Biotech Racking Up Successes Against Ultra-Orphan Diseases

10/15/12Follow @cathyarnst

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significantly reduce their dependence on intravenous feeding, in some cases forgoing it completely. Given the huge advantage teduglutide may offer patients, the FDA is willing to entertain some risk in return for efficacy. In fact, a study  released last year by the National Organization for Rare Disorders determined that the FDA showed flexibility in its review procedures for two out of every three orphan drugs that came before it.

Cambridge, MA-based Aegerion’s oral drug, lomitapide, is also widely expected to get a positive FDA staff and advisory committee review, again because the FDA is inclined to look favorably on drugs for very rare diseases that can clearly save lives. People with Homozygous Familial Hypercholesterolemia have four to ten times the normal levels of LDL cholesterol, often called “bad” cholesterol, and most die of heart disease before age 30. Aegerion reported in January that in a phase III clinical trial enrolling 29 patients, lomitapide reduced average LDL levels by 40 percent during the efficacy phase, a level sustained over the remainder of the 78-week study.  Industry analysts expect that lomitapide will be approved by year end, possibly as soon as November.

Despite the small markets, these ultra-orphan drugs can be very lucrative, due to sky-high prices that can sometimes even turn them into blockbusters. Alexion Pharmaceuticals (NASDAQ: ALXN), in Cheshire, CT, won FDA approval  for its drug eculizumab (Soliris) in 2007 for a rare life-threatening blood disease that afflicts between 4,000 and 6,000 people in the U.S. The drug costs $409,000 and is expected to generate more than $1 billion in sales this year.

Aegerion and NPS should also do well. Industry analysts expect Aegerion to charge around $250,000 a year for its drug, and estimate that peak sales could reach $450 million a year. NPS has said it expects to bring in $350 million in revenues for teduglutide.

Those kinds of successes are attracting new players to the ultra-orphan market. On Friday Intercept Pharmaceuticals  (NASDAQ: ICPT), based in New York, went public, selling 5 million shares for $15 each and raising $75 million in gross proceeds. Intercept’s shares ended their first day up 29.3 percent, at $19.40 per share. The IPO came just two months after Intercept raised $30 million in a Series C funding round. The biotech firm is developing therapeutics for rare forms of chronic liver disease. Its lead product, obeticholic acid, targets primary biliary cirrhosis, and Intercept says about 30,000 people are eligible for the treatment. The drug is currently in a Phase III clinical trial and the results are expected to be available by mid-2014.

According to the National Organization for Rare Disorders, there are nearly 7,000 known orphan diseases, with more discovered every year, but only about 200 have FDA-approved treatments. With that magnitude of unmet need, the search for ultra-orphan disease treatments is certain to continue to drive the biotech industry.

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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?