Biotech Values

[DewDiligence]

FDA Fast Tracks Pharming’s rhC1 Inhibitor

[This is the drug for which Pharming submitted an MAA to the EMEA last week (#msg-12155565). If approved by the EMEA, it will become the second transgenically-derived drug to be approved anywhere in the world. An FDA Fast Track designation per se is not especially meaningful, but it increases the likelihood of the FDA’s granting a six-month Priority Review as opposed to a standard ten-month review. Please see #msg-6210858 for background on Pharming’s global rhC1 inhibitor program, which supports both the already submitted MAA in Europe and a yet to be submitted BLA in the U.S.]

http://yahoo.reuters.com/news/articlehybrid.aspx?storyID=urn:newsml:reuters.com:20060728:MTFH60887_2...

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Fri Jul 28, 2006 4:00 AM ET
By Nicola Leske

AMSTERDAM, July 28 (Reuters) – Shares in Pharming <PHAR.AS> soared on Friday after the Dutch biotechnology firm said its rabbit-derived lead drug, a potential treatment for [hereditary] angioedema, has received a fast track designation for review by the U.S. Food and Drug Administration.

Pharming produces therapeutic proteins in the milk of genetically modified animals and a fast track status provides an expedited review process for products used for life-threatening diseases with limited treatment options.

The Pharming drug candidate, rhC1INH, is a treatment for hereditary angioedema, which causes painful and potentially life-threatening swelling of the body's soft tissues.

Approximately one in every 30,000 people suffer from the condition.

Shares in the mid-cap firm rose 6.38 percent to 3.59 euros by 0721 GMT on the news.

"With today's announcement Pharming shows that they are on track to deliver their lead product ... to the U.S. market as soon as possible," said Bernd Hilhorst at Dutch brokerage AEK, adding that he expects Pharming to deliver its product to the market ahead of German competitor Jerini. [Jerini is partnered with KOSP on their program in HAE: #msg-10801773, #msg-8387838.] Jerini's main drug hope, icatibant, also targets hereditary angioedema.

Pharming's product is a recombinant human protein expressed in the milk of transgenic rabbits and could be the second medicine from a genetically modified animal to get to market.

Last month the European Medicines Agency (EMEA) decided to recommend approval of GTC Biotherapeutics Inc's <GTCB> goat-derived drug ATryn for people with a rare inherited disease that leads to blood clotting.

ATryn had initially been rejected by European experts but the EMEA finally gave it a green light on June 2, providing a boost for the emerging field of "pharming", in which medicines are produced in animals or plants.

Last week Pharming applied for European approval for rhC1INH, which analysts expect to generate at least 125 million euros ($159.3 million) in annual sales assuming a market share of more than 50 percent.

The drug already has orphan drug status [in both the U.S. and EU (#msg-11574371)], which is granted for products targeting rare diseases and provides a period of exclusivity to approved products and various financial incentives.
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