Aegerion Pharmaceuticals, Inc. (AEGR), ISIS Pharmaceuticals, Inc. (ISIS), BioMarin Pharmaceutical Inc. (BMRN): Orphan Drug Makers – Robust Investment Opportunities

The US pharmaceutical industry has been revolutionized by the emerging biotech companies which have discovered new ways to cure even the rarest of diseases. So the concept of an orphan drug is no more a mystery for the people who invest into pharmaceutical companies. According to the findings of Thomson Reuters, at the end of 2011 orphan drug sales attributed to 6% of the total pharmaceutical sales. Along with that the CAGR (compound annual growth rate) of orphan drugs from 2001 to 2010 was 25.8% compared to the 20.1% of the non-orphan drugs. Keeping that in mind and incorporating the increasing amount of FDA approvals being granted to orphan drugs, I think that the growth of orphan drugs will be way more than non-orphan drugs in the next 30 years. Thus I consider the following orphan drug makers as huge growth potential companies.

Aegerion takes a big leap

News came out this week that Needham increased the target price of Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) stock to a $100 per share. This was very much expected because of the recent success bagged by the company. In late 2012, the company’s first orphan drug i.e. Juxtapid received its FDA approval for the treatment of HoFH. Analysts believe that this will escalate the sales of the company by $15 to $25 million till the end of 2013. In 2014 sales are expected to spike up to $144 million, which ensures a positive outlook for the company’s future. Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) is determined to reach out for over 200 to 300 patients of HoFH this year which should ignite revenue growth. But the long-term target is to get hold of over 4000 different patients of HoFH so that Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) can operate to its full potential.

The therapeutic focus of Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) is Homozygous Familial Hypercholesterolemia (HoFH). It is a big name and if it seems scary to you then it is rightly so. This is because HoFH is a disease in which the cholesterol level in the blood increases due to a rare genetic disorder inherited from both parents. It can go as bad as turning your blood from red to dark shade of yellow. But it is a rare disorder and Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR)’s orphan drug Juxtapid has been granted a seven years exclusivity by FDA for the treatment of this ailment.

The patent portfolio of Juxtapid consists of 5 separate patents issued in US. Similarly it also has patents issued in EU, Canada and Australia. The patent which covers the composition of the matter of Juxtapid is scheduled to expire in 2015 but the company has already filled an application for an extension in the patent duration. Likewise the other patents would also ensure that Juxtapid is the only drug for the treatment of HoFH.