BIO Comments on FDA Draft Guidance on Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition Resulting from Single Enzyme Defects: Providing Evidence of Effectiveness for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BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition that Results from Single Enzyme Defects: Providing Evidence of Effectiveness for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
On behalf of its member organizations, Biotechnology Innovation Organization (“BIO”) respectfully submits this Comment in response to the United States Patent and Trademark Office’s (the “Patent Office” or “PTO”) February 13, 2024 Request for…
BIO submits comments on the MDH’s proposed regulations regarding Drugs of Substantial Public Interest: Draft Methodology for Public Comment as required in statute by the Prescription Drug Price Transparency Act.
BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition that Results from Single Enzyme Defects: Providing Evidence of Effectiveness for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.