Replies to post #1702 on Insmed Inc (INSM)

[Christiaan F]

You'll find the whole EMEA & COMP process and the advantages it has on this link. http://www.eurordis.org/rubrique.php3?id_rubrique=24
http://www.eurordis.org/article.php3?id_article=440

Orphan medicinal product designation assigns orphan status based on a defined number of criteria. These can be resumed as:

- The product should be intended to be use for an indication concerning no more than 5 in 10 000 persons in the European Community.
- The disease concerned should be life-threatening, seriously debilitating or a serious and chronic condition.
- There should exist no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorized in the European Community; or, if such a method exists, then the medicinal product will confer a significant benefit compared to the existing one.

The request for Orphan Medicinal Product designation can be made at any stage of drug development as soon as sufficient scientific evidence can be presented. The request for Orphan Medicinal Product designation can be made at any stage of drug development as soon as sufficient scientific evidence can be presented. The research may therefore be pre-clinical (not yet tested on human subjects) or may have reached the human clinical trial phase.

INSM received positive opinion from he COMP for Iplex as a treatment for:
- IGFD
- Noonan syndrome (even if phase II trial has not been finished yet)


CF

[rod5247]

Thanks, that had me confused as it appeared both COMP & CHMP are set up the same and go through almost identical process.

"One other point if TRCA is granted Orphan status for severe primary IGF-I deficiency, this does not prevent INSM from seeking co-orphan status based on safety."

Noticed that but since INSM is ahead time wise and gains approval first, TRCA could be denied co-orphan status because IPLEX works as well and safer. If what I think I read is correct, another drug for same indication will not receive COMP approval unless it is shown to be an improvement or safer than an existing drug with orphan status. Since orphan status can receive temporary (quick) MAA under certain conditions and the normal MAA is quicker for orphan anyway, IPLEX could gain a huge to market advantage. Have read so much past two days, it gave me two headaches. I probably understand enough to be totally stupid.