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Surge in federal approvals buoys drug makers

SAN FRANCISCO — Something odd is going on in the pharmaceutical world: Though the industry is coping with a sluggish economy and bracing for federal cuts in medical research funding, the mood among the 8,000 executives at the industry’s premier investors conference is decidedly upbeat.

As the 31st annual J.P. Morgan Healthcare Conference got underway Monday, talk was about the pace of US drug approvals picking up after a relative dry spell. Last year, the Food and Drug Administration allowed 39 new treatments on the market, up from 30 in 2011 and the most in 16 years.

Some of the drugs approved in 2012 are made by Cambridge-based biotechnology companies, including a cystic fibrosis treatment from Vertex Pharmaceuticals Inc., a leukemia drug from Ariad Pharmaceuticals Inc., a cholesterol medicine from Aegerion Pharmaceuticals Inc., and an irritable bowel treatment produced by Ironwood Pharmaceuticals Inc.


And the roster for this year could be just as full, according to industry executives and insiders.

“It’s a very, very exciting time,” said Harry Glorikian, managing partner at Scientia Advisors, a life sciences consulting firm in Cambridge. “I don’t see any reason why it’s going to slow down.”

Boston-area companies at the conference this week — including ImmunoGen Inc. of Waltham, Biogen Idec Inc., based in Weston, and AVEO Pharmaceuticals Inc. of Cambridge — are awaiting FDA rulings on applications for drugs to treat conditions ranging from breast cancer to kidney cancer to multiple sclerosis. Companies from other parts of the country are seeking permission to start selling drugs for Parkinson’s disease, hospital-acquired pneumonia, and many other illnesses.

For patients, the flood of novel medicines means saving or extending lives. For companies and the investors who take a chance on them, it is an opportunity to potentially cash in after years of research and many millions of dollars in spending.


“There’s a broad commercial opportunity,” said Daniel M. Junius, chief executive of ImmunoGen, which is expecting an FDA decision by next month on a promising breast cancer drug that was developed using the company’s targeted cell-killing agent, but was brought before the FDA by its marketing partner, Swiss drug maker Roche AG.

Although FDA rulings can be unpredictable, analysts say several factors have converged to produce the rise in the approval of new treatments. Financial backers have been funding later-stage drug programs. Partnerships between large drug makers, smaller biotechs, and academic researchers have pushed programs forward. And companies have become more disciplined about how to move drugs from clinical trials into the marketplace.

“All of these trends are coming together,” said Boston consultant Jonathan P. Gertler, cofounder and senior partner at Back Bay Life Science Advisors. “It’s the coming to fruition of a lot of the new focus in biotech. There’s a lot of rational exuberance here. And I think it’s going to be another good year for biotech and pharmaceutical companies.”

In part, the accelerated pace of drug approvals reflects an industry shift from blockbuster medicines that treat large numbers of patients to more targeted therapies aimed at smaller population pools. Federal regulators have been seeking to accommodate the rise of personalized medicine by fast-tracking some targeted drugs or giving them priority reviews.

“The agency has tried to bring in more [regulators] and gotten better at moving things through their process,” Glorikian said. Also, he added, drugs aimed at smaller groups of patients can come out of the clinical trial phase faster.


Vertex, for instance, last year got the green light to sell its cystic fibrosis drug Kalydeco three months before its scheduled decision date. The drug targets a genetic mutation that causes the disease in roughly 4 percent of the 30,000 Americans who have cystic fibrosis, a rare and often fatal genetic disease. Vertex scientists are working on variants of Kalydeco that could treat up to 90 percent of patients, company chief executive Jeffrey Leiden told investors in San Francisco Monday.

“Innovation is Vertex’s life blood, our secret sauce,” Leiden said, noting that two of its experimental drugs have been designated “breakthrough therapies” by the FDA, giving them the potential to receive speedy approval.

Junius, for his part, said his company’s breast cancer treatment will be the first of a number of drugs — marketed by larger partners such as Amgen Inc., Sanofi SA, and Novartis AG — to use ImmunoGen’s proprietary cancer-killing agents, which he said are 100 to 1,000 times more potent than traditional chemotherapy. Drugs in the firm’s development pipeline will treat cancers of the lung, ovaries, head, and neck, as well as non-Hodgkin’s lymphoma.

“You have to understand that this is a system we’re working on,” he said. “This is truly personalized medicine.”

But even with the rash of innovation, drug makers and other life science companies know it is not enough to make a great medicine or diagnostic device these days. They must work extra hard in a stubbornly weak economy to show commercial and government payers that their products not only meet unmet medical needs, but can save the health care system money.


“You can imagine that in the US and Europe today, going to market like we used to five or six years ago isn’t going to cut it anymore,” Jose E. Almeida, chief executive of Mansfield medical device maker Covidien PLC, told investors Monday.

Robert Weisman can be reached at weisman@globe.com.