Transkaryotic Therapies, Inc (TKT) has entered into an agreement with Orphan Australia for marketing and distribution of Replagal (agalsidase alfa) for the treatment of Fabry disease in Australia and New Zealand. Under the terms of this agreement, Orphan Australia will have exclusive marketing and distribution rights to Replagal for 15 years.

In Australia, the Pharmaceutical Benefits Advisory Committee has recommended Replagal be considered for funding under the Life Saving Drug Category, which is an important step toward receiving government reimbursement in Australia for Replagal. A final decision on Australian reimbursement approval is expected in the first half of 2004. A decision on reimbursement approval in New Zealand is pending.

"We are preparing to commercialize Replagal in a number of important new markets in 2004. We look forward to working with Orphan Australia and believe this agreement will enable us to best serve patients in Australia and New Zealand," said Michael Astrue, president and CEO of TKT.

"Replagal is an important addition to our portfolio of marketed products, complementing our strategic and therapeutic focus in specialty hospital products," said Alastair Young, managing director of Orphan Australia. "With our company's expertise targeting niche segments of the hospital market, Orphan Australia is well positioned to provide the necessary marketing support to launch Replagal in this territory."

TKT also announced that it would not out-license a Gene-Activated protein by the end of 2003, although it continues to work toward achieving that goal as early as possible in 2004. The company is seeking to partner several of its Gene-Activated protein products, which are versions of proteins that would compete with proteins currently being marketed by third parties.

Replagal is a fully human alpha-galactosidase. A protein, produced by genetic engineering technology, intended for long-term treatment of Fabry disease. It replaces the deficient alpha-galactosidase A with active enzyme to halt or reverse the disease process. Since August 2001, Replagal has been approved for use in 27 countries, including the EU, Australia and New Zealand. In the US, Replagal is an investigational product.

Transkaryotic Therapies, Inc. is a biopharmaceutical company committed to developing treatments for rare diseases caused by protein deficiencies with a core focus on lysosomal storage diseases.

Orphan Australia is a privately owned pharmaceutical company based in Melbourne. It specializes in the development, marketing and distribution of products typically used by specialists to treat serious and/or life threatening disorders.