Aegerion Pharmaceuticals, Inc. (AEGR), NPS Pharmaceuticals, Inc. (NPSP): Three of the World’s Rarest Diseases (and the Uncommon Profits They Bring)

2. Paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria, or PNH, is a blood disease that frequently leads to life-threatening blood clots. Symptoms of PNH include red urine and anemia. Patients can require blood transfusions. Only one to two people in a million suffer from the disease.

The FDA has approved only one drug for treating PNH — Soliris from Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN). Soliris is also used in the treatment of another rare blood disease, atypical hemolytic uremic syndrome, or aHUS, which affects around one in 500,000 Americans each year.

Soliris costs $440,000 per year, making it the world’s most expensive drug. Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) made more than $1.1 billion from sales of the drug last year, which was 45% higher than the year before. The pharmaceutical company’s profit amounts to nearly 24% of those sales dollars — and those profits continue to grow.

3. Homozygous familial hypercholesterolemia
Homozygous familial hypercholesterolemia, or HoFH, is a rare genetic disorder that results in very high bad cholesterol levels, which can lead to serious heart diseases. HoFH can sometimes require removal of cholesterol in a manner similar to dialysis and occasionally even liver transplants. The disease occurs in around one in a million people.

Juxtapid costs up to $295,000 per year, while Kynamro’s price tag is $176,000 per year. Analysts project that Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) could ultimately see annual sales ranging from $300 million to $450 million for Juxtapid. That lines up with the $400 million peak sales estimated for Kynamro by Natixis Securities. These sales figures sound pretty good, but remember they’re only projections at this point for peak sales down the road.

HoFH is a rare disease in another sense in that it has two drugs on the market. This can be great news for patients. Because it also splits the potential profits among multiple players, though, it isn’t as great for Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR), ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS), and Sanofi SA (ADR) (NYSE:SNY).

A long way to go
Much progress has been made in treating the world’s rarest diseases, thanks largely to governments that have established orphan drug programs. However, there remains a long way to go. Well over 6,000 rare diseases remain for which there are no available treatments. For the rarest of the rare diseases, there still isn’t enough hope.

The article 3 of the World’s Rarest Diseases (and the Uncommon Profits They Bring) originally appeared on Fool.com is written by Keith Speights.

Fool contributor Keith Speights and The Motley Fool have no position in any of the stocks mentioned.

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