Genzyme rival gets positive test results

British drug maker Shire PLC stepped up the pressure on Cambridge biotechnology company Genzyme Corp. yesterday, reporting positive test results for a competitor to Genzyme’s drug for Gaucher disease and disclosing that US regulators will let doctors prescribe the rival drug before it is approved for commercial use.

Supplies of Genzyme’s enzyme replacement therapy Cerezyme, currently the only drug on the market to treat the rare genetic disorder, were interrupted in June when a virus was detected in the company’s Allston Landing plant.

While the plant resumed production last week after the decontamination of its bioreactors, Genzyme, the state’s largest biotechnology company, will continue rationing drugs to the least vulnerable patients until late this year, when new batches of the drugs can be shipped and inventories return to their full strength.

Shire’s competing drug, velaglucerase alfa, could be administered to some Gaucher disease patients in a matter of weeks, following Food and Drug Administration approval of a company plan for introducing the drug, called a treatment protocol. The agency solicited the protocol, a relatively uncommon arrangement that allows doctors to prescribe drugs for up to 12 months before they have received FDA approval. A treatment protocol is typically reserved for situations such as supply disruptions, where there are acute medical needs.

The early rollout of Shire’s drug could give the company a leg up in its efforts to grab a share of the market for Gaucher disease treatments. But the company had planned to submit a new drug application to the FDA even before Genzyme’s supply interruption.

“We’re just responding to the patient need at this point, which is what matters most,’’ said Sylvie Gregoire, the president of Shire’s human genetic therapies division in Lexington, which produces velaglucerase alfa, and other enzyme replacement drugs. “That’s what the FDA wants. If this turns into an opportunity for us, that’s fine.’’

Genzyme officials declined to comment yesterday. The company’s shares shed 2.91 percent, or $1.51, to $50.38 on the Nasdaq exchange. It was the second consecutive session of retreat for Genzyme shares, pushing its stock down 10.8 percent since Thursday.

The first drop came Friday, when the company disclosed that federal in spectors will be paying another visit to the Brighton facility because of concerns that previously identified production problems there hadn’t been “fully or adequately’’ addressed by Genzyme.

Shares of Shire, meanwhile, edged up 0.65 percent, or 29 cents, to $45.10 in Nasdaq Composite trading yesterday.

Gaucher disease is an enzyme deficiency in which fatty substances accumulate in the spleen, liver, lungs, bone marrow, and sometimes, the brain, causing bruising, enlarged organs, and lung and kidney ailments. It affects only about 5,700 people worldwide. But the treatment for it, which is often subsidized by governments or insurance companies, costs approximately $200,000 per year per patient, bringing the potential market for drug makers to about $1.2 billion.

In addition to Shire, two other drug companies, Actelion Pharmaceuticals Ltd. of Switzerland, and Protalix BioTherapeutics Inc. of Israel, are developing drugs to treat Gaucher disease.

Genzyme is likely to keep the lion’s share of the market in the near future, but finds itself under increasing regulatory and competitive pressure, said Christopher J. Raymond, senior biotechnology analyst for financial services firm Robert W. Baird & Co. in Chicago.

“They’ve had a string of setbacks,’’ Raymond said of Genzyme, citing the FDA inspection and the intensifying competition. “The situation has deteriorated since we first learned about the problems in Allston. I still have the utmost confidence they will get these problems fixed, but there’s a risk that the FDA might not get back in the plant on a timely basis or might find something else that they don’t like.’’

On the competitive front, Shire, which has nearly 1,000 employees in Lexington and Cambridge, yesterday reported positive results from the first of three phase 3 studies of veraglucerase alfa.

The data were consistent with previous studies that helped convince the FDA to accept the company’s treatment protocol that will allow doctors to prescribe the drug before it wins final approval by the agency.

Shire said it began a “rolling submission’’ of its new drug application last Wednesday, three weeks after it received fast-track designation from the FDA. The designation enables companies to file sections of the application as they become available, and allows the agency to review those sections before the entire application is filed. Shire said it expects to complete the application by Sept. 30.

Source : www.boston.com

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FDA Approves New Drug Treatment for Type 2 Diabetes

The U.S. Food and Drug Administration today approved Onglyza (saxagliptin), a once-daily tablet to treat Type 2 diabetes in adults. The medication is intended to be used with diet and exercise to control high blood sugar levels.

The hormone insulin keeps blood sugar (glucose) levels within a narrow range in people who don’t have diabetes. People with Type 2 diabetes are either resistant to insulin or do not produce enough insulin to maintain normal blood sugar levels.

Onglyza is in a class of drugs known as dipeptidyl peptidase-4 (DPP-4) inhibitors which stimulate the pancreas to make more insulin after eating a meal.

“Keeping blood sugar levels in adequate control is essential to the good health of the 24 million people in the United States with Type 2 diabetes,” said Mary Parks, M.D., director of the Division of Metabolism and Endocrinology Products in the FDA’s Center for Drug Evaluation and Research. “High blood sugar levels can cause blurry vision and excessive urination and eventually result in such serious conditions as kidney and eye disease.”

The most common side effects observed with Onglyza are upper respiratory tract infection, urinary tract infection, and headache. Other side effects include allergic-like reactions such as rash and hives.

Approval of Onglyza was primarily based on the results of eight clinical trials. The application seeking FDA approval was submitted before December 2008 when the agency recommended that manufacturers of new diabetes drugs carefully design and evaluate their clinical trials for cardiovascular safety. Although Onglyza was not associated with an increased risk for cardiovascular events in patients who were mainly at low risk for these events, the FDA is requiring a postmarket study that will specifically evaluate cardiovascular safety in a higher risk population.

Onglyza is manufactured by Bristol-Myers Squibb Co. of Princeton, N.J., and marketed by Bristol-Myers and AstraZeneca Pharmaceuticals LP, of Wilmington, Del.

Source : www.fda.gov

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New biological therapy Ilaris® approved in US

The US Food and Drug Administration (FDA) has approved Ilaris® (canakinumab) for the treatment of children and adults with cryopyrin-associated periodic syndrome (CAPS), which includes a number of rare but life-long auto-inflammatory disorders with debilitating symptoms and limited treatment options. The FDA granted priority review to Ilaris based on its potential to meet an important clinical need for patients with CAPS.

Ilaris is the first approved treatment for patients as young as four years old suffering from two forms of CAPS: familial cold auto-inflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS).

CAPS is caused by a single gene mutation that leads to overproduction of interleukin-1 beta (IL-1), which causes sustained inflammation and tissue damage. Symptoms, such as debilitating fatigue, rash, fever, headaches, joint pain and conjunctivitis, can be present from birth or infancy, and can occur daily throughout patients' lives. Long-term consequences may be serious and potentially fatal, including deafness, bone and joint deformities, central nervous system damage leading to visual loss, and amyloidosis resulting in renal failure and early death.

Ilaris, previously known as ACZ885, is a fully human monoclonal antibody that rapidly and selectively blocks IL-1. The dosing schedule for Ilaris is once every eight weeks, which is less frequent than the current approved therapy. Also, more than 90% of patients studied did not experience any injection site reactions and those that did occur were of a mild-to-moderate nature.

"Until now, treatments for CAPS patients have been limited to traditional inflammatory-disease medications that work by suppressing the entire immune system, and newer therapies that control the disease better but require more frequent injections," said Hal Hoffman, MD, Associate Professor of Pediatrics and Medicine at University of California, San Diego, USA.

He added: "CAPS is a life-long disease and the convenience of administration of medicine is of the utmost importance for these patients. With rapid and sustained response, good tolerability, and a significantly less-frequent dosing schedule, Ilaris represents an important treatment advance for children and adults with CAPS."

CAPS comprises three disorders of increasing severity: FCAS, MWS and neonatal-onset multisystem inflammatory disease (NOMID). There are believed to be approximately 300 cases in the US, but many patients may remain undiagnosed due to poor disease recognition. A clinical study is ongoing to evaluate the potential of Ilaris to treat patients with NOMID. There are currently no approved therapies for the treatment of NOMID.

"Children and adults affected by these inflammatory diseases have to cope daily with distressing and debilitating symptoms," said Trevor Mundel, MD, Head of Global Development at Novartis Pharma AG. "We are excited about this first Ilaris approval which reflects our commitment to developing innovative treatments that address unmet medical needs, regardless of the size of the patient population."

In addition to ongoing studies in CAPS, clinical trials are also under way with Ilaris in systemic juvenile idiopathic arthritis (SJIA), and more common disorders such as some forms of gout, chronic obstructive pulmonary disorder (COPD) and type 2 diabetes. Further development in rheumatoid arthritis (RA) is not planned.

The approval of Ilaris is based on a three-part, one-year Phase III study involving 35 patients aged nine to 74 years old with varying degrees of disease severity. Results published in The New England Journal of Medicine on June 4, 2009 show that Ilaris produced a rapid, complete and sustained response in the majority of patients.

Part two of the study included the primary endpoint, a comparison between the number of patients treated every two months with Ilaris who experienced disease outbreaks or 'flares' vs. those on placebo. Results showed that none of the patients in the Ilaris group (0 out of 15) experienced a disease flare compared to 13 out of 16 patients in the placebo group (0% vs. 81% respectively, p<0.001).>

Source : www.worldpharmanews.com

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How long will FDA keep investors and chronic fatigue syndrome patients waiting for Ampligen decision?

Investors and CFS patients eagerly anticipating approval from the FDA on Hemispherx Biopharma's Ampligen may have to wait as much as 1-2 additional weeks for a decision according to the company's latest news release and Form 8K filing.

Trading on Tuesday sent shares up over 25% and volume was up over 4 times the daily average on the news as investors interpreted the development as a positive sign that approval for the drug may be coming.

Hemispherx Biopharma, Inc. , on Tuesday, announced that the U.S. Food and Drug administration (“FDA”) has advised the company that it may require up to 1-2 additional weeks to take action beyond the scheduled Prescription Drug User Fee Act action date of May 25, 2009 on the New Drug Application for Ampligen® (Poly I Poly C12U), a selective TLR3 modulator, for the management of Chronic Fatigue Syndrome. Reason for the possible delay was attributed by the Agency to certain staff scheduling changes which might (or might not) delay the report. Accordingly the Company’s development plan for Ampligen® continues as described in the recently filed 10Q and 10K, as the FDA did not request additional information from the Company at this time.

The delay has nothing to do with HEB or Ampligen and everything to do with the FDA, according to those familiar with the situation. In addition, the FDA did not request additional information.

Adrienne Dellwo, who serves as About.com's Guide to Fibromyalgia and Chronic Fatigue Syndrome had this to say to BioMedReports on the topic of Ampligen's approval:

"Of course, I hope that Ampligen will help a lot of people regain functionality and quality of life. With any new drug, however, it takes time to figure out just how effective it's going to be, and for how many people. On one hand, Ampligen's long, strange history casts some doubt on its effectiveness, but on the other hand some very prominent people in the CFS community have used it and believe it will change a lot of lives.

"The bottom line is that people with CFS need more treatment options, and studies show Ampligen may be a very good option, especially for certain CFS sub-groups. For example, it could work wonders for someone with a post-viral case, but not for those who developed it after exposure to toxins. I hope this is the treatment for which millions of people have waited."

Some are speculating that the delay may be, at least in part, due to the new appointment (confirmed by the Senate last week) of Dr. Margaret A. Hamburg, as the new commissioner of the Food and Drug Administration.

The Wall Street Journal has posted a story titled: "New FDA chief must divest several stock, fund holdings." In it, the Journal states that Margaret Hamburg and her husband, Peter Fitzhugh Brown, must divest themselves of several hedge-fund holdings as well as some of Mr. Brown's inherited drug-company stocks so Dr. Hamburg can take the post as the nation's top food and drug regulator.
Interestingly, a hedge-fund sponsor Renaissance Technologies, where Mr. Brown works owned 34,500 shares in HEB and added 23,000 more recently according to the latest Mutual Fund Facts report.

Hamburg is a former New York City health commissioner who also is a public health and bioterrorism expert. Hamburg told senators at her confirmation hearing that she wants to restore public confidence in the FDA by putting science first and by running an open and accountable operation. The FDA had been under criticism recently for its handling of several issues related to food and pharmaceutical products.

The company says it's development plan for Ampligen continues as described in their recently filed 10q and 10k.

Source : www.officialwire.com

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