy (DMD) in children and adults aged four years and older. The CHMP is the European Medicines Agency’s committee responsible for human medicines, playing an important role in the authorization of medicines in the European Union.
- In its recommendation for approval, the CHMP acknowledges the positive benefit-risk profile of vamorolone in this patient population, including certain safety benefits of vamorolone compared to standard of care corticosteroids in the treatment of DMD.
- European Commission (EC) decision on marketing authorization is expected in late 2023. Santhera plans for a first commercial launch in Germany in Q1-2024, subject to approval by the EC.
Canadian contributions to the clinical development of vamorolone
Several Canadian sites, clinicians, study personnel and many Canadian DMD families have participated in vamorolone’s clinical trials, playing a significant role in testing the drug’s safety and effectiveness, with many study participants still receiving the drug through expanded access to these trials. Canadian patient organization Defeat Duchenne Canada has made significant financial contributions to the development program of vamorolone.
Clinical evidence
The clinical evidence for the efficacy and safety of vamorolone in the regulatory submission was derived from the positive pivotal VISION-DMD study and three open-label studies (including extensions), in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months, as well as an external comparator study (FOR-DMD) and several clinical pharmacology studies. The studies were carried out by Santhera’s partner ReveraGen and 32 academic clinical trial centers in 11 countries, including multiple Canadian sites.
In the pivotal VISION-DMD study, boys treated with vamorolone on average maintained growth similar to those treated with placebo, whilst those treated with prednisone on average experienced growth stunting. Patients who switched from prednisone to vamorolone after 24-weeks were, on average, able to resume growing in height over the remainder of the study.
On October 27th, 2023 the Journal for Neuromuscular Diseases published the open-access research article “Considering the Promise of Vamorolone for Treating Duchenne Muscular Dystrophy”, providing an independent commentary that considers data related to the drug vamorolone (VBP15) as an alternative steroid proposed for treatment of DMD.
Read next...
New Publication from the NMD4C Clinical Trial Network Strengthens Trial Capacity in Canada
We are pleased to share a new publication from the NMD4C Clinical Trial Network led by Kerri Lynn Schellenberg, Homira Osman, Maria Masnata, Rhiannon Hicks, Corinne Kagan, Ana Stosic, Stacey Lintern, Erin Beattie, Drs. Hanns Lochmüller, Craig Campbell, and Jean K Mah. This work focuses on the implementation of a coordinated Canadian neuromuscular clinical trial network to enhance trial readiness, capacity, and access in rare disease research.
Canadian-Led Phase II Studies Advance Vamorolone Evidence in Duchenne Muscular Dystrophy
NMD4C investigators contributed to two phase II studies evaluating vamorolone in boys with Duchenne muscular dystrophy across early childhood and adolescence, demonstrating safety, favorable pharmacokinetics, and potential efficacy. Led by Dr. Jean Mah and conducted across Canadian clinical trial sites with national and industry collaborators, the findings supported Health Canada approval of vamorolone as the first treatment for DMD in Canada. Leaders from both the clinical and patient advocacy communities, including Defeat Duchenne Canada, highlighted the impact of collaboration among patients, families, clinicians, and researchers in advancing care and improving access to new therapies.
Advancing AFC in Neuromuscular Medicine: Program Launch & September Workshop
The Royal College has approved National Training Standards for the AFC in Neuromuscular Medicine, creating a formal pathway to recognize enhanced competence with the DRCPSC credential. This designation establishes a national training standard, supports portable certification, and strengthens clinical capacity across Canada. PGME offices can now apply to offer accredited AFC programs, with a goal of five programs submitting applications by the end of 2026.