BIO Comments on FDA Draft Guidance on Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition Resulting from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies
September 25, 2018
BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition that Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies.
It’s important to support and advance drug development for individuals with rare diseases. BIO suggests the FDA eliminate the phrase “low-prevalence rare disease” from the guidance.
Download Full Comments Below
BIO Comment Letter Slowly Progressive, Low-Prevalence Rare Diseases FDA 2018-D-2456
As the federal government begins to implement IRA, BIO urges the administration to take additional steps to foster the development and deployment of pioneering technologies that will further reduce greenhouse gas emissions in manufacturing,…
BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition that Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies.
It’s important to support and advance drug development for individuals with rare diseases. BIO suggests the FDA eliminate the phrase “low-prevalence rare disease” from the guidance.