Fighting a Global War on Rare Diseases


Xconomy New York — 

Are we winning the war against rare, or “orphan,” diseases? A casual observer could be forgiven for thinking so, given the drumroll of news. The crescendo came in 2011, when the U.S. Food & Drug Administration (FDA) approved 26 drugs in this category, meaning for illnesses that afflict fewer than 200,000 people. It was the highest number since Congress passed the Orphan Drug Act in 1983, offering incentives to companies that entered the rare-disease field. The same year, the agency granted a record 199 requests from companies seeking an “orphan” designation for their products.

In 2012, there was a stream of orphan-related announcements from Pfizer, GlaxoSmithKline, Sanofi and other industry giants – a break from the past, when orphan diseases were the turf of small companies. Last week, a columnist in this space fretted that the excitement over rare diseases might one day lead to a reduction in research into hard-to-treat ailments such as cancer or Alzheimer’s, which affect many more people.

Yet despite the frenzy – and as the columnist duly noted – we are a long way from winning this war. Around the world, an estimated 60 million people suffer from some 7,000 rare diseases. Fewer than 5 percent of orphan diseases have treatments approved by the FDA.

For all the enthusiasm on the part of Big Pharma, it is small and midsize players that are stewarding most orphan drugs on the market today or in the pipeline. Strapped for cash, most of these companies don’t try to expand beyond their local market. In short, companies on which the world depends to fight this war typically license away the opportunity, and the responsibility, to treat the vast majority of their target patients.

I am intimately familiar with their predicament. Until this week, I was among their ranks.

My company, Bedminster, NJ-based NPS Pharmaceuticals (NASDAQ: NPSP), sells teduglutide (Gattex), a newly-approved medicine for patients with a debilitating rare condition called short bowel syndrome. A second NPS drug in late-stage development helps people who produce too little parathyroid hormone. After years entrusting all global business to a licensed partner, NPS this week retrieved international rights to both drugs. We’re not the first midsize company to take this step. For reasons I will discuss below, I’m hoping many others decide to do the same.

The chief incentive for seizing the reins overseas is also the most obvious: the wide world is where the patients are. Short bowel syndrome has an estimated addressable patient population of 3,000 to 5,000 patients in the U.S., and the illness doesn’t respect geographic boundaries. Europe has a population of about 500 million, so the patient cohort is probably double or triple that of the U.S. We haven’t made plans for Japan, Latin America, China or India, but each beckons with opportunities to do good and to prosper. The most cursory glance at a world map reveals the moral and business imperatives of … Next Page »

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One response to “Fighting a Global War on Rare Diseases”

  1. Carri Levy says:

    As a mother of a child with a rare disease and the producer for the Behnd the Mystery series on Lifetime TV, I am thrilled that these companies are not only stepping up to the plate , but are being innovative. Its enough to learn that your child has a rare disease, but to learn that there is nothing they can give them, nor is there anything being created is shocking for most families. Personally I want to thank all of these companies for coming to the party and playing with the Rare world.