Pfizer spokeswoman Gwen Fischer told Pharm Exec that the deal came together in a matter of weeks, and that FoldRx will be fully incorporated into Pfizer’s research division. All of FoldRx’s employees are expected to become Pfizer employees when the deal is completed.

FoldRx made its name—literally—by discovering compounds that treat diseases related to protein misfolding, using a proprietary yeast-based target discovery platform. Its pipeline leader is tafamidis meglumine, a treatment for TTR amyloid polyneuropathy, which is a genetic neurodegenerative disease.

“Over the past five years the FoldRx team has successfully developed tafamidis from the bench stage to MAA submission,” stated Richard Labaudiniere, president and CEO of FoldRx, in a release. “Pfizer’s strong clinical and regulatory resources, global marketing reach, and commitment to the treatment of rare diseases will significantly enhance the ability to pursue the goal of efficiently bringing tafamidis to all patients affected by this devastating neurodegenerative disease.”

According to Pfizer, the deal is a reflection of the company’s new business unit structure. The original intent of the business unit was to target specialized expertise in specific research areas.

“This particular transaction will add another important component to the specialty care business unit and investigation medicines for rare and orphan diseases,” Fischer said. “The company also compliments our neurosciences disease area, which is currently a part of Pfizer’s specialty care.”

Earlier this year, Pfizer created an orphan and genetic disease research unit charged with discovering diseases affecting fewer than 200,000 patients in the United States and fewer than 10,000 patients in the European Union.

Pfizer has been upfront with the general public in stating that one of the key ways it will drive growth in the next few years will be through strategic business development, focusing on small to mid-size business deals such as the acquisition of FoldRX.

“Without exception, each member of the Genzyme board believes this is not the right time to sell the company, because your opportunistic takeover proposal does not begin to recognize the significant progress under way to rectify our manufacturing challenges or the potential for our new product pipeline,” Genzyme CEO Henri Termeer wrote.

The letter goes on to state that the company outlined a laundry list of improvements it plans to make in its manufacturing facilities—an area that Genzyme has been chastised for recently—as well as future earnings from its much lauded multiple sclerosis treatment alemtuzumab, currently in development.

The board feels that Sanofi is low-balling the company with its $69-per-share offer, and should be willing to boost the price since Sanofi has openly talked about how much they are willing to pay for mergers and acquisitions as recently as last year.

According to analysts interviewed by Bloomberg, Sanofi should expect to pay upwards of $77 per share for Genzyme, but might not bump up the offer until its had time to thoroughly examine Genzyme’s financials.

Sanofi CEO Chris Viehbacher originally offered the same price for Genzyme in early July when the genetic firm’s stock hovered around $50.

“Notwithstanding this information and assistance, you have not increased your price above $69 per share,” Termeer stated. “You and your advisors claim you are willing to pay more but that you are unwilling to ‘bid against yourself.’ The Genzyme board is not prepared to engage in merger negotiations with Sanofi based upon an opportunistic proposal with an unrealistic starting price that dramatically undervalues our company.”

This is the first time AARP has measured the retail price of Rx drugs. In the past, the lobby group only tracked the wholesale manufacturers’ price of generic, branded, and specialty drugs, which is traditionally considered to be much higher than the retail price.

The brand name pharma firms had criticized those reports for not taking into account rebates and couponing offered to pharmacies and hospitals.

“What we found was that the prices are still trending way up, and for the most part they are relatively similar increases that we received when we were measuring the manufacturer prices,” AARP spokesperson Jordan McNerney told Pharm Exec in an interview. “Those discounts and rebates aren’t making much of a difference on the bottom line as far as the consumer is concerned.”

The concern was that if the wholesale price of drugs was increasing, then the cost had to be passed along to the consumer.

Boehringer Ingelheim’s prostate drug Flomax saw the biggest jump in price, increasing nearly 25 percent. McNerney pointed out that the treatment’s price appears to have jumped as the drug prepared to go off patent.

In response, the pharmaceutical lobby group Pharmaceutical Research and Manufacturers of America (PhRMA), released a diatribe on its website, supporting drug companies for innovating new treatments. PhRMA also supported industry against catcalls from AARP and another study released at the meeting of the American Sociological Association that claimed that few of the new treatments released in recent years offer significant improvements to patients’ lives.

“AARP’s report is misleading because nearly half of the drugs on its top 25 brand-name drug list were filled as generics in the first part of 2010, but AARP counts these drugs as if they were brand-name drugs,” stated PhRMA Senior Vice President Rick Smith in a release. “The report calculates costs in this inaccurate way even though it acknowledges that brand-name drugs typically lose about 90 percent of their sales after going generic.  The result is an overstatement of consumers’ actual costs for these medicines and there is a tremendous disparity between AARP’s report and the numerous independent analyses showing drug costs growing slowly.”

McNerney explained that the patients most likely to feel the spike in price are those in the Medicare Part D “donut hole.” Once a patient reaches a certain threshold of drug treatment, they hit a cap where they have no coverage and must pay out of pocket.

“We are looking at Congress and industry to control these prices,” McNerney said. “We would love to see Medicare negotiate directly with drug companies so patients can get the best prices on drugs and we would like to see any legislation to get generic drugs on the market faster.”

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.

A research team in Belgium discovered a new test that has proven successful at identifying patients that are developing Alzheimer’s disease, according to a report published in the Archives of Neurology on Tuesday.

The study took spinal fluid from patients with varying levels of cognitive memory and looked for a specific protein signature or biomarker that has been attributed to Alzheimer’s.

Researchers tested more than 300 seniors—some with the disease, some suffering with recall difficulty, and some with no sign of Alzheimer’s. The study proved that 90 percent of the patients already diagnosed with the disease had the particular protein characteristic. Seventy-two percent of patients with some memory problems tested positive for the protein, and only 32 percent of patients in the normal cognition group had the biomarker.

Even more fascinating were the results of a second test of deceased patients confirmed to have Alzheimer’s. In that study, 64 out of 68 patients tested positive for the protein.

Healthcare professionals hope that the test can be used to detect Alzheimer’s way before the disease takes hold. Presently, the only way physicians can prove that a patient has the disease is post-mortem, making it very hard to isolate and treat it. Instead, doctors diagnose the disease through process of elimination—not the most reassuring method.

The downside to the test is that the spinal tap used to capture the fluid is notoriously painful, so it’s doubtful that healthy adults would elect to undergo the procedure—especially if insurance doesn’t cover it. There is also a fear of depression due to early detection or a false positive. The fact is, there are no medicines on the market to stop or slow the disease, so treatment—at this time—is fairly futile.

For the pharma industry, the new test could help researchers isolate trial participants by those most likely to have the disease, rather than test a broad range of people with cognitive problems, many of which might not have Alzheimer’s.

“We have to go very early to patients who have just the beginnings of Alzheimer’s in their brains—those are the people we need to identify to test the treatments,” Stephen Ferris at New York University’s Alzheimer’s Disease Center told CBS News. “That’s why these spinal fluid tests are going to be extremely important over the next few years.”

Novartis got hit with an untitled letter from FDA’s Division of Drug Marketing, Advertising, and Communications (DDMAC) on July 30 (posted August 4) for including a “Share on Facebook” widget on its branded website for Tasigna. This the first known enforcement by FDA in regards to Facebook—the massively popular social network.

The agency argued that the trackback link that the button generates is in violation of its marketing code because the link represents the benefits of the drug and not the adverse reactions.

To clarify, “Facebook Share” widgets are plug-ins added to blogs or websites that allow visitors to a site to repost a link to that site onto their Facebook profiles. What appears on the user’s Facebook page (or Wall) is a short excerpt from the site, a link to that page, and usually a thumbnail photo generated automatically. 

According to Digitas Health, the letter concerned the content provided by Novartis to the user, not about comments or messages left by random Facebook users. A major concern by industry and regulators has been how to control user-generated content on social media sites. For example, should a pharma company be held responsible if a banner ad for a drug appears on a public online forum where adverse reactions to the drug are being discussed—even if it’s a third-party site not associated with the drug firm?

“FDA seems more explicitly to be endorsing the view that it will hold pharmaceutical companies accountable solely for the material they provide for use in social media venues, not for any comments made about that material,” Digitas stated in a release. “DDMAC applied the principle of holding pharmaceutical companies accountable for filing only those materials generated by the pharmaceutical company.”

Digitas suggests that pharma companies eschew using the brand name on sites with a Facebook Share widget enabled or simply avoid making claims about the drug’s effectiveness.

FDA stated that the information in the Facebook link was pulled from the website’s metadata description, which Novartis has full control over. Users cannot modify the message; only add additional comments below the link. Digitas recommends that pharma companies planning on including a Facebook Share button should submit the proposed shared content as well as the site’s metadata for FDA review prior to a site’s launch.

“This is a critical milestone on the path to potential approval for short-course therapy with Cladribine tablets, moving us one step closer to meeting an unmet need as an oral, disease-modifying drug available for relapsing MS,” stated Fereydoun Firouz, president and CEO of Merck’s US affiliate, EMD Serono, in a release.

FDA originally rejected Merck’s application for any kind of approval in November 2009, prior to the release of CLARITY trial data. It’s possible that FDA reconsidered the application after reviewing the positive results from the two-year global study of 1,326 patients suffering from relapsing-remitting MS.

According to Merck KGaA, patients taking the short-course tablet treatment experienced “rapid and sustained improvements in clinical and magnetic resonance imaging outcomes, which were accompanied by rapid and sustained effects on blood-cell subtypes implicated in the pathogenesis of multiple sclerosis.”

Gavin Giovannoni, principal investigator of the study, told ThePharmaLetter, “The introduction of an oral therapy, particularly one that has no short-term side effects and is as easy to use as oral Cladribine, will have a major impact on the treatment of MS. However, the use of this drug as a first-line therapy will have to be weighed up against the potential long-term risks which have yet to be defined.”

Side effects included headaches, upper respiratory tract infections and nausea—similar to adverse reactions experienced by patients taking the placebo.

Merck is neck-and-neck with Novartis, which received priority review for its own MS tablet-based treatment, Gilenia, in February. There are currently no non-injectable MS treatments on the market, so an early approval is going to be a big win for patients and a financial windfall for whichever company is first to market.

This is a huge problem for Roche who purchased the treatment as part of its merger with Genentech. The drug firm could see sales drop by $1 billion if FDA agrees with the panel and ceases use of the drug for breast cancer. The drug earned Roche $5.7 billion in 2009.

“We are disappointed by the committee’s recommendation and believe Avastin should continue to be an option for women with this incurable disease,” stated Sandra Horning, Roche’s global head of clinical development hematology/oncology, in a release. “We will continue to discuss the data from the more than 2,400 women who participated in three Phase III studies with the FDA. This recommendation does not impact Avastin’s approved uses for other cancer types.”

Avastin (in combination with chemotherapy) was given fast-track approval in early 2008, because it treated HER2 negative breast cancer, a form of the disease with few treatment options. FDA requested that Genetech release the results of two post-market trials to determine how effective the drug is at improving patient lifespan.

According to the two trials, the treatment only slowed cancer progression by approximately a month and didn’t do anything to boost patient survival. Additionally, patients taking Avastin had more adverse reactions than patients taking chemotherapy alone.

FDA will give a final ruling on Sept. 17.

Antiretroviral sales in 2009 were estimated at $11.8 billion. While that figure is forecast to hit $14.4 billion in the next ten years, the number of drugs exceeding $400 million in annual sales is expected to drop from 10 to just six due, in part, to the strength of cross-class, fixed-dose combination (FDC) treatments.

FDC regimens replace the drug cocktails that patients have relied on for years. First out of the gate was Gilead Sciences’ and Bristol-Myers Squibb’s Atripla, which raked in $2.3 billion in sales in 2009, claiming 19 percent of the HIV market. The drug’s market share is so dominant that companies that aren’t trying to compete with this type of drug will probably be in trouble.

Besides Atripla, Truvada/rilpivirine and the Quad pill are being tagged as major HIV players when they launch in the next few years. “Atripla has been hugely successful, and we believe that follow-up FDCs will be just as successful, if not more,” said Hedwig Kresse, head of Vaccines & Infectious Diseases, Datamonitor.

Kresse said that pharma companies that sell HIV drugs but don’t have a combination treatment in their portfolio are going to have a tough time penetrating the market or keeping share in the market

“One example that shows that the market is getting tougher is Avexa’s apricitabine, which was discontinued by the company because it couldn’t find a development partner despite significant results in efficacy trials,” said Nele Jensen, associate analyst, Datamonitor.

Merck-Schering-Plough faced similar problems with its CCR5 inhibitor, vicriviroc, after the treatment failed to meet end points in late-stage trials.

Reimbursement is also becoming an issue in light of the push for restricted healthcare spending in many countries. Traditionally, drug combinations were priced as the sum of the single agents. But with mandatory discounts on the way, pricing for HIV treatments will become a challenging environment, Jensen said.

The only single-pipeline agent predicted to be a success is Shionogi & Co and ViiV Healthcare’s integrase inhibitor S/GSK1349572, which has shown positive Phase II results.

“HIV is a very sizeable market, and it’s a market that has been growing for the past decade,” Kresse said. “And for now, it’s a chronic market. You maintain patients for a very long period, which gives you a relatively stable revenue stream for good and efficacious drugs. But it’s getting harder to differentiate your drugs from the competition. And in light of the dominance of fixed-drug combinations, I think it won’t become an easier environment in the next decade—I think it will become tougher.”

Crestor’s patent is not scheduled to expire until 2016. However, a group of generics firms had claimed that a substance patent (314) protecting one of the active ingredients in Crestor was invalid, and that other companies had the right to file abbreviated new drug applications for generic versions of the cholesterol treatment. AstraZeneca sued them when they filed the applications, and a judge agreed with AZ.

“We are pleased with the court’s decision upholding the validity and enforceability of the ‘314’ substance patent,” said David Brennan, AstraZeneca’s CEO. “The court’s decision reaffirms the strength of the intellectual property protecting Crestor.”

The defendants claimed that Shionogi Seiyaku Kabushiki Kaisha—the patents original owner—engaged in shady tactics to get approval from the US patent office.  The court, however, disagreed chalking any filing discrepancies up to clerical errors.

“The court is not persuaded that the evidence presented by defendants rises to the level of the clear and convincing evidence required to establish inequitable conduct,” said Judge Joseph Farnan, US District Court in Delaware.

“While in hindsight it may be attractive to construct a deliberate scheme of deceptive intent from the actions of these individuals given the success of Crestor in the marketplace, it is at least equally plausible from their testimony and the contemporaneous documentary evidence, that a scheme to defraud was the furthest thing from the minds of these individuals at the relevant time, and that their vision was limited to the overwhelming demands they faced daily in their severely understaffed department.”

The generics companies also argued that the patent should not stand because the substance could have been created by anyone. The judge disagreed, and stated “there was much skepticism in the industry concerning the safety of rosuvastatin and the court finds it telling that no other pharmaceutical companies attempted to create a comparable product despite research in the area and the economic incentives of entering an additional player in the statin market.”

Analysts predict that the generics firms will fight the decision, but none of the companies have made a public statement as of Wednesday afternoon.