PPMD was recently joined by Dr. Aravindhan Veerapandiyan of Arkansas Children’s Hospital to discuss the FDA’s recent approval of AGAMREE (vamorolone), a new steroid approved for the treatment of Duchenne muscular dystrophy in individuals aged 2 years and older.
Dr. Veerapandiyan described the study on which AGAMREE was approved, which was a randomized, double-blind, placebo- and prednisone-controlled study over 24 weeks, with an additional 24 weeks of follow-up. The study found that both primary and secondary endpoints were met in the group dosed at 6mg/kg/day. Dr. Veerapandiyan then explained adverse events and the safety profile of AGAMREE. You can learn about common adverse reactions (side effects) and drug safety here. He also highlighted the decreased fracture burden with AGAMREE compared to classical steroids, like prednisone or deflazacort. However, long-term safety data is not yet available, nor is data around cardiac and pulmonary function.
During the question and answer portion of the webinar, Dr. Veerapandiyan provided his clinical perspective on the community’s questions, including how to transition from a classical steroid to AGAMREE, alternative dosing regimens like weekend dosing, the use of AGAMREE for carriers or individuals with Becker, and how to navigate stress dosing. He also emphasized that while we are thrilled to have another option for the treatment of Duchenne, much remains unknown, and a discussion with your neuromuscular specialist is key in understanding whether AGAMREE is right for you.
PPMD is here to support you and answer your questions. You can schedule a PPMD For You appointment with the care team, check out our Access and Coverage Resources for New Therapies, and tune in to our upcoming webinar with Catalyst Pharmaceuticals on Wednesday, November 15 at 1:00pm ET to learn more about how Catalyst is bringing AGAMREE to patients in the US. You can register for the webinar here.