Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side of the coin, the drug decreases bone fragility, insulin resistance, delayed growth, and changes in behavior.
As reported in Neurology Live, the CEO and founder, Pat Furlong, noted that the FDA approval this week brings a therapy option with improved tolerability, potentially fulfilling a critical unmet need.
The treatment is tentatively scheduled to be launched by Catalyst Pharmaceuticals in early 2024.
The VISION-DMD clinical trial (NCT03439670) Phase 2b provided the basis for the FDA approval. The trial is a double-blind, controlled, and placebo study evaluating the safety and efficacy of oral vamorolone administered daily over 24 weeks at doses of 2.0 mg/kg and 6.0 mg/kg. Vamorolone was evaluated against prednisone administered daily at 0.75 mg/kg and placebo during this period.
Medication compliance and persistence involving young boys with Duchenne muscular dystrophy ages 4 to 7, who are ambulatory, were also analyzed over a period of 48 weeks.
The original review was published in June 2021 with almost identical efficacy in both dosed cohorts. 94% of 121 patients were able to complete the study. No vamorolone patient discontinued participation in the study due to adverse events.
Earlier this year, Santhera and Catalyst Pharmaceuticals entered into an agreement whereby Catalyst would acquire the license and commercial rights for AGAMREE® (Vamorolone). The New Drug Application will, therefore, be transferred to Catalyst.
The Catalyst CEO, Patrick McEnany, issued a statement in which he said that AGAMREE appears to have the potential to increase mobility and ambulation and improve patients’ quality of life.
He added that at the time of AGAMREE’s launch, the company will introduce a program to address accessibility, patient deductibles, and copays in an effort to create more affordability for DMD patients.
Dr. Edward Smith, an adjunct professor at Duke University, commented that AGAMREE is a novel steroid providing the benefits of steroid therapy with fewer adverse events.