Do You Have Any Of These Rare Disease Biotech Stocks In Your Portfolio?

(RTTNews) - Rare commodities are worth more than good is a Chinese adage. And more so when it is in the sought-after drug space.

Rare and ultra-rare diseases, also called orphan and ultra-orphan diseases, as the names imply, affect very small numbers of patients. Estimates by the National Human Genome research Institute pitch the number of rare diseases at over 6,800 and the affected population between 25 million and 30 million. Childhood cancers, cystic fibrosis, Huntington's disease, muscular dystrophy, cystinosis and retinitis pigmentosa are some examples of rare diseases.

By U.S. standards, a rare disease affects 650 patients per one million of the total population and ultra-rare disease 20 per million. So why the clamor?

Given the high development costs relative to the market opportunity, pharma companies charge staggering amounts for the treatment options for these rare diseases in order to break-even and make profit on the investment.

Glybera, the first-ever gene therapy approved in Europe, and indicated for the treatment of adult patients diagnosed with familial lipoprotein lipase deficiency, with a price tag of $1.4 million per treatment, is the world's most expensive drug. UniQure, the developer of Glybera, however has no plans to seek approval for this gene therapy in the U.S.

In the U.S., the tag of the "Most Expensive Drug" is held by Soliris, which is indicated for the treatment of paroxysmal nocturnal hemoglobinuria, a rare and serious blood disease wherein the red blood cells break down sooner than they should. Soliris, developed by Alexion Pharmaceuticals, is priced at $525,000 for a year's course of treatment.

Here is a glimpse of some biotech companies developing innovative therapies for rare diseases and their current pipeline status.