Aegerion Pharmaceuticals, Inc. (AEGR), Raptor Pharmaceutical Corp. (RPTP): Two Orphan Drugmakers With Upcoming Catalysts

Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR)Within the next few days, stocks for two orphan drugmakers could either soar or sink. Both Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) and Raptor Pharmaceutical Corp. (NASDAQ:RPTP) have catalysts coming next week. Here’s what could happen.

Successful launch?
For Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR), all eyes will be on how well the launch for Juxtapid has gone. The company announces first quarter results on April 30.

Juxtapid received approval from the U.S. Food and Drug Administration last December. The drug is used to lower cholesterol levels in patients with homozygous familial hypercholesterolemia, or HoFH. This disease is a rare and severe lipid disorder that affects around one in a million births.

Earlier this year, Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) laid out its plans to commercialize Juxtapid. The company said that it would focus on identifying patients who are potential candidates for the drug and educating health care providers. Aegerion CEO Marc Beer stated that he expected to “end 2013 with approximately 250-300 patients on therapy on a global basis.”

Aegerion faces competition, though. The FDA approved another HoFH drug, Kynamro, in January. The drug was developed by ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) and is marketed by Sanofi SA (ADR) (NYSE:SNY) .

Kynamro costs $176,000 per year per patient. That’s expensive, but not as much as Juxtapid, which costs $235,000 to $295,000 per year.

Investors will be anxious to find out if the higher pricing hurt Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR)’s efforts. That might not be the case if the company’s sales team was able to effectively position Juxtapid as the safer alternative. While both drugs can have severe side effects, the FDA advisory committee appeared to view Juxtapid more favorably than Kynamro. The committee voted 13-to-2 for Juxtapid and 9-to-6 for Kynamro.

Yea or nay?
April 30 is an important date for Raptor Pharmaceutical Corp. (NASDAQ:RPTP) also. The FDA is scheduled to announced its decision on the company’s New Drug Application for Procysbi.

The drug targets the treatment of nephropathic cystinosis, a rare and sometimes fatal metabolic disorder. Cystinosis is currently treated most often with Cystagon, which is marketed by Mylan Inc. (NASDAQ:MYL).

Procysbi and Cystagon are two different forms of cysteamine bitartrate. Cystagon is released immediately and must be taken on a strict schedule every six hours — including during the middle of the night. Procysbi is a delayed release form of cysteamine bitartrate that can be taken twice daily.

Clinical studies found that Procysbi was statistically as effective as Cystagon. While there were some adverse effects, Raptor Pharmaceutical Corp. (NASDAQ:RPTP) reported that no serious unexpected issues emerged that were attributable to Procysbi.

Although the FDA pushed back its decision on Procysbi from January to April, that shouldn’t raise concerns about the drug’s chances. Delays of three months aren’t unusual.

Analysts see annual sales for Procysbi of around $150 million. Considering that Raptor’s current market cap is less than $360 million on no revenue, a positive decision by the FDA should make the stock take off.

Predictions
We still have to wait a few days to find out what will happen, so whether Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) and Raptor Pharmaceutical Corp. (NASDAQ:RPTP) shares sink or soar is anyone’s guess at this point. It’s a tough call on how well Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR)’s efforts on Juxtapid fared during the first quarter. I think things could have gone either way.

While anything can happen with FDA decisions, I expect Raptor Pharmaceutical Corp. (NASDAQ:RPTP) will receive good news by next week. A positive FDA announcement would mean even better news to the families with loved ones suffering from nephropathic cystinosis.

The article 2 Orphan Drugmakers With Upcoming Catalysts originally appeared on Fool.com and is written by Keith Speights.

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

Copyright © 1995 – 2013 The Motley Fool, LLC. All rights reserved. The Motley Fool has a disclosure policy.

blog comments powered by Disqus
Insider Monkey Headlines
Insider Monkey Small Cap Strategy
Insider Monkey Small Cap Strategy

Insider Monkey beat the market by 52 percentage points in 24 months Click to see monthly returns in table format!

Lists

10 Cities with High Demand for Nurses

6 of the Worst Greeting Card Messages Ever Crafted

6 Ways to Make Money in ArcheAge and Build Your Empire

10 Foods To Eat To Lower Cholesterol Levels

The 10 Most Hated Television Characters of All Time

The 30 Worst Halloween Costume Ideas Ever Brought to Horrible Life

10 Vocational Skills in Demand Today with Jobs Waiting to be Filled

10 Best Places to Visit in Central and South America

The 10 Greatest Empires in History Which Nearly Conquered the World

The 6 Cheapest Boarding Schools In America 2015

5 Clear Reasons LoL is Better than DotA, Continues to Rule MOBAs

The Only 9 Teams with a Chance to Win the Super Bowl

The 15 Most Common Phobias in America that Induce Fits of Panic

Top 6 Least Expensive Tourist Destinations in 2014

Jim Goetz, Peter Fenton, Jim Breyer: Top 6 Venture Investors for 2014

Top 15 Billionaires in 2014

5 Pitfalls To Avoid When Buying a Franchise

Top 20 Medical Schools in the US – 2014 Rankings

4 Business Strategies that Turned Jamie Oliver into the World’s Richest Chef

6 Qualities That Make You A Good Team Player

10 High Paying Seasonal Jobs in America this Holiday Season

The 10 Busiest Shipping Lanes in the World

5 Most Valuable Brands in China

The 10 States with Highest Substance Abuse Rates Crippling Their Populace

The Top 10 Things to Do Before You Die That Will Echo for Eternity

The 10 Best Selling Items on Etsy

Top 10 Things to Do in Tokyo, the Greatest City in the World

10 Mistakes on Social Media that Can Harm You and Will Probably Get You Canned

The 10 Best Cities to Find Jobs in 2014

The 10 Most Controversial Songs Of All Time to Hit (and get Banned from) the Airwaves

The 20 Biggest IPOs in US History

The 10 Best Places to Visit in Mexico that Are Beautiful and Safe

7 Bad Habits that Age You Beyond Your Years

The 40 Best Fortune Cookie Sayings That Will Leave You Bemused, Befuddled, or Beguiled

10 Foods to Eat Before a Workout to Make Every Drop of Sweat Count

The 5 Best Documentaries On Netflix You Must See

The Most Heartwarming and Inspirational Story Of This Halloween Season, It Will Make You Cry and Jump For Joy

10 Best Party Songs of All Time to Bring the House Down With

5 New World Order Conspiracy Theories that Will Strangle the World

The 10 Highest Rated Movies of 2014

The 10 Largest Container Shipping Companies in the World

The 10 Largest Armies in the World: Who Should We Be Afraid Of?

Best Warren Buffett Quotes on Money You Need to Hear

The 10 Highest Suicide Rates by Profession

The 20 Most Underrated Movies of All Time

The 10 Fastest Growing Companies in America

The 10 Biggest Outlet Malls in USA

The 5 Most Popular Rap Songs of All Time

The 10 Countries that Eat the Most Meat

The10 Most Expensive Countries to Fly To

Subscribe

Enter your email:

Delivered by FeedBurner

X

Thanks! An email with instructions is sent to !

Your email already exists in our database. Click here to go to your subscriptions

Insider Monkey returned 47.6% in its first year! Wondering How?

Download a complete edition of our newsletter for free!