BIO Comments on Draft Guidance on Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)
November 14, 2018
BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs).
BIO says the comprehensive draft guidance effectively covers a range of chemistry, manufacturing, and control (CMC) topics relevant to gene therapy products.
However, BIO notes the draft guidance is quite prescriptive and does not necessarily match modern manufacturing. BIO would like to see a better balance between the information FDA needs to see in order to allow an IND to move forward, and over-reporting of information that may be unnecessary. On the whole, it seems much of the information required by the draft guidance is more appropriate for a BLA filing, an approved product, or postapproval changes to a product rather than an IND. BIO suggests specific changes to avoid misinterpretation and clarify the scope, the stage of development the guidelines apply to, and testing expectations, among other topics.
Download Full Comments Below
2018-12-10 BIO Comments On CMC Information For Human Gene Therapy INDs FINAL
Dear Majority Leader Thune, Minority Leader Schumer, Speaker Johnson, and Minority Leader Jeffries:The Council of State Bioscience Associations (CSBA) is a coalition of independent state and territory based non-profit trade associations, each of…
“Most favored nation is a deeply flawed proposal that would devastate our nation’s small- and mid-size biotech companies – the very companies that are the leading drivers of medical innovation in the United States and the cornerstone of America’s…
BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs).
BIO says the comprehensive draft guidance effectively covers a range of chemistry, manufacturing, and control (CMC) topics relevant to gene therapy products.
However, BIO notes the draft guidance is quite prescriptive and does not necessarily match modern manufacturing. BIO would like to see a better balance between the information FDA needs to see in order to allow an IND to move forward, and over-reporting of information that may be unnecessary. On the whole, it seems much of the information required by the draft guidance is more appropriate for a BLA filing, an approved product, or postapproval changes to a product rather than an IND. BIO suggests specific changes to avoid misinterpretation and clarify the scope, the stage of development the guidelines apply to, and testing expectations, among other topics.