Regulatory Open Forum

This message was posted by a user wishing to remain anonymous

Dear RA Community:

For an NCE compound in the process of filing an Orphan Drug Application that does not presently have an INN, would it be possible to only provide the company compound number, e.g. ABC-414, in-lieu of the chemical name, or is it required to disclose the chemical name in the ODD application? This is for patent reasons. 

Thank you.

Dear Anonymous,

It is hard for me to see how you could get orphan designation without identifying the chemical for which you intend to obtain the designation, based on the wording of the regulation: 21 CFR 316.20. Item 4 of the required elements for a designation application states: "A description of the drug, to include the identity of the active moiety if it is a drug composed of small molecules, or of the principal molecular structural features if it is composed of macromolecules; its physical and chemical properties, if these characteristics can be determined ...."

I would need to know more about the patent concern to understand why that is the reason, since most sponsors would file all necessary patents, matter and use, well before applying for orphan designation.

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Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States
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Original Message:
Sent: 29-Sep-2022 20:11
From: Anonymous Member
Subject: Orphan Drug Application - Chemical Name Required?

This message was posted by a user wishing to remain anonymous

Dear RA Community:

For an NCE compound in the process of filing an Orphan Drug Application that does not presently have an INN, would it be possible to only provide the company compound number, e.g. ABC-414, in-lieu of the chemical name, or is it required to disclose the chemical name in the ODD application? This is for patent reasons.

Thank you.

This message was posted by a user wishing to remain anonymous

Thanks Glen. You are correct, the sponsor would need to file the necessary patents, and be comfortable with the publishing of the chemical name in FDA's ODD database. It is not an option to not properly identify the drug, per the regulation. Thank you.
Original Message:
Sent: 03-Oct-2022 08:52
From: Glen Park
Subject: Orphan Drug Application - Chemical Name Required?

Dear Anonymous,

It is hard for me to see how you could get orphan designation without identifying the chemical for which you intend to obtain the designation, based on the wording of the regulation: 21 CFR 316.20. Item 4 of the required elements for a designation application states: "A description of the drug, to include the identity of the active moiety if it is a drug composed of small molecules, or of the principal molecular structural features if it is composed of macromolecules; its physical and chemical properties, if these characteristics can be determined ...."

I would need to know more about the patent concern to understand why that is the reason, since most sponsors would file all necessary patents, matter and use, well before applying for orphan designation.

------------------------------
Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
New York NY
United States

Original Message:
Sent: 29-Sep-2022 20:11
From: Anonymous Member
Subject: Orphan Drug Application - Chemical Name Required?

This message was posted by a user wishing to remain anonymous

Dear RA Community:

For an NCE compound in the process of filing an Orphan Drug Application that does not presently have an INN, would it be possible to only provide the company compound number, e.g. ABC-414, in-lieu of the chemical name, or is it required to disclose the chemical name in the ODD application? This is for patent reasons.

Thank you.