Biotech Racking Up Successes Against Ultra-Orphan Diseases

10/15/12Follow @cathyarnst

There are two U.S. Food & Drug Administration hearings coming up this week that highlight the reasons why the biotech industry is increasingly focused on developing drugs for very rare diseases.

Tomorrow, an advisory committee of independent experts will hear evidence about NPS Pharmaceutical’s (NASDAQ: NPSP) experimental drug for short bowel syndrome, a debilitating condition that prevents patients from absorbing food, and affects only about 10,000 patients in the U.S. The following day an advisory committee will convene for Aegerion Pharmaceuticals’ (NASDAQ: AEGR) treatment for Homozygous Familial Hypercholesterolemia, an even rarer inherited disease that causes very high cholesterol levels, leading to almost certain death from heart disease. The condition afflicts only one person out of a million, amounting to about 6,000 people in the U.S. and Europe.

Both of these drugs are part of a growing trend by biotechs to develop treatments for conditions affecting only a few thousand, or even a few hundred, patients—often called “ultra-orphan” diseases. Although their markets are beyond tiny, these drugs are usually placed on a fast track toward consideration by the FDA, which requires only a few hundred patients for a clinical trial, making their development financially feasible for a startup biotech. And they are getting approved. The success of ultra-orphan drugs before the FDA stands in stark contrast to medications for much more common diseases, such as obesity or diabetes, which have had one failure after another.

The Orphan Drug Act of 1983 granted developers of drugs for diseases that afflict less than 200,000 people special tax considerations and an extra seven years of protection from competition, making these rare diseases a feasible market opportunity. Some 380 orphan drugs have been approved by the FDA since 1983, compared with 38 prior to the act.

A market analysis issued by Thomson Reuters in August, “The Economic Power of Orphan Drugs,”  found that many orphan drugs are as economically viable as medicines for far more common diseases. According to Thomson Reuters there are some 25 million people in the U.S. alone suffering from rare diseases, and the worldwide value of the orphan drug market will be slightly more than $50 billion at the end of this year, about six percent of total pharmaceutical sales.

NPS, based in Bedminster, NJ, already got some good news on Friday when the FDA posted its briefing document indicating that FDA staffers think the company’s drug, teduglutide (Gattex), is effective and its safety issues manageable, even though three patients in a clinical trial developed cancer. The drug helps rehabilitates the intestinal lining, allowing patients to absorb nutrients when their bowels have been damaged by surgery or disease. Currently such patients must receive nutrition intravenously 10-12 hours a day; those treated with the drug were able to … Next Page »

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  • Bill Ghormley

    Powerful history — yet the big questions hang over us — is $409,000 a fair price to pay? Who will pay this?