FDA’s CDER Partners with Critical Path Institute to Focus on Lysosomal Diseases

The FDA’s Center for Drug Evaluation and Research (CDER) is partnering with the nonprofit organization Critical Path Institute to explore the feasibility of launching the Lysosomal Diseases Consortium.

The consortium is slated to be a public-private partnership that includes the FDA, several academic institutions, pharmaceutical companies, patient groups and non-government organizations to address unmet drug development needs.

The plans for the new organization include finding ways 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, the FDA said.

Lysosomal storage diseases are inherited metabolic conditions involving enzyme deficiencies that lead to buildup of toxic materials in the body’s cells.