UPDATED: In rare slap, FDA review rips up Pfizer’s case for tafamidis

Posted under Blog, Companies, Diagnostics, familial amyloid polyneuropathy, FDA, FoldRx Pharmaceuticals, Funding, Medical Devices, Medical Supply, neurodegenerative diseases, Pfizer, Pharmaceuticals, rare diseases, Regulatory, Startups, Tafamidis, Universities, Videos by John Carroll

Pfizer gambled big when it took data from a single study to back its application for tafamidis as a treatment for a rare neurodegenerative disease. And an FDA review of the data concludes decisively that the company lost. After considering the trial's failure to meet its co-primary endpoints and questioning the data gathered at 8 sites, the agency is recommending that the experts gathering to discuss the drug's fate on Thursday reject the therapy.

"I recommend a Complete Response action, based on inadequate evidence of effectiveness," stated the FDA reviewers in a rare, outright slap at an application. The agency isn't bound by either the review or the panel vote on Thursday, but Pfizer's ($PFE) team will face an uphill struggle when it makes its case for tafamidis--a therapy for familial amyloid polyneuropathy--later in the week.

In the review, the agency investigator picks apart the data, wondering why most of the responders were clustered in one site while 7 other sites offered little by way of supporting data. "Overall, Study 005 does not have the characteristics of a single adequate and well-controlled study that could make the study adequate support for an effectiveness claim," the review states.

Pfizer, though, plans to put its case on the drug in the best light possible.

"There is currently no FDA-approved treatment designed specifically to treat TTR-FAP," Pfizer noted in a statement to FierceBiotech this morning. "If approved by the FDA, tafamidis will be the first and only medication in the U.S. to treat patients with this debilitating genetic disease. Pfizer believes that the data for tafamidis provide substantial evidence of effectiveness and meaningful therapeutic benefit where there is a high unmet medical need. We look forward to discussions with the FDA and its Advisory Committee to further the understanding of TTR-FAP and the data supporting our NDA."

Pfizer landed tafamidis when it acquired FoldRx in a buyout back in 2010 as it pushed into the rare disease field. Like other pharma companies, Pfizer was lured by the prospect of big returns for drugs that meet an urgent need in tiny patient populations. About 8,000 people around the world, including 2,500 in the U.S., suffer from the neurodegenerative disease Pfizer was focused on. But the pharma giant, which is still trying to shed its reputation as a poster child for clinical ineptitude, may be rethinking the business plan this morning.

- here's the FDA review
- read the story from Reuters

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Posted under ASCO, Blog, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, non-small cell lung cancer, Pfizer, Pharmaceuticals, Pipeline, Startups, Universities, Videos, Xalkori by John Carroll

Some of the biggest headlines coming out of the massive ASCO abstract release aren't focused on a promising experimental treatment. They're about Pfizer's ($PFE) recently approved treatment Xalkori. Now targeted at non-small-cell lung cancer tumors with ALK gene abnormalities, Xalkori was tested in children with rare cancers also linked to those defects. And its success in those kids illustrates how targeted drugs can progress beyond their initial uses to other cancers with similar genetic characteristics. FiercePharma's Tracy Staton reports on the ASCO news in our team coverage. Report

UPDATED: ASCO abstracts reveal added appeal for key cancer drug prospects

Posted under Array BioPharma, ASCO, AVEO Pharmaceuticals, Blog, Cancer, Cancer Drugs, Companies, Diagnostics, Funding, kidney cancer, Medical Devices, Medical Supply, Nexavar, Pfizer, Pharmaceuticals, Pipeline, rilotumumab, selumetinib, Startups, tivozanib, Universities, Videos by john

The avalanche of ASCO abstracts last night triggered a flurry of news reports about the most notable new data on experimental therapies to be revealed ahead of next month's big meeting in Chicago. Here are a few of the highlights:

Aveo Pharmaceuticals

Aveo Pharmaceuticals ($AVEO) is concentrating on new safety data to make the case that tivozanib will be superior to Nexavar in treating kidney cancer. Investigators already announced that patients taking the treatment gained several months of progression-free survival over Nexavar. Now the biotech's abstract reveals that the experimental therapy triggered fewer cases of serious hand-foot syndrome. And the company took the initiative to tout the fact that 35% of Nexavar patients had to interrupt treatment, compared to 18% in the tivozanib crowd. 

"I think the important point is that this is a next-generation VEGF TKI with a cleaner mechanism of action that's focused more intensely on the VEGF receptor, and the VEGF pathway appears to be the most critical pathway in kidney cancer, so if you can more selectively focus on that you can produce equivalent or better efficacy with less toxicity. That's the real potential value of tivozanib," Dr. Michael Atkins, a TIVO-1 study investigator and deputy director of the Georgetown Lombardi Comprehensive Cancer Center in Washington, DC, tells FierceBiotech Executive Editor Ryan McBride.

Pfizer

The data dump included a look at some positive new mid-stage results for Pfizer's ($PFE) dacomitinib, an experimental lung cancer therapy. Three out of four patients in the study demonstrated a response to the treatment, according to Reuters. The preliminary median  progression-free survival rate for patients with advanced cases was an impressive 17 months. Three of the 92 patients in the study had to stop therapy due to toxicity. The drug inhibits a variety of proteins that spur cell division and the pharma giant has a number of studies underway to determine its overall potential.

 

Cancer drug experts brace for ASCO abstract dump tonight

Posted under ASCO, Blog, Bristol-Myers Squibb, Companies, dabrafenib, Diagnostics, Funding, GlaxoSmithKline, Johnson & Johnson, Medical Devices, Medical Supply, personalized medicine, Pfizer, Pharmaceuticals, Pipeline, Roche, Startups, trametinib, Universities, Videos, Xalkori, Zytiga by john

Tonight's the big night for cancer drug watchers. ASCO is releasing a mountain of data on cancer studies, and some of the biggest names in the business will be angling for star billing on some closely watched therapies.

Peter Loftus at Dow Jones lists Pfizer ($PFE), GlaxoSmithKline ($GSK) and Johnson & Johnson ($JNJ) among the big names that will be featured tonight. But Bristol-Myers Squibb ($BMY) and Roche (T-DM1) plan to hold back on the headlines until the confab in Chicago gets under way.

For GSK, dabrafenib and trametinib are slated for top billing. Together, the two therapies could earn more than $700 million a year, Cowen reports. Pfizer, meanwhile, will be looking to make a case to expand the approval for Xalkori while J&J will make a case for Zytiga.

As Loftus notes in his report, one of the big themes this year will be the continued focus on personalized medicine, as investigators look for better results from cancer therapies targeted to specific patient populations.

- here's the story from Dow Jones

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