Ever wonder how the cancer drug Sutent made it to market? Barely, according to a behind-the-scenes piece from Bloomberg. The drug almost hit the R&D discard pile back in 2003, said Dr. George Demetri, an oncologist who was involved in testing it. It was a meeting with FDA officials encouraged by the data that got the drug back on track, Demetri said.

Sutent has gone on to prove effective for both kidney cancer and gastrointestinal stromal tumors–and to become one of the more expensive cancer drugs on the market at around $50,000. But even at that price, it’s become key to a treatment approach known as daisy-chaining, in which patients use one drug until it stops working, then move on to another. Some patients have survived as long as seven years this way.

“Improving cancer survival rates are a real success story that sometime get lost in the noise over our healthcare system,” Douglas Blayney, president of the American Society of Clinical Oncology, tells Bloomberg. “Targeted drugs are driving that survival in a major way.”

Is paying for that survival sustainable? In Sutent’s case at least, even the U.K.’s cost watchdog thinks so; the National Institute for Health and Clinical Excellence finally OK’d the drug after Pfizer agreed to pay for the first cycle of treatment, which should weed out the patients who just won’t respond to the drug at all.

But taken together with other expensive therapies, some experts doubt that society can keep paying for such costly treatment. “All the recent health policy talk is that in the U.S. we don’t ration, but that isn’t a true statement,” said UnitedHealth Group’s SVP Lee Newcomer. “We just keep pricing more and more people out of the ability to afford health insurance.”

Source: FiercePharma

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The FDA has approved Cell Therapeutics’ facility to manufacture Pixuvri (pixantrone), an experimental cancer drug that is currently under review. The NerPharMa facility, which belongs to Nerviano Medical Sciences, is based in Nerviano, Italy. “FDA approval of the NerPharMa facility to manufacture our drug product is a major milestone in the drug approval process and we are pleased that our manufacturing partner is prepared to provide commercial supplies when pixantrone is approved,” says Craig Philips, president of CTIC.

Cell Therapeutics’ closely-watched pixantrone is a potential treatment for relapsed/refractory aggressive non-Hodgkin’s lymphoma. The FDA’s decision will be a make-or-break moment for the struggling developer, with many analysts expecting the FDA to rule against the drug. FDA staffers have raised questions about the treatment, expressing concerns about both the drug’s effectiveness, as well as its safety profile. An ODAC meeting is scheduled for March 22, and the FDA is expected to make a final decision on approval of the NDA for pixantrone by April 23.

Source: FierceBioteck

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That U.S. Senate probe of GlaxoSmithKline and its diabetes drug Avandia may not yield any new regulatory action. But with more than 13,000 personal injury lawsuits outstanding, UBS analysts say, the company could be looking at a multibillion-dollar liability.

The safety of Avandia has been open to debate for almost three years now, and the Senate report didn’t bring up anything entirely new about the drug’s risks. The FDA already was planning to look at safety info on the diabetes med again, with a new advisory panel meeting on the schedule for this summer. The worries focus on a.) political pressure from the Congressional committee that sponsored the probe; and b.) those lawsuits.

“[O]ur concerns are solely on personal injury lawsuits,” UBS analyst Gbola Amusa said. Experts polled by UBS put the potential liability at anywhere from $1 billion to $6 billion; the bank itself expects something less than $3.5 billion. “Bellwether trials start from 1 June and will help narrow our liability range,” Amusa also notes.

Source: FiercePharma

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The FDA staff said it has a bunch of questions about InterMune’s new drug to slow deteriorating lung functioning. But investors figure the concerns weren’t as bad as they could have been, sending the biotech’s shares soaring.

The stock jumped as much as 74% after FDA reviewers said only one of InterMune’s two late-stage trails for the experimental drug pirfenidone had met its main goal and added that “the clinical significance of the treatment effect size is uncertain.” The FDA documents, which were released ahead of an advisory panel meeting to discuss the drug Tuesday, also had a mix of other things to say.

All things considered, an Oppenheimer analyst noted the “tone was less negative than expected,” all things considered and there was room for the drug to still get FDA approval. The shares settled down in later trading, but still finished up nearly 60% for the day.

InterMune often appears on lists of likely biotech takeover candidates. FDA approval of pirfenidone would give it two drugs on the U.S. market.

The new drug would treat idiopathic pulmonary fibrosis, which affects about 200,000 Americans, the majority of whom eventually die of respiratory failure. The FDA noted that there aren’t any approved drugs to treat IPF, although drugs like corticosteroids and drugs that suppress the immune system are used, according to Dow Jones Newswires.

The company also put out material noting that “the slowing of progression in loss of lung volume constitutes a clear benefit to patients,” Reuters said.

Source: The Wall Street Journal

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In what could be a win-win decision for both Teva Pharmaceutical and Merck, a federal court said that Teva was entitled to six months of exclusivity to sell generic versions of two hypertension medicines made by Merck.

A district court ruled in July that Teva, the largest of the generic drug makers, had forfeited the 180 days of exclusivity that usually goes to the first applicant seeking FDA approval to make a generic versions of drugs — in this case, copycats of Merck’s Cozaar and Hyzaar.

But Teva said today an appeals court found the Israeli company should get the exclusive sales period once its gets the FDA go-ahead to market the drugs. Teva said it would be eligible to begin sales in April.

Merck benefits because it was expecting a gaggle of generics for the two drugs to hit the market right away, according to Reuters. The U.S. company said its lawyers were reviewing the decision, the report said.

World-wide sales of the hypertension drugs were $3.6 billion in 2009. The exclusive-sales period allows a generic maker to charge a little more for having the first copycat version on the market, Reuters notes.

Source: The Wall Street Journal

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CombinatoRx’s complex merger deal with Neuromed will pay off with a $40 million milestone earned with today’s announcement that the FDA has approved Exalgo, a once-daily pain therapy. Covidien, which partnered on the program, will handle the commercial rollout and fork over the cash.

CombinatoRx (CRXX), which was forced to restructure last year after its lead program failed a key clinical trial, also saw its share price jump 44 percent in pre-market trading. Aside from the milestone, the approval also nails down the respective equity stakes for shareholders.

Neuromed shareholders obtained a sliding stake in the merged outfit based on the timing of an FDA approval, with their equity shrinking based on any delays. Pre-merger CombinatoRx shareholders saw their equity stake jump to 40 percent when 2009 closed without an approval. That figure is now set in stone.

Source: FierceBiotech

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FDA cleared Shire’s Gaucher disease enzyme replacement therapy (ERT), VPRIV™ (velaglucerase alfa for injection). The drug is a human cell line derived hydrolytic lysosomal glucocerebroside-specific enzyme, indicated for use as long-term ERT in adult and pediatric patients with type 1 Gaucher disease.

FDA approval took just six months, following the agency’s designation of VPRIV for priority review. Meanwhile, regulatory review of VPRIV in the EU is ongoing via an accelerated assessment route. Shire anticipates launching the drug in the EU by the end of 2010 and in other countries starting in 2011.

Shire says it plans to price VPRIV 15% lower than the currently available commercial Gaucher disease therapy, Genzyme’s Cerezyme. The firm also aims to make access to VPRIV in the U.S. easier through the implementation of a new Co-Pay Assistance Program within its existing OnePath Access Program. The Co-Pay route is designed to simplify the process and paperwork associated with the initiation of therapy and reduce financial burden on patients, Shire states. Co-Pay will also be available to eligible Hunter syndrome patients requiring therapy with Elaprase®.

Genzyme reported Cerezyme-related revenues of $793,024 in 2009, down from about $1.24 million in 2008. The shortfall relates largely to a four-month halt in production of the drug between June and September 2009, caused by the shut-down of Genzyme’s Allston Landing production plant due to viral contamination. With production now back up and stocks being replenished, Genzyme says patients are expected to be able to resume normal dosing schedules in the first quarter of 2010.

Just last month, Genzyme also said that to more consistently manage the re-supply of Cerezyme to patients worldwide and reduce the interruptions in shipping that occur in the absence of inventory, it is working to immediately build a small inventory buffer. “This buffer will allow a more predictable schedule for Cerezyme delivery through the remainder of 2010 and help avoid the challenges many physicians and patients have experienced in scheduling infusions,” the firm states.

Within the last two weeks Genzyme separately reported positive two-year data from a Phase II trial with its oral type I Gaucher disease candidate, eliglustat tartrate. In 2009, the company confirmed that the Phase II trial had met its primary composite endpoint of a clinically meaningful response in at least two of three endpoints following the initial 52-week study period. These included improvements in spleen size, hemoglobin levels, and platelet levels.

New two-year data from 22 patients continuing with eliglustat therapy showed that spleen volumes decreased from baseline by a mean of 52%, and liver volumes decreased from baseline by 24%. The study data also found hemoglobin levels increased from baseline by a mean of 2.1 grams per deciliter, and platelet counts increased from baseline by a mean of 81%. Additional data from a preplanned analysis suggested that eliglustat therapy over two years may also positively impact on indicators of bone disease, including bone mineral density.

Genzyme is currently enrolling patients into two Phase III trials with eliglustat. ENCORE, is a randomized, open-label study for adult patients with Gaucher disease type 1 and will compare eliglustat tartrate with Cerezyme in patients who have currently been treated using Cerezyme. The Phase III ENGAGE study will evaluate eliglustat in Gaucher disease patients who have received no treatment for their disease over the last 12 months.

Source: GEN News

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A California county has accused GlaxoSmithKline of falsely advertising its diabetes drug Avandia, aiming to recoup the cost of the drug for hospitals, clinics, and counties across the state. It appears to be the first government lawsuit over the diabetes med, which has been the subject of a debate over its potential links to heart attacks and strokes, Pharmalot notes.

The lawsuit estimates that Avandia may have caused 60,000 to 200,000 heart attacks, strokes and cardiovascular deaths in the U.S. from 1999 to 2006, according to the San Jose Mercury News. Over about the same period of time, Santa Clara County spent $2 million on the drug for indigent patients, and, the lawsuit alleges, absorbed the cost of treating heart patients whose CV troubles could have been avoided if the drugmaker had warned of the risks.

Experts have been jousting over Avandia for almost three years now, ever since a meta-analysis found a big increase in the risk of heart attacks in patients who used the diabetes med. The FDA had an expert advisory panel consider the evidence, and it concluded that while the drug did boost the risk of heart failure, the data on heart-attack and stroke risks was inconclusive.

With more data now available–and a U.S. Senate report accusing GSK of downplaying the drug’s risks–the agency is revisiting Avandia sometime this summer. Will more lawsuits hit the courts in the meantime?

Source: FiercePharma

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The FDA and the NIH say they’ll collaborate on a new program aimed at improving the regulatory science used to review the safety and efficacy of new drugs, promising that better science will lead to swifter decision-making.

“We have allowed the arm of regulatory science to become weak and underdeveloped,” FDA Commissioner Margaret Hamburg told reporters. If not fixed, “instead of pulling us into an exalted future, we will row in circles.”

To get the partnership up and running, the agencies agreed to form a committee of six top scientists drawn from both organizations. They will provide $6.75 million over three years to fund regulatory science research. President Obama’s proposed budget also includes $25 million to improve the science in use at government agencies. The regulators even announced a few priorities, like shortening the time it takes to test the potency of flu vaccines. Instead of three or four months, the agencies believe that wait can be halved.

HHS Secretary Kathleen Sebelius promised that “this is going to mean that new treatments are safer and available sooner.”

Anyone outside the vaccine field, though, isn’t likely to shorten their timelines on regulatory reviews anytime soon. If scientific committees and small budgets were all it took to transform the FDA’s review record, the average cost of development would be much smaller than it is today.

Source: FierceBiotech

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The backlog of generic-drug applications at FDA is growing. With increasing numbers of applications–and not enough staff to review them–the amount of time a potential generic waits for approval grew to 26.7 months in 2009, up from 16.3 months back in 2005. It’s an increase that Generic Pharmaceutical Association Chair and Watson Chief Executive Paul Bisaro calls “astonishing.”

There are 2,000 generic-drug apps awaiting action at FDA right now, the New York Times reports. The longer they languish without review, the longer Medicare and other payers have to spend more on brand-name drugs. That may be good for branded drugmakers, but not so good for fighting fast growth in healthcare costs. As Bisaro tells the Times, ”Those are direct tangible savings to consumers being lost.”

At GPhA’s recent annual meeting, FDA chief Margaret Hamburg promised to speed approvals along. Even generics makers agree that FDA’s generics office is underfunded, even if they might not agree with Hamburg’s solution to that problem: User fees. One way or the other, Hamburg says the agency will expedite things. As she told her audience at GPhA, “I don’t pretend to believe that the status quo is acceptable.”

Source: FiercePharma

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