Treatment Pipeline

The Friedreich Ataxia Treatment Pipeline is a visual tool for communicating the progress of research and development on lead therapeutic candidates.

On the vertical axis lead candidates are grouped based on the mechanism of action or approach to treatment, e.g. where or how each drug might work in the cell, technological approach, or the problem being addressed.

The horizontal axis indicates the stage of the research or where the therapeutic candidate is in development.

The first two stages, ‘Discovery’ and ‘Pre-Clinical Development’, take place in the research laboratory and represent early discovery and development.
The ‘IND Filed’ stage is an important milestone. This is when there is a regulatory filing with the United States Food and Drug Administration (FDA) to begin human studies.
‘Phase 1’ through ‘Phase 3’ are the stages of clinical trials or studies, when a drug is tested in individuals for safety and efficacy.
With sufficient evidence proving a drug benefit, a new drug application (NDA) can be filed with the FDA. Once an NDA is approved, the drug can then be manufactured for distribution & sale in the United States.

FDA approval only applies for the United States. For a drug or treatment to be approved in Australia, a registration and review process must be undertaken by Australia’s regulatory authority, the Therapeutic Goods Administration (TGA). The TGA assesses the medication for safety, quality and efficacy.

This pipeline focuses on drugs that are already in preclinical development or have advanced to human clinical trials.

Please note that SKYCLARYS is only approved and available in the United States and Europe at this time. fara Australia is in ongoing discussions with Biogen regarding registration in Australia and New Zealand

For further information about the Pipeline click here