FDA initiates the Lysosomal Diseases Pre-Consortium at the Critical Path Institute

The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) has announced its partnership with the  Critical Path Institute (C-Path) to explore the feasibility of launching the Lysosomal Diseases Consortium, a public-private partnership among FDA, leading academic institutions, pharmaceutical industries, patient groups, and non-government organizations to address unmet drug development needs for patients living with lysosomal diseases – inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies.

C-Path is an independent, non-profit, public-private partnership that aims to accelerate the pace and reduce the costs of medical product development through the creation of new data standards, measurement standards, and methods standards that aid in the scientific evaluation of the effectiveness and safety of new therapies.

The proposed Lysosomal Diseases Consortium will be under the umbrella of the Accelerating Rare disease Cures (ARC) Program and will focus on generating actionable solutions to accelerate drug development for lysosomal diseases, and to ensure that those solutions reflect current scientific knowledge of lysosomal diseases and regulatory standards for implementation of drug development.