In May, the FDA outlined their new toxicology requirements for ultra-rare diseases in their draft guidance for industry, titled “Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment.” The guidance potentially allows for only three months of chronic toxicology animal studies if there are no adverse findings, which is a significant improvement in policy.
The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life-saving treatments to millions of people suffering from rare diseases.
Recommended Unified Screening Platform Roundtable, Webinar, August 26th, 2015
Annual Rare Disease Scientific Workshop, Washington DC, September 15th, 2015
Global Genes Rare Patient Advocacy Summit, Huntington Beach CA, September 24-25th, 2015
EveryLife Foundation Community Congress Meeting, Washington DC, November 4th, 2015
Rare Voice Awards Gala, Washington DC, November 4th, 2015