- In December 2023, Amicus Therapeutics announced that its pharmacological chaperone drug Galafold (migalastat) continues to expand its global footprint, with approvals in over 40 countries for treating Fabry disease in patients with amenable GLA variants. The therapy has now been adopted by over 2,400 patients globally. Galafold remains the first and only oral monotherapy approved in multiple markets for Fabry disease
- In January 2024, Teva Pharmaceutical Industries Ltd. entered into a licensing agreement with Amicus Therapeutics to commercialize a generic version of Galafold in the United States. The agreement permits Teva to launch the product no earlier than January 2037, ensuring future market competition while preserving access
- In September 2023, Georgia Tech researchers received a grant from BrightFocus Foundation to develop a novel pharmacological chaperone targeting mutant myocilin protein, a key factor in inherited primary open-angle glaucoma. The aim is to stabilize the protein structure and prevent disease progression, a potential breakthrough in ophthalmic chaperone therapies
- In October 2023, a peer-reviewed study published in Pharmaceuticals (MDPI) showcased the growing potential of non-inhibitory pharmacological chaperones, which stabilize misfolded proteins without inhibiting enzyme function. This class is being explored for diseases including Gaucher, Pompe, and Tay–Sachs
- In November 2023, Ambroxol—a known mucolytic agent—was highlighted in clinical trials for its pharmacological chaperone properties in Gaucher disease type 3, demonstrating ability to enhance glucocerebrosidase function in neuronal cells, a promising approach for neuronopathic forms of the disease
