The FDA approves new drug for gout

The Food and Drug Administration said Tuesday that it has approved a new drug to treat gout in patients who do not respond to existing therapy. The drug is called Krystexxa and is manufactured by Savient Pharmaceuticals Inc. of East Brunswick, N.J. The FDA had rejected the drug in August of 2009 because of manufacturing problems.

In a letter to the company then, the agency said that the potential commercial supplies of the drug were not identical to the product used in clinical trials.

Gout occurs because of an excess of the metabolic waste product uric acid, which precipitates out of the bloodstream and into joints and soft tissue as needle-like crystals that can be very painful. They also cause intermittent swelling, redness, heat, pain and stiffness in the joints. Although most people tend to associate the disorder with rich foods, it is more commonly linked to obesity, high blood pressure, high cholesterol and diabetes. It is most common in men, post-menopausal women and people with kidney disease.

The conventional treatments include the drugs allopurinol and febuxostat, both of which inhibit the enzyme xanthine oxidase, which produces uric acid from DNA in food. Krystexxa, known generically as pegloticase, is an enzyme that acts directly on uric acid, converting it into harmless chemicals that are excreted in the urine. The drug is given by an intravenous infusion every two weeks.

"About 3% of the 3 million [American] adults who suffer from gout are not helped by conventional therapy," Dr. Badrul Chowdhury, director of the FDA's division of pulmonary, allergy and rheumatology products, said in a statement. "This drug offers an important new option for them."

But the agency cautioned that about 1 in every 4 patients receiving an infusion of the drug suffers a severe allergic reaction and urged physicians to give patients a corticosteroid and an antihistamine before infusing the drug.

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New emergency contraceptive approved by FDA

The newest form of emergency contraceptive, ellaOne, was approved August 13 by the U.S. Food and Drug Administration.

Although the new drug has been around since fall of 2009 in select European countries, it was not introduced to the United States until June 2010.

The new drug is a type of emergency contraceptive pill that allows a woman up to five days after sexual intercourse to consume the pill.

The more popular contraceptive pill, Plan B One-Step, also known as the morning-after pill, offers only three days.

"The great thing about ellaOne is the efficacy lasts for all five days," said Morgan Molnar, '10, a marketing major pursuing her masters in sociology, who attended part of the first FDA hearing in June about ellaOne.

"The effects of the pill will be the same if you take it on the first day or the last day," she said. "If you take Plan B One-Step the day after unprotected sex, you will have a better chance of not getting pregnant than if you took the pill three days later. EllaOne lasts all five days and your chances don't decrease."

However, like most new drugs, long-term effects are unknown for ellaOne.

"As far as my opinion, it's a great drug, but my only concern is that it is so new," Molnar said. "They haven't done too many long-term studies, so in the case that women have become pregnant and have taken the drug, they don't know what will happen to the unborn fetus."

"Plan B One-Step doesn't have a lot of effects on the fetus, but ellaOne is so new that they are unsure of its effects in the long run," she said.

Susan Kitei, director of the Health Center, said she is not ready to endorse ellaOne because significant problems with a new medication are not always apparent until it has been widely used for two to five years.

"This is why, occasionally, we will hear on the news that a drug is being removed from the market," she said. "EllaOne has been used in Europe only since 2009, so it is wisest to wait a while to make absolutely certain it is as safe as other medications used for emergency contraception. The best alternative, Plan B, has been in wide usage for a number of years and is known to be safe and effective."

Kitei also said Plan B is available without a prescription for those 17 years and older.

The new drug will be prescription-only, and how its cost will compare with Plan B is still unknown.

"Since the decision of taking emergency contraception is such a big choice for women, this now gives them more time to make a good decision of whether or not they want to pursue with emergency contraception," Corry Starr, '13, said. "However," she said, "since ellaOne has to be prescribed, I don't think girls will want to use it as often as Plan B due to confidentiality."

"Based on what I saw, it looks like ellaOne is more effective than Plan B," Rita Jones, director of the Women's Center, said.

"It's advantageous because it offers women five days opposed to three days," she said.

"However, women should still think about securing emergency contraception as soon as possible. Just because they now have three to five days, doesn't mean they should simply wait around."

Karen Hicks, an adjunct professor of women's studies and sociology, also attended part of the first FDA hearing and recommended getting as much information as possible before using the medication.

"Time will tell how popular ellaOne will be in the U.S.," Kitei said. "The more educated a woman is, the less likely she is to need it. Because most of our students are aware of Plan B, I am expecting only rare requests."

Jones considered a separate advantage of the new drug and said the availability of the new drug is a benefit in cases of incest or sexual assault.

"In some incidents, the survivor might need a couple of days to process what happened," she said. "Five days provides more time for the survivor and helps them, as opposed to a smaller three-day window."

The price of ellaOne has yet to be determined in the United States but according to Molnar, it will be about three times the price of Plan B.

"I think the approval of ellaOne was definitely a step in the right direction toward giving women more choices," Molnar said.

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FDA approves new oral contraceptive

The U.S. Food and Drug Administration, FDA, has approved a new oral contraceptive for women called Natazia. The new drug contains two female hormones, estrogen and progestin.

Scott Monroe, M.D., director of FDA's Division of Reproductive and Urologic Products reports, “Nearly 12 million women in the United States and more than 100 million women worldwide currently use oral contraceptives. The approval of Natazia provides another option for women who choose to use an oral contraceptive as their method of contraception.”

The most common side effects include irregular bleeding, breast tenderness, headaches, nausea, vomiting, increased weight, and acne. Women over 35 years of age who smoke are being advised to not use Natazia. Evidence has shown that cigarette smoking increases the risk of serious cardiovascular events from combination oral contraceptive use.

In Michigan, over one million women are in need of contraceptive services and supplies. Over 213,000 of the over 2 million women of childbearing age become pregnant each year. Natazia becomes another choice women can make in contraception use.

Manufactured by Bayer Schering Pharma, Natazia is expected to be available by this summer in the U.S.

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FDA Approves Orphan Drug Status for Type 1 Diabetes Stem Cell Therapy

The US Food and Drug Administration (FDA) has approved orphan drug designation for a stem cell therapy in patients with newly diagnosed type 1 diabetes mellitus.

The product is an intravenous formulation of adult mesenchymal stem cells (MSCs) that are isolated from the bone marrow of healthy young adult donors, thereby avoiding the controversy associated with embryonic and fetal cell sources. Cell culture allows the large-scale production of thousands of doses from a single donation.

According to a company news release, MSCs are designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration, and preventing scar formation. Preclinical studies indicate that MSCs may delay the progression of type 1 diabetes by preserving beta cell function.

Treatment safety and efficacy are currently being evaluated in a 62-patient, double-blind, placebo-controlled phase 2 study conducted at 20 medical centers across the United States. To be eligible, patients must be aged 12 to 35 years and have been diagnosed 2 to 20 weeks before study entry.

The MSC therapy previously was granted FDA orphan drug status and expanded access approval for graft vs host disease, allowing its use in patients whose condition is life-threatening.

Other potential indications under investigation include Crohn's disease, myocardial infarction, and pulmonary disease.

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Abbott’s Creon Granted Approval For Supplemental New Drug Application - Update

Broad-based health care company Abbott Laboratories (ABT) announced Monday that the U.S. Food and Drug Administration approved its supplemental New Drug Application or sNDA for Creon to include dosing guidance in the prescribing information specific to patients with exocrine pancreatic insufficiency or EPI, a condition of limited production of pancreatic enzymes due to chronic pancreatitis or removal of the pancreas. Prior to this FDA approval, dosing guidance for medications such as Creon was based on patients with cystic fibrosis.

Creon (pancrelipase) delayed-release capsules now include dosing guidance in the prescribing information specific to patients with EPI due to CP or chronic pancreatitis or a pancreatectomy.

EPI is the inability to properly digest food due to a lack of digestive enzymes made by the pancreas. Patients with chronic pancreatitis or those who have had their pancreas removed develop EPI.

This insufficiency reduces the production or secretion of enzymes that are necessary to digest fats, proteins and carbohydrates in food and can result in a type of oily diarrhea known as steatorrhea, malabsorption of nutrients, weight loss, bloating, abdominal pain and even chronic malnutrition if left untreated.

EPI can occur as a complication of a variety of diseases or conditions, such as cystic fibrosis, chronic pancreatitis, pancreatic cancer and gastrointestinal surgery.

Pancrelipase is a form of enzyme therapy for the treatment of various digestive disorders. It is a mixture of three digestive enzymes, amylase, protease, and lipase, which are normally produced by the pancreas.

Abbott in its statement said the approval makes Creon the first medication in its class to have guidance and information for use in treating EPI due to CP and pancreatectomy.

Creon’s sNDA approval was based on results of a double-blind, randomized, placebo-controlled, two-arm, parallel-group study, enrolling 54 adults with EPI due to CP or pancreatectomy. The primary efficacy endpoint was to measure the amount of fat absorbed by the patient’s body rather than excreted.

In April 2009, Creon was approved as a Pancreatic Enzyme Replacement Therapy or PERT by the FDA. PERTs work in patients with EPI by delivering pancreatic enzymes to the small intestine to help break down fats, proteins and carbohydrates in food, thereby acting as a replacement for digestive enzymes physiologically secreted by the pancreas.

“Without proper dosing guidance, some patients have previously attempted to control EPI symptoms by modifying their diets and limiting fat intake. However, this approach has yielded limited treatment success,” said Orelle Jackson, executive director of the National Pancreas Foundation.

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FDA aprroves a new pain drug

Shares of Pozen Inc. surged more than 21% in the after hours trading session Friday after the U.S. Food and Drug Administration or FDA approved VIMOVO delayed-release tablets for the relief of signs and symptoms of osteoarthritis, rheumatoid arthritis, and ankylosing spondylitis, and to decrease the risk of developing gastric ulcers in patients at risk of developing NSAID-associated gastric ulcers.

VIMOVO, co-developed by Pozen and UK-based drug maker AstraZeneca Plc is a fixed-dose combination of enteric-coated naproxen, a pain-relieving non-steroidal anti-inflammatory drug or NSAID, and immediate-release esomeprazole, a proton pump inhibitor or PPI.

The FDA approval was supported by data from a clinical development program, including results from the pivotal PN400-301 and PN400-302 studies, which showed patients taking VIMOVO experienced significantly fewer endoscopic gastric ulcers, compared to patients receiving enteric-coated naproxen.

Nearly 27 million US residents and 140 million people worldwide suffer from osteoarthritis, which is the most common form of arthritis. According to the company, half of osteoarthritis patients on chronic non-steroidal anti-inflammatory drugs therapy are at the risk of developing gastric ulcers associated with it.

Howard Hutchinson, M.D., Chief Medical Officer, AstraZeneca, said, “In a single pill, VIMOVO provides a proven pain reliever with a built-in PPI for arthritis patients at-risk for NSAID-associated gastric ulcers.”

In the PN400-301 and 302 studies, the primary end point was the cumulative incidence of gastric ulcers through six months. Patients received either VIMOVO or enteric-coated naproxen 500 mg, twice daily, in each of the trial, over a six-month treatment period.

Data from study PN400-301 showed a 4.1% incidence of gastric ulcers in patients taking VIMOVO, compared to 23.1% among patients taking enteric-coated naproxen. Study PN400-302 showed a 7.1% incidence of gastric ulcers among patients taking VIMOVO, compared to 24.3% with enteric-coated naproxen.

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Dendreon’s Provenge Approval Sparks New Era for Cancer Vaccines

Dendreon Corp. won approval for its only product, the prostate cancer vaccine Provenge, concluding a three-year battle with U.S. regulators to introduce the first therapy to train the body’s immune system to destroy tumors.

Provenge may reap annual sales of $4.3 billion by 2020, said George Farmer, a Canaccord Adams Inc. analyst in New York. Its clearance will also benefit more than a dozen other companies developing drugs in the emerging field of cancer immunotherapy, said Joseph Pantginis, an analyst with Roth Capital Partners in New York.

“This approval represents the first cancer immunotherapy product approved in the U.S. and is truly a landmark event,” Pantginis said in a research report yesterday.

Provenge, cleared yesterday for use against advanced prostate tumors, will initially be available at about 50 sites used for clinical trials, and more widely distributed after four plants are cleared for use by mid-2011, Seattle-based Dendreon said in a statement. It will cost $93,000 for a typical three- dose course of treatment, the company said.

Food and Drug Administration approval of Provenge “will definitely inform the clinical development programs of other companies” working on vaccine treatments for cancer, said Janice Reichert, a senior research fellow at the Tufts Center for the Study of Drug Development in Boston, in a telephone interview before the decision was announced.

The most advanced of these vaccines include Seattle-based Oncothyreon Inc. and German drugmaker Merck KGaA’s Stimuvax for breast and lung malignancies; ipilimumab from Bristol-Myers Squibb Co. of New York, for melanoma; and British drugmaker Oxford BioMedica Plc’s TroVax for prostate and kidney cancer.

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New Drug-Interaction Warnings for All HIV Protease Inhibitors

Package inserts for all of the approved protease inhibitors (PIs) used to treat HIV have been updated to reflect important drug-drug interactions, according to an April 27 e-mail announcement from the U.S. Food and Drug Administration.

Drug-drug interactions are a common obstacle among people living with HIV receiving antiretroviral therapy. This is especially true for those using PIs, widely known for their ability to compete with other medications for enzymes that regulate drug levels in the body. This can elevate blood levels of one or more drug, ultimately increasing the risk of side effects; it can also reduce blood levels of medications and reduce the effectiveness of treatment.

Among the important interactions to be added to all PI package inserts include those involving Revatio (slidenafil), Uroxatral (alfuzosin), Advair and Serevent (both contain salmeterol), Tracleer (bosentan), Adcirca (tadalafil) and colchicine.

Revatio, a version of the erectile dysfunction medication sildenafil, is prescribed to treat pulmonary arterial hypertension. It is contraindicated among people living with HIV using PIs, meaning that the drug should not be used under any circumstances.

Tracleer and Adcirca are also used to treat pulmonary arterial hypertension and should be used cautiously. According to the FDA, their doses may need to be adjusted when combined with protease inhibitors. The agency also warns against using Tracleer with the PI Reyataz (atazanavir) if a Norvir (ritonavir) booster is not also included.

Advair and Serevent are frequently prescribed to help manage symptoms of asthma, chronic obstructive pulmonary disease (COPD) and sometimes seasonal allergies. They should not be combined with protease inhibitors.

Uroxatral, used to treat benign prostatic hyperplasia (BPH) in men, is also contraindicated among people using HIV protease inhibitors.

Colchicine, a natural produce derived from the Colchicum genus of plant, is used to treat gout and familial Mediterranean fever. Depending on the PI used, dose adjustments may be necessary. Additionally, colchicine should be avoided among those who take PIs and have a history of either liver (hepatic) or kidney (renal) impairment.

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Children overdosed during Pfizer drug study

In the article titled “Pharmaceutical injury alert: FDA slams Pfizer with warnings for deadly clinical trials,” posted on April 23, 2010, it was incorrectly reported that participants of a drug trial died. Twenty-six pediatric patients received overdoses of a psychiatric drug in 2006, and three more overdoses occurred in 2007; the overdoses did not result in any fatalities.

The U.S. Food and Drug Administration (FDA) issued Pfizer a warning letter, stating they have failed to rectify its testing procedures, which resulted in overdoses in over two-dozen patients during a company trial of Geodon. Several children experienced tremors, restless legs and other complications as a result of the overdoses, as reported by Reuters.

The letter, which was released on Tuesday, April 20, 2010, states that Pfizer was not properly monitoring physicians who were testing the experimental medication. Pfizer said in a statement that only 10 of the overdoses were caused by physician error, and the rest were probably because of patient error.

A new warning letter was prompted after a July 2009 inspection found that the drug maker was still failing to follow its own rules for safely conducting a study. A Pfizer spokeswoman pointed out, “Pfizer has communicated with the FDA about our conduct of clinical trials and, over the next two weeks, will provide an outline of new and existing processes for preventing similar issues with Pfizer clinical trials.”

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NexMed Receives FDA Clearance for PrevOnco Phase 2 Study as First-Line Therapy for HCC

NexMed, Inc. , a specialty CRO with a pipeline of products based on the NexACT technology, today announced that the U.S. Food & Drug Administration (FDA) has cleared the Company to proceed with the proposed Phase 2 trial of PrevOnco , its proprietary cancer treatment for patients with advanced, unresectable hepatocellular carcinoma (HCC), or liver cancer.

The FDA granted PrevOnco orphan drug status in August 2008, and in March 2010, NexMed filed its Investigational New Drug application for the product candidate.

The Company also noted that in IND review communication, the FDA has given NexMed the opportunity to move PrevOnco directly into a Phase 3 trial that would support marketing approval, subject to positive study results. In order to pursue this regulatory path, NexMed would need to expand the proposed Phase 2 study design to use PrevOnco in combination with Doxorubicin as a second-line therapy for patients who have failed NEXAVAR , the currently marketed first-line anticancer treatment for patients with either HCC or advanced renal cell carcinoma (cancer of the kidney).

PrevOnco incorporates lansoprazole, which is the generic anti-ulcer compound approved under the name Prevacid(R) and marketed in the U.S. by Takeda Pharmaceuticals North America, Inc. In vitro and in vivo data generated to date has demonstrated the ability of lansoprazole to inhibit tumor cell growth and enhance survival in mouse models of cancer alone, and in combination with Doxorubicin.

Commenting on today's news, Dr. Bassam Damaj, President and Chief Executive Officer of NexMed, stated, "We are very pleased that the FDA agreed with our protocol for the HCC Phase 2 trial for PrevOnco as a first-line therapy for HCC. Additionally, we are actively assessing the suggestion made by the FDA to move directly into a Phase 3 trial, by studying PrevOnco in combination with Doxorubicin as a second-line therapy for patients who have failed NEXAVAR therapy. Following this path could be very advantageous for NexMed since advancing the drug directly into a Phase 3 study would save us at least 12-24 months in development time."

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A New Drug Approved By The FDA For The Treatment Of Fibromyalgia Pain

NMS Labs launches another unique test suitable for the evaluation of toxicity vs. efficacy, the potential for physicians to establish therapeutic drug monitoring (TDM) of individual patients, and the assessment of drug diversion of a new pain management drug therapy.

NMS Labs is the only known commercial laboratory to develop and perform a clinical quantitative assay for Milnacipran (Savella®) using the highly specific and sensitive technique of liquid chromatography/tandem mass spectrometry (LC-MS/MS).

Eric Rieders, PhD, President and CEO expressed the importance of innovation at NMS Labs by saying, “We best serve the medical community by deploying our professional and technical resources to rapidly identify and develop diagnostic tests within our area of expertise that might otherwise only become available at a much later date.” Dr. Rieders added, “NMS Labs is well known for providing the rare and hard to find tests that other labs do not.”

NMS Labs has responded to the need of the pain management community by filling a void in testing options. Milnacipran is a new medication used for the treatment of pain associated with fibromyalgia. NMS Labs specializes in testing for various classes of pharmacological agents, including: analgesics; anesthetics; anticonvulsants; antidepressants; hallucinogens; muscle relaxants; NSAIDs; opioids; sedatives and hypnotics; sleep aids; and stimulants.

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FDA approved leukemia drugs shows promise in ovarian cancer cells

The drug Sprycel, approved for use by the U.S. Food and Drug Administration in patients with chronic myeloid leukemia, significantly inhibited the growth and invasiveness of ovarian cancer cells and also promoted their death, a study by researchers with UCLA's Jonsson Comprehensive Cancer Center found.

The drug, when paired with a chemotherapy regimen, was even more effective in fighting ovarian cancer in cell lines in which signaling of the Src family kinases, associated with the deadly disease, is activated.

The study appears in the Nov. 10, 2009 edition of the British Medical Journal.

Ovarian cancer, which will strike 21,600 women this year and kill 15,500, causes more deaths than any other cancer of the female reproductive system. Few effective therapies for ovarian cancer exist, so it would be advantageous for patients if a new drug could be found that fights the cancer, said Gottfried Konecny, an assistant professor of hematology/oncology, a Jonsson Cancer Center researcher and first author of the study.

"I think Sprycel could be a potential additional drug for treating patients with Src dependent ovarian cancer," Konecny said. "It is important to remember that this work is only on cancer cell lines, but it is significant enough that it should be used to justify clinical trials to confirm that women with this type of ovarian cancer could benefit."

Recent gene expression studies have shown that about one-third of women have ovarian cancers with activated Src pathways, so the drug could potentially help 7,000 ovarian cancer patients every year.

In this study, the UCLA team tested the drug against 34 ovarian cancer cell lines and they conducted genetic analysis on all cell lines. Through these analyses, the researchers were able to identify genes that predict response to Sprycel. If the work is confirmed in human studies, it may be possible to test patients for Src activation and select those who would respond prior to treatment, personalizing their care.

"We were able to identify markers in the pre-clinical setting that would allow us to predict response to Sprycel," Konecny said. "These may help us in future clinical trials in selecting patients for studies of the drug."

Sprycel is what is known as a "dirty" kinase inhibitor, meaning it inhibits more than one pathway. Konecny said it also inhibits the focal adhesion kinase and ephrin receptor, also associated with ovarian cancer.

The next step, Konecny said, would be to test the drug on women with ovarian cancer in a clinical trial. The tissue of responders would then be analyzed to determine if the Src and other pathways were activated. If that is confirmed, it would further prove that Sprycel could be used to fight ovarian cancer. In studies, women would be screened before entering a trial and only those with Src dependent cancers could be enrolled to provide further evidence, Konecny said, much like the studies of the molecularly targeted breast cancer drug Herceptin enrolled only women who had HER-2 positive disease.

"Herceptin is different because we knew in advance that the only worked in women with HER-2 amplification," he said. "In this case, we don't clearly know that yet. The data reassure us that the drug works where the targets are over-expressed but we need more testing to confirm this."
The tests combining the drug with chemotherapy are significant because chemotherapy currently is the first line treatment for ovarian cancer patients following surgery. Because Sprycel proved to have a synergistic effect when combined with chemotherapy both made the other work better it may be possible to add the targeted therapy as a first line treatment if its efficacy is confirmed in future studies, adding a new tool to an oncologist's arsenal.

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FDA Approves New Drug to Treat Psoriasis

The U.S. Food and Drug Administration approved Stelara (ustekinumab), a biologic product for adults who have a moderate to severe form of psoriasis.

Ustekinumab is the first in a new class of biologics, for psoriasis. This new human interleukin-12 and -23 antagonist is indicated for the treatment of adult patients (18 years or older) with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy. Ustekinumab treats psoriasis by blocking the action of two proteins that contribute to the overproduction of skin cells and inflammation.

Ustekinumab is administered subcutaneously as a weight-based dose. For patients weighing less than 100 kg (220 lbs), the recommended dose is 45 mg initially and four weeks later, followed by 45 mg every 12 weeks. For patients weighing >100 kg (220 lbs), the recommended dose is 90 mg initially and 4 weeks later, followed by 90 mg every 12 weeks. Nurses should use caution when drawing up and administering the drug to avoid dosing errors as it is available and packaged in two different dosage strengths: 45 mg/0.5 mL in a single-use glass vial and 90 mg/1 mL in a single-use glass vial.

The most common adverse reactions reported include nasopharyngitis, upper respiratory tract infection, headache, and fatigue. Adverse drug reactions may also occur at the site of administration including cellulitis, pain, swelling, pruritus, induration, hemorrhage, bruising, and irritation.

Because ustekinumab reduces the immune system’s ability to fight infections, it poses a risk of infection. Serious infections have been reported with ustekinumab, some leading to hospitalization. These infections were caused by viruses, fungi, or bacteria that have spread throughout the body. There may also be an increased risk of developing cancer, and ongoing studies are evaluating this risk. Before initiation of therapy, patients should be evaluated for tuberculosis and, if indicated, treated with antitubercular drug therapy before initiating ustekinumab therapy.

Ustekinumab therapy complicates vaccination administration. Non-live vaccines may be ineffective when administered during treatment. Patients receiving ustekinumab therapy must not, however, receive any live vaccines, nor should their household contacts because of the potential risk for shedding from the household contact and transmission to patient.

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Geron, FDA agree on spinal injury drug trial

Stem cell research company Geron Corp said it reached an agreement with U.S. health regulators which may enable it to restart the early stage trials of its cell therapy to treat complete thoracic spinal cord injury.

In August, the U.S. Food and Drug Administration (FDA) placed a clinical hold on Geron's investigational new drug application after some of the animals developed cysts in the injury site.

While the early stage trial still remains on clinical hold, a recent agreement with the FDA outlines what is necessary to move the spinal cord injury program forward, Merriman Curhan Ford analyst Joe Pantginis said in a note to clients.

Geron, which expects to re-initiate the early stage trial in the third quarter of 2010, said the FDA has advised that positive data from an ongoing preclinical study using its product can be used to support both release of the clinical hold and the drug's expansion to cervical patients.

However, analyst Pantginis said, "A great amount of work still needs to be done before any patient is ultimately enrolled into the study."

Currently Geron is developing three different drugs in the field of oncology, including cancer vaccine Grnvac1, which is in a mid-stage trial, and cancer drug Grn163l, which is in an early-stage trial.

"While volatility is still expected from the stem cell platform at Geron, we believe the increased visibility and data flow from the telomerase program (Grn163l) should drive the stock going forward," Pantginis said.

Investors are expecting Geron to report Grn163l's safety and dosing data at a Nov. 15 conference.

Shares of the company, which reported narrower-than-expected quarterly loss on Thursday, touched a high of $6.49, before paring some gains to trade up 5 percent at $6.07 Friday afternoon on Nasdaq.

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FDA concerns remain over new use for Schering drug

The U.S. Food and Drug Administration has "outstanding concerns" about Schering-Plough Corp's bid to market its drug PegIntron to treat certain skin cancer patients who also undergo surgery, the drugmaker said on Friday.

The company is seeking FDA permission to sell the injectable drug for melanoma. But the agency declined against approving the new use, at least right now, instead issuing a complete response letter about remaining issues.

"Schering-Plough will work closely with FDA to respond to outstanding concerns related to the PegIntron melanoma filing," the company said in a statement.

Schering did not say what the FDA's concerns were and company spokeswoman Mary-Francis Faraji said the drugmaker had no additional comment.

PegIntron, a type of alpha interferon protein-based drug that is self-injected once-a-week, is already approved for liver disease. The new use would allow it for patients whose melanoma has spread to the lymph nodes and require surgery to remove the cancer and the nodes.

Earlier this month, an FDA panel of outside experts narrowly recommended approval of the drug despite its toxic side effects. Some panelists as well as FDA staff reviewers also questioned whether the drug actually helped people live longer or just increased the time before cancer recurred.

Side effects with PegIntron include heart problems and depression.

Schering's Intron A, which is already approved for melanoma after surgery, has the same active ingredient as PegIntron but must be given intravenously five times a week for four weeks at a doctor's office.

Shares of Schering were off 23 cents, or less than one percent, at $28.20 on the New York Stock Exchange.

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FDA warns Procter & Gamble over Vicks products with vitamin C

The U.S. Food and Drug Administration sent a warning letter Friday to Procter & Gamble Co. saying the company was illegally marketing two Vicks cold and flu medicines containing vitamin C.

The FDA said the single-dose combinations of drug and dietary ingredients in Vicks DayQuil Plus Vitamin C and Vicks Nyquil Plus Vitamin C cannot be marketed legally because they have not been proven safe and effective.

The agency also said it had previously determined that there was insufficient data to show vitamin C is safe and effective in preventing or treating the common cold.

The FDA said it allows some over-the-counter drugs to be marketed without agency approval, but that the two Vicks products did not comply with its rules and first must be evaluated and approved under the FDA's new drug approval process.

A spokeswoman for the Cincinnati consumer products company said in an e-mail message that the FDA warning was "not new news" and was similar to a letter earlier this month that the FDA indicated had been posted to its Web site due to an internal error.

"It's also not about the safety or quality of the Vicks NyQuil or DayQuil brands," wrote Kate DiCarlo. "We believe we are marketing within FDA guidelines and regulations, and we'll continue to work closely with the FDA to resolve any concerns."

Shares of P&G, the world's largest consumer products maker, slid $1.46, or 2.5 percent, to $58.08 in afternoon trading.

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Amylin says Byetta gets FDA approval for new use

Amylin Pharmaceuticals Inc said on Friday that its Byetta drug has won U.S. regulatory approval for use as a stand-alone medication to improve glycemic control in adults with type 2 diabetes.

Previously, it only had U.S. Food and Drug Administration approval for use in patients who were also taking other common diabetes medications and had not achieved adequate glycemic control.

Byetta has been used by more than one million patients since its 2005 market introduction, Amylin said in the statement.

Byetta, an injected drug, has annual sales of about $700 million. It was the first approved drug from the GLP-1 analog class of Type 2 diabetes medicines that work by stimulating release of insulin only when blood sugar levels are high.

The drug is co-marked with Eli Lilly and Co .

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Spectrum says FDA refuses to OK new plea on cancer drug

Spectrum Pharmaceuticals said U.S. health regulators declined to approve its supplementary marketing application for its advanced metastatic colorectal cancer drug Fusilev in its current form, sending its shares down 22 percent.

Spectrum said the U.S. Food and Drug Administration (FDA) noted that the submission did not show that Fusilev is non-inferior to standard treatment leucovorin, and it did not request any changes to the currently approved indications.

Spectrum, which received a label expansion approval for another cancer drug last month, said it plans to request a meeting with the FDA to discuss options for the approval of Fusilev.

"There is going to be a delay (in Fusilev approval) and it wasn't really expected," said Morgan Joseph & Co analyst Shiv Kapoor, who in September downgraded Spectrum shares to "hold" from "buy."

Kapoor said he would wait for Spectrum to meet the FDA and determine the next step needed for the approval of the drug.

"The question really here would be, will this be a meaningful delay or not," he said.

Fusilev is currently approved by the FDA as a rescue after high dose of methotrexate therapy in patients with osteosarcoma, a type of bone cancer.

The drug is also indicated to diminish the toxicity and counteract the effects of impaired methotrexate elimination and of inadvertent overdosage of folic acid antagonist, a type of chemotherapy.

Spectrum shares were down 17 percent at $5.17 in late morning trade on Friday. They touched a low of $4.82 earlier in the session, making them one of the top percentage losers on Nasdaq.

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Acorda’s MS Drug May Need More Study

Acorda Therapeutics Inc.’s famprdine- SR, the first pill to improve walking in multiple sclerosis patients, may not be “clinically meaningful,” U.S. regulators said. The shares fell the most ever in Nasdaq trading.

Food and Drug Administration reviewers said they had doubts about the trial design for the drug, fampridine-SR, in a report posted today on the agency’s Web site. An FDA advisory panel will meet Oct. 14 in Adelphi, Maryland, to discuss whether the drug works well enough to justify an increased risk of seizures.

Fampridine, designed to restore nerve signals, was tested in 25-foot timed walks, a novel way to measure effectiveness. Older drugs are injected to reduce relapses and slow progression of the disease. MS damages the brain and spinal cord, affecting mobility, according to the National Institutes of Health.

The improvement patients saw with fampridine “was numerically quite small, and the average time to complete the 25-foot walk was not different between the treatment groups in either study,” said Eric Bastings, deputy director of the FDA’s Division of Neurology Products, in a memo to the panel. “For these reasons, it appears that the clinical meaning of the differences seen on the primary outcomes is in question.”

Shares Fall

Acorda, of Hawthorne, New York, dropped $4.76, or 21 percent, to $17.52 at 4 p.m. New York time in Nasdaq Stock Market composite trading. It was the biggest percentage decline since the drugmaker’s initial public offering in February 2006.

“Fampridine’s panel on Wednesday is going to be somewhat more contentious than we had anticipated,” said Phil Nadeau, a Cowen & Co. analyst in New York, in a note to clients today. “It would seem from the documents that the FDA may be moving the goal posts on Acorda” with new hurdles for proving the drug works, he said.

Acorda spokesman Jeff Macdonald declined to comment on the FDA report in a telephone interview today. The company will comment after the Oct. 14 FDA panel meeting, he said.

“How relevant is walking speed to MS?” said Mike King, an analyst for Merriman Curhan Ford & Co., in a telephone interview. “It’s risk versus benefit for the FDA, and this is a small benefit in a minority of patients, and a real risk of seizure. How do you balance those questions?”

Acorda is racing European drugmakers Merck KGaA and Novartis AG to introduce the first pill for MS. Current treatments, led by Teva Pharmaceutical Industries Ltd.’s Copaxone and Biogen Idec Inc.’s Avonex, are shots that reduce relapses and prevent symptoms of the disease from getting worse.

Damage to Myelin

MS strikes more than 2.5 million people worldwide and is caused by damage to myelin, the protective sheath around nerve cells in the brain and spinal cord, according to the National Multiple Sclerosis Society. MS worsens as damage to myelin causes muscle weakness, trouble with coordination and critical thinking, and memory loss. Women are at least twice as likely as men to get the disease.

“There might be someone using a walker or cane who finds it difficult to get from the kitchen to the bedroom, and fampridine could make it easier,” said Howard Weiner, director of the Partners Multiple Sclerosis Center at Brigham & Women’s Hospital in Boston, in a phone interview before the FDA documents were released.

A timed walking test is a logical way to measure the effectiveness of fampridine because there is a “physiologic” connection between the walking ability and the progression of the disease, Weiner said.

Priority Review

The FDA said on May 6 it would review fampridine under an expedited “priority review” program to speed clearance of novel medicines for serious illnesses. A drug awarded priority review is supposed to be decided on within six months, four months quicker than the review process for most medicines. Acorda in May said it anticipated an approval decision from the agency by Oct. 22.

Ron Cohen, Acorda’s founder and chief executive officer, said he didn’t ask for a quick review of fampridine. Transparency is critical for investors in the unprofitable biotech company, and Cohen said he was worried the application might get held up if it was taken out of normal review channels.

“To the extent that there are delays, or there are uncertainties in the process, that’s not a good thing because the last thing an investor wants to see is uncertainty,” Cohen said in an Aug. 18 phone interview.

Decades of Development

Development of fampridine was started by Dublin-based Elan Corp. in the 1990s and later licensed by Acorda. The drug is a sustained-release form of 4-aminopyridine, which some MS patients now get from compounding pharmacies with a doctor’s prescription. These medicines are custom made for patients and overdoses can cause seizures and other side effects.

Fampridine helped 35 percent of MS patients walk faster in a study whose results were reported in February. The drug’s ability to restore nerve signals after myelin is lost suggests it may also reduce other symptoms of the disease, regardless of what other medicines patients are taking, said John Richert, executive vice president of research and clinical programs for the New York-based National Multiple Sclerosis Society.

“Potentially, it could be tried in the large majority of people with MS,” Richert said in an Oct. 7 phone interview. “It has at least the theoretic potential for being able to help with virtually any MS symptom.”

Elan will manufacture fampridine if it is approved and receive royalties on sales. The drug is the first that Acorda has submitted to the FDA. The company also sells Zanaflex capsules, a treatment for spastic muscles that it acquired from Elan in 2004.

Biogen Partnership

Cambridge, Massachusetts-based Biogen agreed in July to pay as much as $510 million for rights to market fampridine outside the U.S. after Acorda said it wouldn’t be able to operate beyond next year without a partner. Naomi Aoki, a spokeswoman for Biogen, didn’t immediately return a call seeking comment.

German drugmaker Merck asked the FDA last month to approve cladribine tablets to reduce relapses of MS. Novartis, of Basel, Switzerland, plans to submit its experimental pill, FTY720, to U.S. regulators by the end of the year to reduce relapse and progression of disability. Paris-based Sanofi-Aventis SA is also in late stages of testing its teriflunomide pill for MS.

“Patients tell us they’re desperate for an oral drug,” Richert, of the MS Society, said. “We know that some people don’t go on medication in the first place because they can’t stand the thought of injection.”

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New Standard Reduces Heparin Potency

Beginning next week, heparin shipped to hospitals and pharmacies will be about 10% less potent than that currently available, a change that is likely to have clinical implications when the drug is administered as a bolus IV dose, the FDA said today.

The loss in potency is the result of new manufacturing controls -- including a new reference standard for the drug's unit dose -- adopted by United States Pharmacopeia (USP), a nonprofit standards-setting organization.

The FDA has known for months that the reference standards for heparin would change, but learned only three weeks ago that the change would reduce potency, said John K. Jenkins, MD, director of the office of new drugs, at the FDA's Center for Drug Evaluation and Research.

The difference in potency, which is estimated at about 10% per labeled unit, is unlikely to affect subcutaneous administration because there is a wide range of bioavailability when heparin is administered by that route, he said.

Jenkins said the new standard has been in the works since 2007, when contamination of heparin resulted in hundreds of severe allergic reactions and deaths as well as widespread recalls and shortages.

When USP circulated the new drug monograph for comment during the spring and summer, "there was little concern about clinical significance," but as manufacturers began making heparin using the new standard, one of the companies discovered the difference in potency and informed the FDA, Jenkins said.

The FDA then contacted other manufacturers and confirmed that the reduced potency was observed by all drugmakers using the new reference standard.

At that point, the FDA contacted USP to determine if the rollout of the new standard could be delayed until in vitro and in vivo testing could confirm the exact potency range.

But by then, it was too late. The four makers of heparin -- APP, Hospira, Baxter, and B. Braun -- had all retooled their manufacturing processes to make the drug based on the new standard, which takes effect today.

The best that the FDA could manage was an agreement to delay shipment of the new heparin until Oct. 8, Jenkins said.

To differentiate "new" from "old" heparin once supplies hit the shelves, the four manufacturers have agreed to special labeling on the new product.

Three companies -- APP, the largest manufacturer, which markets heparin in vials; Baxter, which sells the drug in IV bags; and B. Braun, which also markets heparin in IV bags -- have all agreed to add the letter "N" next to the lot number or expiration date on labels of "new" heparin.

The fourth manufacturer -- Hospira which markets heparin in intravenous bags, vials, and syringes -- said it will identify the new heparin with a unique numbering system.

Meanwhile, the FDA is cooperating with the companies to conduct both in vitro and in vivo testing to determine the exact extent of the potency difference. The in vitro tests should be completed in a few weeks, but "animal tests will take longer," said Jenkins. He estimated the in vivo results would be available in less than two months.

But even with those test results, the FDA may not require changes in heparin labeling because dosing of the drug has always been a matter of "individual monitoring of patients" to determine the anticoagulant effect.

Regardless of potency, Jenkins said, heparin should continue to be dosed based on results of patient monitoring.

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