The FDA and NIH have debuted the Critical Path for Rare Neurodegenerative Diseases (CP-RND), a public-private partnership focused on developing new therapies for amyotrophic lateral sclerosis (ALS) and rare neurodegenerative diseases.
One of the goals of the CP-RND is to foster innovation in drug discovery and development as well as clinical testing. In addition, CP-RND will aim to develop a therapy that halts the progression of ALS.
In a similar vein, FDA launched its Action Plan for Rare Neurodegenerative Diseases in June, including amyotrophic lateral sclerosis (ALS).
FDA and NIH have chosen Tucson, Arizona-based Critical Path Institute (C-Path) to help guide the CP-RND initiative.
Founded in 2005 as a public-private partnership, C-Path is an independent, nonprofit organization.
Earlier this year, C-Path established Amsterdam as the head of its European operations.
In its work on the CP-RND project, C-Path will gather experts in rare neurodegenerative diseases from patient communities, advocacy organizations and the private sector.
“The partnership we are announcing today will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases,” said FDA Chief Medical Officer Dr. Hilary Marston. “We look forward to working with NIH, C-Path and other public and private partners to carry out this important effort.”
C-Path will also use an FDA-funded project known as the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP). The RDCA-DAP infrastructure offers a central, standardized infrastructure for drug discovery and development to foster rare disease characterization.
The CP-RND initiative and RDCA-DAP platform will enable the integration of various patient data sources spanning various rare diseases.
Approximately 5,000 people in the U.S. receive an ALS diagnosis each year.
An FDA advisory panel recently recommended that the agency approve the ALS drug candidate AMX0035, which won approval in Canada this summer.
Other companies developing ALS drugs include the privately-held biotech QurAlis with its first-in-class molecule QRL-201 and Irvine, California-based Eledon Pharmaceuticals.