Biotech Values

[DewDiligence]

Pharming is up sharply in Amsterdam trading
on the FDA’s granting of orphan status for the
company’s lead drug, Rhucin (rhC1 inhibitor).
This news does not affect GTCB directly but,
in general, what’s good for Pharming is good
for GTCB and vice-versa. The fact that another
transgenically-produced drug could be approved
as soon as mid 2007 ought to attract more
attention to both companies.

http://yahoo.reuters.com/news/articlehybrid.aspx?storyID=urn:newsml:reuters.com:20060614:MTFH16840_2...

>>
Pharming shares soar on orphan status for lead drug

Wed Jun 14, 2006 3:50 AM ET

AMSTERDAM, June 14 (Reuters) - Shares in Pharming <PHAR.AS> soared on Wednesday after the Dutch biotechnology firm said it had received orphan drug designation for its lead drug from the U.S. Food and Drug Administration (FDA).

Orphan drug status is granted for products targeting rare life-threatening diseases and provides a period of exclusivity to approved products and various financial incentives.

Pharming's lead drug, rhC1INH, received the designation for two separate indications in organ transplants, Pharming said.

Shares in the mid-cap firm rose 14 percent on the news as the Amsterdam Stock exchange opened and were up 11.26 percent at 3.26 euros by 0744 GMT.

Pharming's drug is a recombinant human protein that is expressed in the milk of transgenic rabbits. The company supplies skimmed rabbit milk to the Diosynth biotech unit of Akzo Nobel, which then purifies and formulates the product.

The drug is designated for use in complications after solid organ transplants as well as for bone marrow and stem cell transplants.

Pharming also has orphan drug designation for rhC1INH to treat hereditary angiodema, which causes painful and potentially life-threatening swelling of the body's soft tissues. One in 30,000 people around the world suffers from the illness.

The Leiden-based company aims to file for regulatory approval for the drug by the end of 2006, which means it could go on sale in the first half of 2007, since it has fast-track status as an orphan product to treat a rare disease.

Some analysts said although the news is positive they had expected news on approval for hereditary angiodema first. "We now expect this (approval) to happen early 2007, 1 to 2 years later than expected," Huub Verschueren at SNS securities said in a note.

But analysts agreed that the orphan drug status will result in new markets for the drug.

"We expect news on marketing deals for rC1INH ... in several geographic regions ... and Pharming to leverage its technology platform to partners in the years to come," said Mark van der Gees at Rabobank Securities, who estimates a fair value of 5 euros for Pharming.
<<