Menu
Amazon Kindle Voyage Rethinks the Reading Experience

Amazon Kindle Voyage Rethinks the Reading Experience

Mat Franco won America's Got Talent

Mat Franco won America's Got Talent

SpaceX and Boeing build manned NASA Spaceships

SpaceX and Boeing build manned NASA Spaceships

Matt Damon is Jason Bourne Again

Matt Damon is Jason Bourne Again

iPhone 6 Reviews are Glowing

iPhone 6 Reviews are Glowing

Did One Of The Top 10 Upside Surprises In The History Of The Biotech Sector Just Happen?

Dec 22 2013, 8:41pm CST | by , in News

 
 

On Friday, the Food and Drug Administration approved a pill called Orenitram, to treat pulmonary arterial hypertension. It is an oral version of an injected drug called Remodulin, which treats the same disease. According to Mark Schoenebaum at ISI Group, the approval is “an enormous surprise — arguably, one of the top 10 biggest upside surprises in the history of the biotech sector.”

Why? Well, for one thing, Orenitram’s benefit isn’t that compelling. The FDA-approved labeling for the product characterizes the treatment effect as “small.” And the FDA had already rejected it twice, in March and October 2012. It seemed nearly impossible that the medicine would be approved  without additional data. Yet that’s exactly what has happened.

This is obviously a win for United Therapeutics, the drugs maker, and for Martine Rothblatt, the company’s chief executive and founder. It’s not clear how big a drug this will be. It could cannibalize sales from United’s other drugs, Remodulin and an inhaled version of Remodulin called Tyvaso. Those betting against the stock are likely to hope that this is its peak, Schoenebaum writes, and that sales will disappoint. The biggest threat against them is an experimental drug from rival Actelion whose clinical trials will read out later this year. Schoenebaum expects the drug to be priced at $150,000 per year, similar to Tyvaso, and says that every $100 million in sales will amount to about a dollar of earnings per share — about a 10% increase.

It’s another stunning moment in the story of United Therapeutics and Rothblatt. In fact, I’m writing this post mainly so I can link to that tale again.

In 1990, 6-year-old Jenesis Rothblatt’s lips turned blue. The little girl gasped for breath, could not climb stairs and was soon in the intensive care unit of Children’s National Hospital in Washington, D.C.

The disaster began a transformation for Jeni’s father–and a change in the way drugs for rare diseases are developed. Martin Rothblatt, a lawyer and businessman, turned his childhood passion for outer space into founding a series of companies that made him millions of dollars. First came GeoStar, a GPS-based navigation system, in 1986, then an early satellite radio company called Worldspace and finally Sirius Satellite Radio in 1990. In 1994 he underwent gender reassignment surgery and changed his name to Martine. Now a she, 55, Martine remains married to Jeni’s mom.

When Jeni became sick, Martine felt useless. “I was an expert in satellites, and I didn’t know anything about medicine,” she says. Jeni had pulmonary arterial hypertension (PAH), a rare and fatal disease that is caused when the artery between the heart and lungs is damaged. With 200,000 cases worldwide, PAH was too uncommon to win drug-development money. Martine Rothblatt sold her telecom stock and started a $3 million foundation to fund research, but three years went by without any progress.

In the end, this story led to a successful biotechnology company, and an entrant in the new world of drugs with six-figure price tags. It’s just one of those stories nobody could ever make up — at least, not if they expected to be believed.

Source: Forbes

You Might Also Like

Updates

Shopping Deals

 
 
 

<a href="/latest_stories/all/all/31" rel="author">Forbes</a>
Forbes is among the most trusted resources for the world's business and investment leaders, providing them the uncompromising commentary, concise analysis, relevant tools and real-time reporting they need to succeed at work, profit from investing and have fun with the rewards of winning.

 

 

Comments

blog comments powered by Disqus

Latest stories

Cam Newton Not Distracted by Greg Hardy Case
Cam Newton Not Distracted by Greg Hardy Case
Carolina Panthers quarterback Cam Newton told ESPN on Sept. 18 the ongoing controversy involving teammate and defensive end Greg Hardy -- who is involved in a domestic violence case -- won't serve as a distraction for the upcoming Sept. 21 game against the Pittsburgh Steelers.
 
 
Ron Washington Resigned Because He 'Was Not True' To His Wife
Ron Washington Resigned Because He 'Was Not True' To His Wife
Former Texas Rangers manager Ron Washington, who abruptly resigned two weeks ago, said the reason he stepped down was because he "was not true" to his wife Gerry.
 
 
Clay Matthews Relishing More Versatile Defensive Role
Clay Matthews Relishing More Versatile Defensive Role
Green Bay Packers linebacker Clay Matthews, who's been known for his sacking prowess since he came into the league, is dropping back into pass coverage more often in the Packers' 4-3 defensive scheme.
 
 
The Big Bang Theory Season 8 Premieres Monday as Double Pack
The Big Bang Theory Season 8 Premieres Monday as Double Pack
The Big Bang Theory Season 8 premieres with the first two episodes on Monday, Sept. 22 at 8 p.m. on CBS. Watch the newest The Big Bang Theory Season 8 teasers.
 
 
 

About the Geek Mind

The “geek mind” is concerned with more than just the latest iPhone rumors, or which company will win the gaming console wars. I4U is concerned with more than just the latest photo shoot or other celebrity gossip.

The “geek mind” is concerned with life, in all its different forms and facets. The geek mind wants to know about societal and financial issues, both abroad and at home. If a Fortune 500 decides to raise their minimum wage, or any high priority news, the geek mind wants to know. The geek mind wants to know the top teams in the National Football League, or who’s likely to win the NBA Finals this coming year. The geek mind wants to know who the hottest new models are, or whether the newest blockbuster movie is worth seeing. The geek mind wants to know. The geek mind wants—needs—knowledge.

Read more about The Geek Mind.