Progen anti-cancer compound earns orphan drug status

Brisbane-based Progen (ASX:PGL) has earned 'orphan drug' status from the US Food and Drug Administration for its much-touted PI-88 compound, for treatment of malignant melanoma.

The 'orphan drug' designation was set up by the FDA to encourage companies to develop therapies for diseases that affect fewer than 200,000 patients, and which address unmet medical needs. The designation means PI-88 will have seven years' market exclusivity as a malignant melanoma treatment.

Progen business development manager Sarah Meibusch said that while most Australian biotechs opted to take a product to the local Therapeutic Goods Administration, Progen opted to apply to the FDA in 1999.

Meibusch said FDA approval would give the company -- which is listed on the Nasdaq as well as the ASX -- extra leverage with its investors, many of whom are in the US.

One of the advantages of orphan designation, she said, was that it would help to speed the product to the marketplace. "You might look at it and say '200,000 people is not very many'," she said. "But the key is to get approval in one indication first."

PI-88 is one of a new class of multi-targeted cancer therapeutics. Progen is taking it through a Phase II clinical program in metastatic melanoma as a single agent therapy, advanced non-small cell lung cancer in combination with chemotherapy, as adjuvant treatment in post-operative primary liver cancer, and as a single agent therapy in multiple myeloma.

Progen's VP of R&D, Robert Don, said PI-88's strength lay its ability to inhibit both angiogenesis and metastasis simultaneously. "We have seen evidence that this potential might be realised across a variety of cancers," Don said in a statement.

MD Lewis Lee said obtaining orphan status proved Progen was able to navigate through the complexities of the US regulatory system.

The market reacted warmly to the news, sending Progen stocks as high as AUD$3.60 after opening today at $3.05.