believe that AGAMREE may offer the potential of increasing the duration of ambulation and mobility in these patients, thereby significantly improving their overall quality of life. We expect to launch the product in the first quarter of 2024. At that time, we will introduce a comprehensive financial assistance program aimed at helping ensure accessibility and minimizing patient co-pays and deductibles, thereby enhancing affordability for all DMD patients. We look forward to successfully commercializing this product with a continued commitment to serving our patient communities.”
Upon the transfer of AGAMREE’s NDA into its neuromuscular franchise, Catalyst will harness its product portfolio synergies by leveraging its well-established expertise and proven commercial capabilities. The Company plans to launch the product in Q1 2024, spearheaded by its seasoned and experienced U.S. commercial and medical affairs neuromuscular teams.
AGAMREE was granted Orphan Drug and Rare Pediatric Disease designations status for DMD in the U.S. and will be eligible for seven years of orphan drug exclusivity upon approval date and has issued pending patents that could provide protection until 2040.
About Duchenne Muscular Dystrophy (DMD)
DMD, the most common form of muscular dystrophy, is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality. Current standard treatment for DMD involves corticosteroids, which often come with significant side effects. It is estimated that between 11,000 and 13,000 patients in the U.S. are affected by DMD, with approximately 70% of patients currently receiving concomitant corticosteroid treatment.
About AGAMREE® (vamorolone)
AGAMREE’s unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity and, as such, is considered a novel corticosteroid with dissociative properties in maintaining efficacy, with a better-tolerated side effect profile. This mechanism of action may allow vamorolone to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients 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.
AGAMREE was granted U.S FDA approval on October 26, 2023, and was granted Orphan Drug and Rare Pediatric Disease designations status for DMD in the U.S. and making it eligible for seven years of orphan drug exclusivity upon approval. AGAMREE also has issued and pending patents that could provide protection until 2040. In Europe, it has received Promising Innovative Medicine (PIM) status from the UK MHRA for DMD.
About Catalyst Pathways AGAMREE® Patient Assistance Program
AGAMREE will be available in the U.S. and marketed by Catalyst, supported by its Catalyst Pathway Program®. This comprehensive patient support program includes a dedicated, personalized support team that assists families throughout the AGAMREE treatment journey, from answering questions to coordinating financial assistance programs for eligible patients. For more information, caregivers and healthcare professionals can visit www.yourcatalystpathways.com or call 1-833-422-8259.
