Profit from Orphan Drugs: 3 Choices (Revised) - Analyst Blog

Shutterstock photo

Once given the moniker of orphans, drugs for rare diseases were originally considered to be an unviable proposition for pharma companies. This was because the number of patients for treatments was so small that their development and production were considered to be an unviable proposition. Today, they have emerged as major money spinners for the pharma sector.

What are Orphan Drugs?

Rare medical conditions were originally called orphan diseases simply because no one was providing treatments for them. The need for drugs targeted at rare conditions led to Congress creating the Orphan Drug Care Act of 1983. This offered companies a seven year monopoly on medicines for rare diseases.

This offers the same protection to companies that a patent does, without having to clear the rigorous procedure which a patent has to go through. Again, orphan drug status can be given to drugs which are already being used in foreign markets or in the U.S. itself for other diseases.

Money Spinners

Such a development offered hope to patients with conditions as rare as hereditary angioedema. This condition affects only around six thousand people in the U.S. But these small numbers ultimately emerged as an advantage.

This is because pharma companies found they could make considerable profits from a small segment with relatively limited options. Treating patients with orphan drugs could cost anything between $200,000 to $600,000, leading to companies raking in billions.

Costs and Competition

The high cost of treatment may emerge as a roadblock in the long run. As attempts to reduce expenditure rise, pharma companies may be find costs unsustainable. However, competition between drug makers is bringing down prices.

For example, Aegerion Pharmaceuticals, Inc. ( AEGR ) produces Juxtapid, used to treat a life threatening inherited cholesterol disorder, which costs patients between $235,000 to $295,000 a year. Sanofi ( SNY ) has recently received approval for a cheaper alternative, Kynamro. However, even this new medication will cost around $176,000 a year.

A Bright Future

Despite the obvious opportunities, not all aspiring orphan drug makers succeed because of the high costs and risks involved. However, the future looks bright according to a report released by research firm TechNavio. The orphan drugs sector is projected to grow globally at a CAGR of 5.67% from 2013 to 2018. This is primarily because of a reduction in the time required for drug development.

Below we present three stocks, each of which is involved with the orphan drugs sector and also have a good Zacks rank. Incidentally, they also find mention in the TechNavio report.

Shire plc

Shire plc ( SHPG ) produces specialty biopharmaceutical treatments. It concentrates on medications for gastrointestinal conditions, attention deficit and hyperactivity disorder, regenerative medicine and human genetic therapies. It is currently developing a treatment for Sanfilippo syndrome, a rare metabolic condition.

Shire plc holds a Zacks Rank #1 (Strong Buy) and has expected earnings growth of 19.50%. The forward price-to-earnings Ratios (P/E) for the current financial year (F1) is 17.41.

Amgen Inc.

Amgen Inc. ( AMGN ) is one of the world's largest biotechnology companies and much of its success is attributable to orphan drugs. It produces medications for kidney conditions, cancer, rheumatoid arthritis and bone diseases. It is a pioneer in the biotechnology arena and is involved in drug discovery, development and manufacturing.

Currently the company holds a Zacks Rank #2 (Buy) and has expected earnings growth of 7%. It has a P/E (F1) of 15.31.

Biogen Idec Inc.

Our third choice is Biogen Idec Inc. ( BIIB ). The company developed its drug Avonex when multiple sclerosis was still recognized as an orphan disease.It focuses on medication for autoimmune disorders, neurodegenerative diseases and hemophilia. Biogen is a Fortune 500 company with annual revenues of $5 billion.

Besides a Zacks Rank #1 (Strong Buy), Biogen Idec Inc. has expected earnings growth of 31.70%. It has a P/E (F1) of 28.96.

Drugs for rare conditions will continue to play a key role in the pharma sector in the future. Adding these excellent choices will go a long way in enhancing the value of your portfolio.

(We are reissuing this article to correct a mistake. The original article, issued Wednesday, March 5, 2014, should no longer be relied upon.)

AEGERION PHARMA (AEGR): Free Stock Analysis Report

AMGEN INC (AMGN): Free Stock Analysis Report

BIOGEN IDEC INC (BIIB): Free Stock Analysis Report

SHIRE PLC-ADR (SHPG): Free Stock Analysis Report

SANOFI-AVENTIS (SNY): Free Stock Analysis Report

To read this article on click here.

Zacks Investment Research

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

This article appears in: Investing , Business , Stocks
Referenced Symbols: AEGR , AMGN , BIIB , SHPG , SNY

More from


Equity Research
Follow on:

Research Brokers before you trade

Want to trade FX?

Find a Credit Card

Select a credit card product by:
Select an offer:
Data Provided by