The Sanofi Genzyme Humanitarian Program has been providing access to treatment for lysosomal storage disorders (LSDs) including Pompe disease for over 30 years, according to a new article published in the Orphanet Journal of Rare Diseases.

The program spans more than 100 countries and has provided treatment to over 3300 patients, some for over 20 years. As of May 2021, over 1000 patients were currently enrolled in the program and 156 patients initiated treatment in 2020 alone.

“The Sanofi Genzyme Humanitarian Program, founded to address the unmet needs of individuals with LSDs who previously had no available treatment options, represents one of the longest-running [charitable access programs] for LSD treatment,” the authors said.


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“Providing sustainable global access to therapy, it has paved the way for other manufacturers of [enzyme replacement therapies] to engage in humanitarian aid and has helped to overcome the world’s fear of ‘new and unknown’ orphan drugs.

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Patients and family members affected by the diseases have also been empowered to become disease advocates and increase awareness through the program. Some examples provided in the article include the creation of the Organization for Rare Diseases India and the Philippine Society for Orphan Disorders by individuals directly affected by these diseases and the Humanitarian Program.

“A key success has been maintaining an open-ended vision to provide treatment for rare diseases in underserved communities, which has resulted in its evolution from a charitable program for a single LSD to one that supports the provision of free treatment for five different LSDs to communities globally,” the authors said.

The Humanitarian Program is a charitable access program for the treatment of LSDs including Gaucher disease, Fabry disease, Pompe disease, and mucopolysaccharidosis type 1 and 2. In addition to Genzyme, the program includes partnerships with nongovernment organizations, local authorities, and medical review boards made up of local and global experts.

Reference

Verma IC, El-Beshlawy A, Tylki-Szymańska A, et al. Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise. Orphanet J Rare Dis. 2022;17(1):87. doi:10.1186/s13023-022-02192-1