Exciting breakthroughs in rare disease treatments mean more patients are gaining access to cutting-edge therapies - and specialty pharmacies are playing a crucial role in delivering these medications with expert support. But what exactly do they do, and when do they come into play?
AllMyHealth’s latest guide breaks it all down - exploring how specialty pharmacies work, their expanding role in patient care, and how individuals can engage with them. Plus, we spotlight some specialty pharmacies that cater to SMA.
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A biopharmaceutical company from the Boston area has taken a significant step forward in the treatment of spinal muscular atrophy (SMA) by submitting a biologics license application for apitegromab to the FDA. This application is bolstered by the positive outcomes from two key studies: the phase 3 SAPPHIRE clinical trial and the phase 2 TOPAZ study. Apitegromab, a fully human monoclonal antibody, is designed to work alongside existing survival motor neuron-targeted treatments for SMA.
The company has also sought priority review for their application, which, if granted, could expedite the FDA's evaluation process. This is a hopeful development for individuals with SMA, as apitegromab represents a potential new muscle-targeted therapy option. The application's inclusion of topline results from the clinical trials underscores the progress being made in SMA treatment research (Healio - Neurology, 2025).
