Orphan Status

Osiris sells stem cell business to NuVasive

Fri, 09 May 2008 06:59:57 -0400

Medical device maker NuVasive is buying Osteocel, Osiris' biologics business, for $35 million up front and an additional $50 million in milestone payments. The Osteocel business includes an adult stem cell bone graft product containing beneficial properties similar to autograft, as well as a processing facility with significant supply stream capacity. Osiris said the move will allow it to focus on launching its core products. "This transaction provides Osiris with a substantial amount of non-dilutive capital over the near-term, and shapes the organization in a way that is optimal for both the Osteocel employees and the long term mission of Osiris," said Osiris' CEO Randal Mills.

- check out the NuVasive release

Pharming shares sink after Rhucin gets thumbs down

Thu, 20 Mar 2008 07:59:57 -0400

There's new trouble for Pharming's Rhucin, an experimental treatment for hereditary angioedema. Pharming shares plunged after the company announced that the EMEA's Committee for Medicinal Products for Human Use is expected to refuse to approve Rhucin, saying that there is insufficient evidence of clinical benefits after repeated use and a possibility of immune system reactions. Pharming's shares were down about 27 percent after initially dropping by almost half.

Rhucin has been a major headache for Pharming since the EMEA first denied approval last December, sending its stock into a downward spiral. The drug is manufactured in the milk of transgenic animals and has long been held up as a leading example of the potential of transgenic therapies. But Pharming isn't giving up. "European denial for the drug is not the end for Rhucin nor for Pharming," Chief Commercial Officer Rein Strijker told reporters. The company insists that additional data will convincingly make their case for Rhucin and win eventual approval.

- see Pharming's release
- check out the ABN story

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Regulators focusing on transgenic animals. Report
Pharming gets orphan status for transgenic therapy. Report

ChemGenex restructures, focuses on cancer drugs

Mon, 29 Oct 2007 07:59:54 -0400

ChemGenex Pharmaceuticals, which recently reported positive data on the experimental leukemia therapy Ceflatonin, says it is restructuring the company so it can focus on its cancer therapies. The company's wholly-owned Autogen Research, which holds its metabolic disease assets, is being renamed Verva Pharmaceuticals. Verva will be demerged from ChemGenex and merged with Adipogen Pharmaceuticals in a stock deal. Following a capital raising, Verva Pharmaceuticals will seek a listing on the ASX in early 2008.

- check out this release for more

Related Articles:
ChemGenex gains orphan status for cancer drug. Report
ChemGenex gains fast track. Report

Pharming raises $99M

Fri, 19 Oct 2007 06:59:55 -0400

Netherlands-based Pharming has raised $99 million (€70 million) through the issue of convertible bonds. The issue was managed by UBS Investment Bank. The company is also finalizing discussions for restructuring its financing agreement with Paul Capital Healthcare which Pharming entered into in February 2006. Pharming will repay Paul Capital EUR $13 million (€9 million) by the end of this year, with an additional $15 million (€11 million) to be paid in the first quarter of 2008.

Pharming is on the verge of a major European approval for Rhucin, a transgenic therapy made from rabbit's milk that's designed to treat hereditary angioedema. An announcement is expected sometime in this quarter.

- see this release

Related Articles:
Pharming says it's closing in on Rhucin approval. Report
Pharming inks creative drug partnering pact. Report
Pharming wins fast track review, shares jump. Report
Pharming gets orphan status for transgenic therapy. Report

Aspreva, Roche put CellCept on ice

Mon, 10 Sep 2007 06:59:56 -0400

Shares of Aspreva Pharmaceuticals slipped a bit this morning after the company and Roche announced that they would not be pushing ahead with a regulatory filing for CellCept, a new therapy for lupus nephritis. The therapy missed its late-stage primary endpoint--it was unable to demonstrate superiority to intravenous cyclophosphamide. There have been no new therapies approved for lupus in the U.S. in more than 40 years.

- check out the announcement

Related Articles:
Aspreva's CellCept fails late-stage superiority study. Report
Aspreva Pharmaceuticals drug fails Phase III trial. Report
CellCept wins orphan status. Report

Pharming says it's closing in on Rhucin approval

Mon, 20 Aug 2007 06:59:57 -0400

Pharming says that the positive interim results it has received from a study of Rhucin is blazing the way to EMEA approval. The Amsterdam-based developer says it expects EMEA approval in the fourth quarter and should be able to launch the drug, which treats hereditary angioedema, in early '08. The company says it is also just weeks away from completing a U.S. study, and should be able to submit data from that trial for FDA approval. Pharming expects the therapy should garner up to €600 million a year in both the U.S. and Europe. The drug developer's shares jumped about 25 percent on the news.

- see Pharming's release
- and here's the report from ABC Money

Related Articles:
Pharming inks creative drug partnering pact. Report
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Pharming gets orphan status for transgenic therapy. Report

Chelsea wins European orphan status

Wed, 15 Aug 2007 06:59:55 -0400

European drug regulators granted orphan drug status to Chelsea Therapeutics' remedy for hypotension, Droxidopa, and recommended the same for Progen's experimental liver drug, known as PI-88. Designed to promote drugs that treat deadly and rare diseases, the E.U.'s orphan drug program offers marketing exclusivity for up to 10 years.

Chelsea's Droxidopa drug treats a particular sort of low blood pressure stemming from Parkinson's or from multiple-system atrophy. Its European orphan status covers both disorders and lasts for 10 years. It already has orphan-drug status in the U.S.

PI-88 treats primary liver cancer; its orphan designation won't be official until the European Commission officially adopts it, probably by the end of the third quarter.

- see the release
- read the report from the Charlotte Business Journal

Related Article:
FDA grants Droxidopa orphan drug status. Report

Regulators focusing on transgenic animals

Mon, 30 Jul 2007 06:59:55 -0400

Genetically engineered animals are producing a range of therapies for human use, and The New York Times catches up on the action at companies like GTC Biotherapeutics in Massachusetts, Pharmathene and Pharming. Developing drugs in this field has been a slow and laborious task, and the FDA is now developing new guidelines to help govern how animals are used to produce therapies. Questions are also being asked about the ultimate fate of these animals. Should they be sold for food?

- read the article from The New York Times

Related Articles:
Transgenic chickens used to manufacture proteins. Report
GTC inks new transgenics collaboration deal. Report
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Pharming gets orphan status for transgenic therapy. Report