FDA on Monday announced that it was looking to withdraw approval for the low-blood-pressure treatment midodrine hydrochloride because the companies manufacturing the drug failed to provide data from post-approval studies.

The kicker: The drug was approved 14 years ago.

FDA gave midodrine hydrochloride—branded by Shire as ProAmatine and produced by a half-dozen generics firms—the green light in 1996 as part of the fast-track approval program designed to speed to market drugs for diseases with no current treatments.

The catch is that FDA requires post-market clinical trials to ensure that the drug is meeting risk/benefit endpoints. In other words, the regulatory body wanted to make sure no hiccups occurred with the treatment when it hit the general population.

In response, Shire—who acquired the drug when it bought Roberts Pharma in 1999—chose to withdraw the drug as of September 30. The drug firm made it clear that the withdrawal had nothing to do with any safety concerns. In addition, Shire stated that it had conducted post-market trials in conjunction with Roberts, but FDA felt the results were “inconclusive.”

According to a release by FDA, none of the companies selling the drug have provided any data to prove that the treatment is beneficial. That said, some 100,000 people were treated with the midodrine hydrochloride last year alone.

“We’ve worked continuously with the drug companies to obtain additional data showing the drug’s clinical benefits to patients,” stated Norman Stockbridge, director of the Division of Cardiovascular and Renal Drugs in the FDA’s Center for Drug Evaluation and Research. “Since the companies have not been able to provide evidence to confirm the drug’s benefit, the FDA is pursuing a withdrawal of the product.”

FDA stated that patients currently on midodrine hydrochloride should not stop taking the medication. Shire now has 15 days to respond to FDA’s inquiry and provide some data supporting the drug. Shire did not respond to calls as of Monday afternoon. In addition, it’s unclear as to why FDA waited so long to ask for more data and whether any adverse reactions have been reported pertaining to the drug.

Emmens, a 35-year pharma industry veteran, has been a Vertex director since 2004. He previously served as CEO of Shire plc and Astra Merck Inc., and as president for Merck KGaA’s global prescription pharmaceuticals.

As Shire CEO, he expanded the company through mergers and acquisitions such as Transkaryotic Therapies Inc. in 2005, and New River Pharmaceuticals Inc. in 2007, diversifying the company’s product portfolio and quadrupling its share price.

With a potential blockbuster in the pipeline—protease inhibitor telaprevir, currently in phase III clinical trials—Emmens will have another chance to flex his management and marketing muscles. Last November, phase II trial results showed a 52 percent SVR12 in Hepatitis C Virus (HCV) treatment-failure patients with a 24-week treatment duration, therefore reaping better results in half the time than the existing standard.

“We have a rare opportunity to create a world-class commercial enterprise that complements Vertex’s excellence in R&D innovation,” said Emmens in a Vertex press release. “In the area of hepatitis C, and also in cystic fibrosis, Vertex has the potential to transform patients’ lives and build tremendous value. Together with the employees my goal is to make this vision a reality.”

But now that the bubble has burst, pharma can expect what Russell predicted a decade ago: a more competitive industry, with greater focus on pharmacoeconomic data, ROI, and less tolerance for me-too drugs. “The economic miracle is over—there will be a struggle to survive in this industry,” he says.

Russell, a speaker at the Reuters healthcare conference today, breakfasted with Pharm Exec at the W Hotel, and offered a preview before tomorrow’s main event, the business update for its Human Genetic Therapies (HGT) business. The meeting will take place in HGT’s new Lexington, MA, headquarters, and with more than 1,000 employees (up from 300 in 2005), it will also provide a visual reminder of how this business is a growth engine for Shire.

Over the last three years, this specialty pharma company has grown and diversified both its product portfolio and geographic reach. A few years back, Adderall XR accounted for the lion’s share of Shire’s sales. But recently announced Q3 earnings show that the company has succeeded in bringing along its newest drugs—and for the first time, new product sales exceeded those of Adderall XR (and not a moment too soon, as generic Adderall arrives on the market in just six months). With almost two dozen potential launches planned for 2008-2015, Russell says the company is in “the best shape it’s ever been.” He attributes this to strong IP (even for its next-stage ADHD drug Vyvanse), a heavy focus on orphan drugs, and Shire’s litany of productive deals including Jerini.

On Tuesday, Russell will address HGT and outline Shire’s seven-year plan. (“Five years was too short, 10 too long,” says Russell.) It includes expansion into China and  a reorganization along patient lines; drugs for diseases that serve more than 50,000 patients will fall under specialty pharma head Mike Cola; smaller drugs will fit within Shire HGT, presided over by Sylvie Grégoire. Russell says this paradigm best capitalizes on the different commercialization models, enabling smaller orphan drugs to fully leverage the “advocacy-based selling model.”

But it’s not all roses for Shire. In the short-term, the company will experience a dip in sales in 2009, and possibly 2010, as it fully transitions away from Adderall XR. Perhaps a bigger issue is the biogenerics bill that’s shaping up among legislators. Russell plans to visit Washington, DC, several times over the next few months to conduct what he characterizes as much-needed education on the interaction between intellectual property and biosimilars. Right now, small molecules have 20 years of patent life, but biologics only have seven. “Without 12 or 14 years of exclusivity, it just might not warrant the investment,” says Russell. “You see the reaction when I tell people that—their eyes light up and you can tell they are beginning to understand.”

While I’m not 100 percent positive, it looks like this might have been the first time FDA actually name-dropped a social networking service in a warning letter. Which poses the question: Is FDA watching YouTube, and what does this mean for pharma?

Ignite Health’s Fabio Gratton told me that this ramification could go one of two ways. Pharma could view it as the feds are now treating YouTube with the same degree of importance as television and other media outlets, thereby validating the online service. Or it could scare pharma away from posting videos on YouTube, because that could warrant a warning letter.

So where does FDA stand?

I sent an email to FDA spokesperson Felicia Stewart with one question: Are you guys watching YouTube for pharma ads?

She missed the deadline for the article, but lo and behold, this morning I got a nice, concise answer from the feds.

DDMAC has been and continues to monitor the many vehicles that companies use to promote their prescription drug products. Our monitoring includes magazine ads, TV ads, promotional exhibits at medical conferences, Internet, sales brochures, journal ads, and consumer mailers and brochures. Internet monitoring includes promotion done by or on behalf of drug companies such as companies’ own product Web sites and their placement of promotion on others Web sites. The action addressing promotion by the company on YouTube is one example. Another example is the action addressing Pfizer’s promotion on cnn.com for Viagra.

There you have it. FDA is watching YouTube, and boy, do I wish I was the guy or gal being paid to troll the Web all day looking for errant pharma ads or remixes of the Rozerem beaver singing the Tay Zonday classic “Chocolate Rain.”