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New Ataxia Consortium Created to Accelerate the Development of Treatments for Ataxia

NAF, along with Ataxia Global Initiative, and Ataxia UK, recently partnered with Critical Path Institute (C-Path) for the launch of their Critical Path to Therapeutics for the Ataxias (CPTA) Consortium. CPTA is a public-private partnership focused on accelerating the development of treatments for inherited Ataxias. The work of the Consortium will focus on improving understanding of disease progression and how it may be measured in the context of clinical trials, thus accelerating the development of treatments for people with Ataxia. Key partners in the industry and academic research communities have joined the Consortium, including Biohaven Pharmaceuticals, Ionis Pharmaceuticals, Roche Pharmaceuticals, Servier Group, Triplet Therapeutics, uniQure, and Vico Therapeutics.

CPTA Mission

“CPTA’s mission is to bring together experts from across different fields of Ataxia research, patient organizations, medical product development, and regulators to work together to develop measures and analyses that will improve our understanding of inherited Ataxias and reduce the time necessary to develop, assess, and approve novel therapies, delivering results to patients faster” said Jane Larkindale, D.Phil., Executive Director of the Rare Disease Cures Accelerator – Data and Analytics Platform (RDCA-DAP)

Although the pipeline of new treatments for the Ataxias is expanding, there are still large gaps in our understanding of these diverse disorders that prevent companies from focusing in this space. There is an urgent need to understand better how to measure the effects of potential therapies and determine if a therapy provides meaningful benefit to individuals living with Ataxias. CPTA will create a neutral, precompetitive space where stakeholders from across different fields and backgrounds, including representatives from global medicine regulatory agencies, can come together to share expertise and better understand the natural history, progression and variance in these diseases, as well as how to measure therapeutic effects in clinical trials. These shared insights will help create new solutions for addressing challenges in therapy development for the Ataxias.

Information on the progression of and inherited Ataxias that have been gathered from global natural history studies and clinical trials over the years will be essential to this initiative. Such data will be hosted on C-Path’s database (RDCA-DAP), an FDA-funded initiative that supports data sharing and analysis across rare diseases. By pooling data from many different patients across many different rare diseases, researchers are able to examine similarities within and across these diseases and gain insight that would be impossible from individuals in isolation or small populations. These data will inform Ataxia research, leading to an optimized understanding of these disorders, how disease progression and therapeutic effects are measured and will help accelerate the development of novel medicines in this space.

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