FDA approval & regulation

Mass. Governor to sign $1B incentives plan

John Carroll — Mon, 16 Jun 2008 07:16:55 -0400

Thousands of biotech execs flocking to San Diego for the annual BIO meeting are likely to hear an earful from Massachusetts Governor Deval Patrick, who will be arriving with a freshly approved, $1 billion incentives program to woo biotech companies. Lawmakers had signed off on the legislation late last week, leaving Patrick just enough time to sign the bill today before hopping a flight to the West Coast confab.

The best thing that Massachusetts has going for it is a thriving cluster of life science companies that have created a legion of experienced executives and top scientists--many of whom work among the universities in and around Boston. That kind of cluster effect--added to a new slate of incentives--is likely to make the state much more effective at attracting more companies to the area. It should also help overcome many of the bureaucratic barriers that start-ups faced in Massachusetts.

Massachusetts made FierceBiotech's list of top economic development areas for the industry this year--a first for the state.

- read the story from the Boston Globe

FDA picking up the pace on '08 approvals

Thu, 15 May 2008 06:59:58 -0400

Last year's awful record of new drug approvals by the FDA has been followed by a burst of new activity. Pharmalot reports new analysis indicating 29 new drug approvals through April, the fastest rate of acceptance since 1998 and 2000. And that's way up from the anemic 16 approvals posted for the same period last year. But ten of the 29 approvals are for therapies that build on existing drugs and there were only three NME approvals for the period.

"We view NME approval trends as a more critical metric than overall new drug approval trends because NMEs represent novel compounds carving out entirely new pharmaceutical niches. These are the compounds that break new clinical ground and require the most effort in educating physicians, patients, and payers," writes Jim Kumpel, a healthcare analyst at Friedman Billings Ramsey.

- read the report in Pharmalot

Intercell snaps up Iomai in $189M deal

Tue, 13 May 2008 06:59:57 -0400

The little Maryland-based vaccine developer Iomai, which has posted some impressive data on a new therapy for traveler's diarrhea as well as an adjuvant for bird flu vaccines, is being bought out by Intercell for about $189 million. Intercell gains a mid-to-late-stage product in the needle-free diarrhea program, adding it to a Japanese encephalitis vaccine it has in development. The diarrhea patch is scheduled to enter Phase III in the first half of 2009. Intercell is looking for approvals of its vaccine by regulators around the globe this year.

In the deal, Iomai's public investors get cash for their shares while a group of large stakeholders will exchange their Iomai shares for shares in Intercell. Iomai was featured as one of FierceBiotech's emerging drug developers.

- here's the release
- read the AP story

Roche posts new data backing Actemra for RA

Mon, 12 May 2008 06:59:54 -0400

Roche has racked up more late-stage data backing Actemra as a new therapy for rheumatoid arthritis. Researchers say their fifth Phase III trial demonstrated that the therapy inhibited structural damage to joints. And physical function was improved in patients after a year of treatment.

Actemra is widely viewed as one of the three most important late-stage RA drugs in the global pipeline. The wave of late-stage therapies includes Cimzia (certolizumab) and golimumbab, both angling for near-term FDA approvals in the hope of joining the group of anti-TNF therapies on the market. Actemra (tocilizumab) is in a new, non-TNF category that is an odds on favorite to gain approval as a second- or third-line treatment.

- here's Roche's release
- read the AFX report

Argos garners $35.2M in third round

Wed, 23 Apr 2008 06:59:55 -0400

TVM Capital led a hefty $35.2 million third round of venture capital for Colorado's Argos Therapeutics. The funding will be used to support Argos' Phase II trial of Arcelis immunotherapy drug candidates in renal cell carcinoma and HIV. In addition, it will finance the company's CD83 protein immunology program through an initial human clinical trial in transplantation.

"We believe that the Company's many near-term data events, combined with its ability to move into additional cancer and infectious disease indications with its Arcelis platform technology, as well autoimmune disorders with its CD83 soluble protein, position Argos as a leader in the immunotherapy field," said Hubert Birner, Ph.D., General Partner of TVM Capital and Argos' Chairman. Argos was featured in FierceBiotech's weekly column on emerging drug developers.

- see this Argos release

Cardiome's CBO discusses changes at FDA

Fri, 28 Mar 2008 07:59:56 -0400

During a conference call with investors yesterday, Cardiome's President and Chief Business Officer, Doug Janzen, had some interesting thoughts on the slow approval process at the FDA:

"The FDA has actually come out and said it will be missing (PDUFA) dates... I think everyone is in a brand new environment... actually, some of the old-timers in the industry are talking about we're back in the old environment where the FDA just doesn't deal with timelines. They're missing PDUFA dates, they're missing type-C meetings, they're missing meetings to discuss protocol..."

Janzen's comments stem from the approval delay of Cardiome's intravenous drug vernakalant for atrial fibrillation. Despite being approved by an expert committee, the company has been awaiting FDA approval since the drug January 19th PDUFA date. His observations highlight the ongoing problems at the FDA. The agency is struggling to meet PDUFA dates, ensure food and drug safety, and maintain enough staff to deal with it's ever-growing list of responsibilities.

On the subject of PDUFA dates, however, it seems that an approval delay may be preferable to a hastily-made decision. Just yesterday a Harvard analysis found that that drugs approved in the two months leading up to the deadline are four to five times more likely to be withdrawn or require serious safety warnings. So is it better for the FDA to scramble to meet deadlines--and possibly miss safety problems--or miss the approval date that companies pay hefty fees for the FDA to meet?

- read the Motley Fool piece
- check out Cardiome's financial results

ALSO: Speaking of that study, the FDA says that it can't replicate the Harvard study's numbers. The agency is sending a detailed letter disputing the results to the New England Journal of Medicine, which published the research. Report

Related Articles:
Study: Last-minute FDA approvals less safe. Report
Developers: Politics behind slow approval process. Report
PDUFA debate highlights drug safety issues. Report
The FDA: Caught between a rock and a hard place. Report
FDA quick to reject drugs as it gets more cautious. Report

In Austin, VC is booming for biotechs

Fri, 28 Mar 2008 07:59:55 -0400

Further evidence of Texas' growing power in the biotech industry: the Austin Business Journal reports that in recent months, central Texas-based life science outfits have attracted about $500 million in VC. "The 'we can't do bio in Austin' attitude is no longer true," said Isaac Barchas, director of the University of Texas' IC2 Institute. "Taken together, it feels like this is the moment when things are happening in bio. I think in two years we'd point to this moment and say, 'that was when things started to happen.'" Texas' biotech efforts--particularly its $3 billion bond program for cancer research--propelled it to first place on FierceBiotech's annual list of the top five regions attracting biotech business.

- see the Austin Business Journal piece
- read FierceBiotech's special report on Texas. Report

Related Articles:
Texas' $3B cancer program draws controversy. Report
Texas touts $3B plan for cancer research. Report
Venture group targets tech transfer deals in Texas. Report

Risk averse? Not us, says top FDA official

Wed, 26 Mar 2008 07:59:58 -0400

Is a rising tide of risk aversion at a post-Vioxx FDA behind the agency's anemic approval record? No way, say the FDA's Janet Woodcock (photo). It's all about the science. And the science, she insists, is pointing to troubling indicators about some of these new drugs.

"We have a different story from the companies," Woodcock told the Financial Times. "We understand that [they] have productivity issues but [we] stick to the same safe and effective criteria as our bedrock. Pharma is having a difficult time but we are seeing drugs that have a lot of questions."

The length and size of clinical trials are growing, Woodcock acknowledges, but that's because regulators know more about what they need by way of data. And new efforts to standardize trials--à la Henry Ford--should help matters. "We need to learn from Henry Ford. Companies collect too much information because they are worried that the FDA will ask for it."

- check out the article in the Financial Times

Related Articles:
2007 FDA approvals. Report
More mixed messages from the FDA. Report
FDA: Hyper-cautious or simply vigilant? Report

2008 NME approvals no better than last year?

Wed, 12 Mar 2008 07:59:54 -0400

In 2007, there was much ado about the slow pace of new molecular entity (NME) approvals. Though over 200 drugs were approved in 2007, only 19 of them represented groundbreaking advances in medicine.

In 2008, according to a post on Pharmalot, the industry is set to repeat last year's lackluster performance. Thus far the FDA has approved 14 NDAs but only one NME--the same rate of approval as last year. Why so slow? Analyst Jim Kumpel of Friedman Billings Ramsey says the FDA is hard at work implementing initiatives that resulted from PDUFA reauthorization. Others, including industry execs, are likely to blame the slowdown on a skittish post-Vioxx FDA that's suffered serious criticism for approving unsafe drugs.

- here's the Pharmalot item
- see the full list of 2007 FDA approvals

Related Articles:
Dry spell or parched desert for NME approvals? Report
'07's drug approval record raises worries. Report
New drug approval lags in 2007. Report
FDA quick to reject drugs as it gets more cautious. Report
Industry exec says FDA approval process too strict. Report

FDA official says approval deadlines will be broken

Wed, 05 Mar 2008 06:59:56 -0500

The FDA is sending signals that it will start to let deadlines for drug approvals go by the wayside. In Vivo has picked up a report in BioCentury that John Jenkins, the head of the FDA's Office of New Drugs, is providing unofficial permission to miss deadlines. With the agency facing a series of stiff new challenges, the BioCentury report suggests that 10 percent of deadlines could be busted. That would be an enormous blow to drug developers, who have provided huge sums to the FDA in fees in return for its assurance that it will stick to a relatively secure timeline for approvals. Missed deadlines would translate into higher costs and lost revenue, something the industry isn't likely to take lying down.

"We are having to prioritize. We are having to set aside some things, hopefully on a temporary basis, until we can get adequately staffed," Jenkins reportedly told BioCentury. Get ready for the backlash, though.

- read the report on the report in the Wall Street Journal

ALSO: The government's own watchdog will soon be sniffing around FDA drug approvals and FDA-ordered post-marketing studies. Report

Related Articles:
'07's drug approval record raises worries. Report
Dry spell or parched desert for NME approvals? Report
New drug approval lags in 2007. Report
FDA quick to reject drugs as it gets more cautious. Report
Industry exec says FDA approval process too strict. Report

Blogster sees lessons in FDA approvable letters

Thu, 21 Feb 2008 06:59:54 -0500

The blogster Eye on FDA has tracked down and posted every approvable letter he's heard about for 2007. The crop of letters highlights some particular challenges for new drugs targeting ADHD, pain and depression. The 29 letters marked a record for the FDA, particularly for multiple letters covering the same therapy. "And even though we are in a severe "risk-averse" environment, one could say that efficacy was every bit as much a factor as safety in the 2007 crop of letters."

- read the blog report

Related Articles:
2007 FDA approvals. Report
FDA more friendly to approvable than approved. Report
'07 drug approval record raises worries. Report
Dry spell or parched desert for NME approvals? Report
New drug approval lags in 2007. Report

Scientists examine Selzentry's ability to stop HIV

Wed, 30 Jan 2008 06:59:57 -0500

Pfizer is set to license its HIV drug Selzentry to a nonprofit research group, which will attempt to reformulate the therapy so it can be used to prevent the spread of the disease. Preventing HIV has been an elusive goal in the development world, most notably with the recent high profile failure of Merck's vaccine program. Now International Partnership for Microbicides (IPM) will have a chance to reformulate Selzentry as a vaginal gel, ring or film that can prevent the spread of the lethal virus that triggers AIDS. Selzentry has inspired hopes for a preventive approach to the disease because it prevents the virus from invading healthy cells at the CCR5 entry point--a common avenue of transmission.

- read the article from The Wall Street Journal

Related Articles:
Special Report: 2007 FDA approvals--Selzentry
Selzentry approval marks big win for Pfizer. Report
New HIV drugs offer breakthrough therapies. Report

Publishing negative data is harder than it seems

Fri, 18 Jan 2008 06:59:59 -0500

We're always glad to hear from readers, even if they're taking exception to something I wrote. Jennie Mather, PhD, the founder, CSO and president of Raven Biotechnologies, had this to say about yesterday's piece on drug research studies and the bias toward positive data.

"I am a regular reader of FierceBiotech and usually find that you maintain a balanced presentation in spite of the brevity of your pieces. However, I must comment on your piece on "Positive data more likely to find it's way to the public" in yesterday's issue.

As someone who spent many years heading an academic research lab, as well as working in both research and development in two biotech companies, I can state that the area of publishing negative findings is one where both the publishing industry and pharma reinforce each other's biases. I have tried on several occasions to publish negative data only to have it returned, sometimes without review, with the statement that it is "only negative data" (I have published more than 150 articles so I have experience with the system). I think attributing the lack of such publications to "developers gaming the system with such indifference to the scientific pursuit of the truth" is a vast oversimplification.

I agree that negative clinical trial data should be in the public domain but it will take more than just the drug/biotech companies to make it happen. Perhaps the FDA could make publication of all clinical trial results a condition of approval of the protocol, but they then need to find a journal or journals that will guarantee publication of these results even when they are negative."

Got a comment? Send it in. - John Carroll

P.S. FierceBiotech will not be published this coming Monday in observance of the holiday. We will return on Tuesday, January 22.

FDA more friendly to approvable than approved

Thu, 10 Jan 2008 06:59:57 -0500

You've probably already heard that FDA approvals took a nasty dive last year, with analysts pondering a variety of reasons for the poor performance. Interestingly, Eye on FDA cites new research from Sargient showing that as approvals were sliding, approvable letters from the FDA were spiking--up 40 percent! Michael Hay, senior analyst and product manager for BioMedTracker, says that "The FDA has received a lot of criticism over several high profile adverse events cases including Vioxx, Tysabri and Avandia. This has triggered an overly cautious approach to new drug approvals at the FDA. Other potential reasons for the decline in approval rates cited in our report include an increase in adverse event reports, increased workload for FDA employees, and a high rate of turnover at the FDA."

Meanwhile, investors are less likely to bid up a stock when they see news of an approvable letter, largely because they are less likely to believe that the drug will be approved. Given the fact that some approvable letters contain a lot more bad news for biotechs than good news, it's likely that investors will be increasingly wary of their impact.

- here's the press release
- check out the report from Eye on FDA

Related Articles:
'07 drug approval record raises worries. Report
Dry spell or parched desert for NME approvals? Report
New drug approval lags in 2007. Report
FDA quick to reject drugs as it gets more cautious. Report

Emerging Drug Developer: Ambit Biosciences

Fri, 14 Dec 2007 06:59:59 -0500

Emerging Drug Developer: Ambit Biosciences

Ambit Biosciences has taken another important step forward. In the middle of last month, CEO Scott Salka announced that it had garnered about $50 million in its fourth round. That money should be enough to get the company through 2009, a key transition period for Ambit. "We're expecting at least clinical proof of concept and completion of that milestone with one compound by the end of '09." Salka tells FierceBiotech, which named Ambit a Fierce 15 company in 2006. Article

Insomnia market attracts drug developers

Mon, 08 Oct 2007 06:59:54 -0400

The market for insomnia drugs in the U.S. is growing at a whopping 29 percent per year, hitting $4.6 billion in 2006 and attracting the attention of a wide range of drug developers. More than a dozen compounds are in mid- to late-stage clinical trials in a race to capture a market that includes some 70 million Americans who suffer from chronic or occasional insomnia. Among the leaders in that race, according to analysts, are Somaxon and Neurocrine Biosciences, both of which are looking for near-term FDA approvals on late-stage drugs. Somaxon researchers believe that their therapy, which relies on an old antidepressant, will work by blocking the release of histamine at the H1 receptor in the brain.

- here's the report from BusinessWeek

Related Articles:
Somaxon touts latest Phase III results for Silenor. Report
FDA wants more data on Neurocrine's indiplon. Report
Arena Pharma produces positive insomnia data. Report

China drug czar executed for corruption

Tue, 10 Jul 2007 00:01:36 -0400

Just weeks after being sentenced to death, China's former drug czar was executed today. The swift punishment--meted out for Zheng Xiaoyu's alleged acceptance of about $850,000 in bribes--is intended to help restore China's tainted reputation in the pharmaceutical field. The country has become notorious for allowing dangerous products onto the market, seriously damaging the country's reputation at a time it is trying hard to expand the industry. A second FDA official was recently sentenced to death as well, but legal experts say there's a good chance that sentence could be commuted.

- read the report from CNN

Related Articles:
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FDA approves Novartis' Alzheimer's patch

Mon, 09 Jul 2007 00:01:34 -0400

Novartis has won FDA approval to market its once daily skin patch Exelon for mild to moderate Alzheimer's. The patch is designed to give a continuous dosage of Exelon that delivers the same efficacy rate as the highest dose of the drug in capsule form. It's also supposed to help patients avoid some of the side effects of the capsule while improving memory and everyday performance.

- check out the release on the patch
- here's the AFX report

Related Articles:
New studies support use of Exelon patch. Report
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Provenge delay triggers furor among cancer victims

Fri, 06 Jul 2007 00:01:35 -0400

The FDA's decision to delay approving Provenge until Dendreon can provide additional efficacy data has triggered intense anger among patients desperate to get the vaccine, which is designed to allow patients to live longer with advanced prostate cancer by stimulating their immune systems. The FDA's delay was at least partly attributed to the opinions of two top oncologists who questioned the drug's effectiveness. Now the experts have been exposed to threats, accusations of conflicts and the full-throated furor of cancer advocacy groups.

One other reason for all the buzz this week about Provenge: A new study is raising questions about the way cancer vaccine trials are designed. Cancer vaccines and therapeutics have been tested the same way as trials sought data related to tumor shrinkage. But vaccines can take longer to demonstrate a benefit and the study in Clinical Cancer Research suggests that the vaccines will allow patients to live longer and suffer less toxicity.

- read the article from The Washington Post
- check out the report on the new study

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FDA regulators question effectiveness of Provenge. Report

Genzyme shifts focus after hylastan fails trial

Thu, 05 Jul 2007 00:01:37 -0400

Genzyme announced that it will focus its commercialization efforts in the arena of osteoarthritis pain on Synvisc-One after its experimental drug hylastan failed to achieve its primary goal in a clinical trial. Hylastan was being studied as a potentially less expensive therapy for OA pain. But researchers found no significant difference in patients' response to either hylastan or steroids. Genzyme has submitted an application to the FDA for Synvisc-One and plans to apply for a CE Mark to introduce Synvisc-One in Europe.

"We structured our development program in a way that provided us with several chances to reach our goal of introducing a single-injection product," said Ann Merrifield, president of Genzyme Biosurgery, which manufactures and markets Synvisc. "It is now clear that we will focus our efforts on bringing Synvisc-One to the market."

- check out the release for more information

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