With many changes at hand in the federal government, the following are some insights on how the U.S. Food and Drug Administration (FDA) is thinking about rare disease regulatory pathways at this time, based on signals from the incoming leaders.
There continues to be an evolution in how the federal government and officials are looking at the challenge of evaluating and regulating the approval of drugs for ultra-rare diseases.
Based on recent a interview with FDA Commissioner Marty Makary and subsequent articles, these are some key take-aways from Cure Sanfilippo Foundation’s perspective:
- The FDA has signaled that it will accept applications for new drugs under the accelerated approval pathway based primarily on the reduction of cerebrospinal fluid levels of heparan sulfate. (Public communications around this have been released by Ultragenyx (Type A gene therapy), Denali (Type A enzyme replacement), and the former Allievex Type B enzyme replacement.
- The new FDA commissioner has suggested they may consider a new pathway that would be more flexible for ultrarare conditions. He gives an example of conditions with 75 affected people in the world as one that would apply to this new pathway.
- There would be many details to work out in regards to any new pathway, however, we feel that this signals FDA’s intent to continue increasing flexibility and recognition of the great unmet need for very small population rare diseases and is an encouraging stance at this time.
- At this time, we do not anticipate that these proposed changes would impact the ongoing FDA review of the Ultragenyx application for accelerated approval (Type A gene therapy). FDA decision on approval is still expected to be provided by mid-August 2025.
As always, if you would like to discuss what is happening, please reach out to Contact@CureSanfilippoFoundation.org to get connected.