FDA Gives Guidance on Developing Treatments for Fabry Disease
The guidance discusses clinical trial design features that can support approval of treatments of Fabry disease.
FDA published guidance on August 7, 2019 in support of the development of treatments for Fabry disease, a rare lysosomal storage disorder that causes gastrointestinal symptoms, slowly progressive organ damage, and early mortality. The guidance provides information on clinical trial design including patient eligibility criteria and efficacy endpoints.
According to FDA, Fabry disease is “a rare, X-linked, slowly progressive, lysosomal storage disorder caused by pathogenic variants (disease-causing mutations) in the galactosidase alpha (GLA) gene resulting in absent or deficient activity of the lysosomal enzyme α-galactosidase A (α-Gal A). The α-Gal A enzyme breaks down glycosphingolipids within lysosomes. α-Gal A deficient activity leads to progressive intralysosomal accumulation of the undegraded substrate globotriaosylceramide (GL-3, also known as Gb3), a glycosphingolipid.” The disease affects both males and females despite being X-linked; although, males experience the most severe symptoms.
Source: FDA
Regeneron Treatment for Multiple Myeloma Gets Conditional Marketing Approval from EC
April 29th 2025The indication is specific to patients who have received at least three prior therapies, including a proteasome inhibitor, immunomodulatory agent, and anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy.
MHRA Approves GSK Therapy Combinations for Multiple Myeloma
April 21st 2025Belantamab mafodotin is approved in combination with bortezomib plus dexamethasone in patients who have had at least one prior therapy, and in combination with pomalidomide plus dexamethasone for those who have had a prior therapy including lenalidomide.
