Replies to post #1681 on Insmed Inc (INSM)

[Christiaan F]

New ruling in EU! has been changed since March this year! Positive opinion to speed up the process of approval!

what is more important is that the second filing is for NOONAN syndrome! So they file for another 30,000 patients in EU with no treatment anymore!

Got also a positive opinion form COMP!!

http://www.emea.eu.int/pdfs/human/comp/18025106en.pdf
Mecasermin rinfabate, from Insmed Europe Ltd., for treatment of patients with growth hormone
(GH) gene deletion who have developed neutralizing antibodies to GH (review time: day 30)

From 10k:

Noonan Syndrome. Noonan Syndrome is a congenital disorder characterized by a deficiency in IGF-1, short stature, heart defects and variable dysmorphic features. Growth failure is a consistent feature of Noonan Syndrome and the response to recombinant human growth hormone therapy has been disappointing, particularly in children with an identified gene mutation.

With an incidence of approximately one in 2,000, we estimate that there are approximately 30,000 children in the United States with Noonan Syndrome.

CF.

[coinstarz]

Young said in the CC was about NDA (new drug application) in EU. It was not orphan application. They have been filing orphan since 2003 for diff indications. However, it was the first time they filed for orphan for primary IGFD using iPlex. I think orphan has nothing to do with approval NDA, it just gives 7-yr copy right protection. For ex, INSM filed orphan in U.S. long time ago before submitted NDA to FDA in late 2004.

Increlex got orphan too but it doesn't mean EU will approve it if they read Dr. Underwood's testimony or read through i-box. I might send EU i-hub link...lol