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WASHINGTON (AP) - The first drug treatment for the rare but potentially deadly inherited disease Hunter Syndrome was approved Monday by the Food and Drug Administration.
Shire Human Genetic Therapies of Cambridge, Mass., manufacturer of Elaprase, said it plans to have the drug available in the United States within 30 days.
The FDA approved Elaprase, also known as idursulfase, as an "orphan" drug. Orphan drugs are developed to treat illnesses that affect relatively small numbers of people and the manufacturer is granted a seven-year period of exclusive marketing...

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This is a small study of a very expensive treatment for a very rare disease. We get few details of the disease and even fewer details of the benefits of the treatment other than the patients are able to complete, on average "35-metre greater increase in the distance walked in six minutes compared to the patients on placebo." We have no idea what the placebo patients could have done. And all this for $300,000 per year?

While there is a list of adverse events, including life-threatening reactions associated with the treatment, the key quote goes to the researcher who conducted many of the trials of the product. This isn't good enough for the reader, who would most benefit from someone, outside the study, commenting on the degree to which this treatment is or isn't an advance for these patients.

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