FDA approves product to prevent bleeding in people with rare genetic defect

The U.S. Food and Drug Administration last week approved Corifact, the first product intended to prevent bleeding in people with the rare genetic defect congenital Factor XIII deficiency.

Patients with congenital Factor XIII deficiency don’t make enough Factor XIII, a substance that circulates in the blood and is important for normal clotting. Without treatment, people with the condition are at risk for life-threatening bleeding.

Congenital Factor XIII deficiency is rare and affects 1 out of every 3 million to 5 million people in the United States. The deficiency may lead to soft tissue bruising, mucosal bleeding and fatal intracranial bleeding. Newborns with Factor XIII deficiency may have umbilical cord bleeding.

“This product helps fill an important need,” said Karen Midthun, M.D., director of the FDA’s Center for Biologics Evaluation and Research.

Corifact received orphan-drug designation by the FDA because it is intended for use in a rare disease or condition. It was approved for marketing under the FDA's accelerated approval regulations that require an on-going study to demonstrate that patients actually receive the clinical benefit predicted by the data obtained so far.

The FDA approved Corifact based on results of a clinical study of 14 people, including children, with congenital Factor XIII deficiency. The most common side effects observed were hypersensitivity reactions (allergy, rash, pruritus, and erythema), chills, fever, arthralgia, headache, elevated thrombin-antithrombin levels, and an increase in liver (hepatic) enzymes.

Corifact is made from the pooled plasma of healthy donors. People receiving Corifact may develop antibodies against Factor XIII that may make the product ineffective. It potentially can cause adverse events from abnormal clotting if doses higher than the labeled dose are given to patients.

Corifact is manufactured by CSL Behring of Marburg, Germany.

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FDA Approves Lysteda to Treat Heavy Menstrual Bleeding

The U.S. Food and Drug Administration today approved Lysteda tablets (tranexamic acid), the first non-hormonal product cleared to treat heavy menstrual bleeding (menorrhagia). Lysteda works by stabilizing a protein that helps blood to clot.

Heavy menstrual bleeding is reported each year by about 3 million U.S. women of reproductive age. Women with uterine fibroids may experience heavy menstrual periods. But in most cases, there is no underlying health condition associated with the condition.

“Menorrhagia can be incapacitating for some women,” said Kathleen Uhl, M.D., FDA’s associate commissioner of women’s health. “Heavy menstrual periods can cause pain, mood swings, and disruptions to work and family life.”

Tranexamic acid was first approved by the FDA in 1986 as an injection, under the brand name Cyklokapron, and is used to reduce or prevent bleeding during and following tooth extraction in patients with hemophilia, a hereditary bleeding disorder caused by the lack of a blood clotting factor.

The most common adverse reactions reported during clinical trials by patients using Lysteda included headache, sinus and nasal symptoms, back pain, abdominal pain, muscle and joint pain, muscle cramps, anemia, and fatigue. There was a statistically significant reduction in menstrual blood loss in women who received Lysteda, compared with those taking an inactive pill (placebo).

Use of Lysteda while taking hormonal contraceptives may increase the risk of blood clots, stroke, or heart attack, according to Scott Monroe, M.D., director of the Division of Reproductive and Urologic Products in the FDA’s Center for Drug Evaluation and Research. Women using hormonal contraception should take Lysteda only if there is a strong medical need, and if the benefit of treatment will outweigh the potential increased risk.

Lysteda is manufactured by Xanodyne Pharmaceuticals of Newport, Ky.

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CryoLife Receives FDA Approval to Begin U.S. Clinical Trial for BioFoam(R)

/PRNewswire/ -- CryoLife, Inc., (NYSE:CRY) , an implantable biological medical device and cardiovascular tissue processing company, today announced that the U.S. Food and Drug Administration (FDA) has granted approval for the company's Investigational Device Exemption (IDE) to conduct a human clinical trial for its BioFoam® Surgical Matrix protein hydrogel technology. BioFoam will be used to help seal liver parenchymal tissue when cessation of bleeding by ligature or other conventional methods is ineffective or impractical.

The approved IDE is for a prospective, multicenter, randomized feasibility study evaluating safety outcomes of BioFoam as compared to a standard topical hemostatic agent. The feasibility investigation will be conducted at two investigational sites and will enroll 20 eligible subjects with 10 subjects in each treatment group. CryoLife now will seek approval from the U.S. Department of Defense (DoD), which will be the final step necessary to begin this trial.

"Following our July 2009 CE Mark approval to distribute BioFoam in the EU, we now have approval to begin a clinical trial, a critical step forward in the process to gain FDA approval of BioFoam in the U.S.," said Steven G. Anderson, CryoLife president and chief executive officer. He added, "We believe that BioFoam may hold tremendous promise for surgeons around the world and are excited by the early data published thus far."

CryoLife is currently conducting a 60-patient controlled clinical launch of BioFoam at up to six centers in the United Kingdom, Germany, France and Italy. Based on the number of liver and spleen procedures performed annually in the European Community, CryoLife estimates the annual European market opportunity for BioFoam to be approximately $30 million and more than $100 million worldwide.

Upon successful completion of the feasibility study, and subsequent FDA and DoD approvals, a follow-on prospective, multicenter, randomized, controlled pivotal study will be conducted. It is currently anticipated that the pivotal investigation will enroll a total of 164 eligible subjects, 82 subjects in each treatment group across a maximum of 10 investigational sites.

The primary objective of the pivotal investigation will be to demonstrate a decrease in the time to achieve intraoperative hemostasis (a complex process that causes bleeding to stop) following open liver resection surgery in subjects receiving an application of BioFoam compared to a standard topical hemostatic agent. The secondary objectives of this investigation will be to compare time to hemostasis and the achievement of immediate hemostasis between the BioFoam group and the control group (a standard topical hemostatic agent) to demonstrate that BioFoam is at least equivalent in performance to the control group.

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FDA Approves Additional Use for IUD Mirena to Treat Heavy Menstrual Bleeding in IUD Users

/PRNewswire/ -- The U.S. Food and Drug Administration today approved Mirena (levonorgestrel intrauterine system) to treat heavy menstrual bleeding in women who use intrauterine contraception as their method of pregnancy prevention. This is the first intrauterine device approved by the FDA for this additional indication.

Mirena was approved as a contraceptive by the FDA in 2000. It is a small, flexible hormone-releasing device inserted into the uterus to prevent pregnancy. The device should be inserted by a trained health care professional.

"Women who suffer heavy, prolonged menstrual periods find the condition unpleasant, disabling, and frightening," said Kathleen Uhl, M.D., director of the FDA's Office of Women's Health. "Bleeding can be so heavy that women must miss work, school, or social activities."

"In the primary clinical trial, women using Mirena showed a statistically significant reduction in menstrual blood loss," said Scott Monroe, M.D., director of the Division of Reproductive and Urologic Products in the FDA's Center for Drug Evaluation and Research.

Participants in the clinical trial had excessive menstrual blood loss prior to treatment and did not have any medical conditions that are known to cause heavy menstrual bleeding, except for small uterine fibroids in some cases.

Mirena is recommended for women who have had a child. Clinical studies to support both the contraception and heavy menstrual bleeding indications have excluded women who have never been pregnant.

Since its approval in 2000, the most serious adverse reactions reported in patients using Mirena for any indication include: ectopic pregnancy (a pregnancy in which the fertilized egg grows outside the uterus); intrauterine pregnancy (a pregnancy with Mirena in place); group A streptococcal sepsis; an infection called pelvic inflammatory disease; embedment of the device in the uterine wall; and perforation of the uterine wall or cervix.

The most common adverse events reported by patients in the primary clinical trial using Mirena to treat heavy menstrual bleeding included uterine bleeding/spotting at irregular intervals, headache, ovarian cysts, vaginitis, pain during menstruation (dysmenorrhea), pelvic pain, and breast tenderness.

Mirena is made by Bayer HealthCare Pharmaceuticals, Inc., Wayne, N.J.

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FDA Issues Early Communication about a Safety Review of Xigris

The U.S. Food and Drug Administration today announced that it is working with the maker of Xigris (drotrecogin alfa activated), Eli Lilly and Company, to further evaluate the incidence of serious bleeding events and death in patients who receive Xigris, a drug used to treat severe sepsis (a blood stream infection).

A recent study and an accompanying editorial published in the journal Critical Care Medicine reported an increased risk of serious bleeding events and death in patients with sepsis and baseline bleeding risk factors who received Xigris. The study, a retrospective review of medical records of 73 patients who were treated with Xigris, found that serious bleeding events occurred in seven of 20 patients (35 percent) who had a bleeding risk factor versus two of 53 (3.8 percent) of patients without any bleeding risk factors. More patients with baseline bleeding risk factors died (13 of 20, or 65 percent) compared with patients without any bleeding risk factors (13 of 53, or 24.5 percent). As noted by the authors, limitations of this study include the retrospective design (looking back at events that already have taken place) and small patient population.

Xigris is known to increase the risk of bleeding. The drug’s current prescribing information (labeling) includes a warning that describes bleeding as the most common serious adverse effect and lists a number of risk factors that should be carefully considered when deciding whether to use Xigris. The labeling contraindicates the use of Xigris in several clinical situations where bleeding could lead to significant adverse reactions or death.

The FDA is not recommending that prescribers stop administering this medication. Consumers and health care professionals should notify the FDA of any complaints or problems associated with this product. These reports may be made to MedWatch, the FDA’s voluntary reporting program, by calling 800-FDA-1088, or electronically at www.fda.gov/medwatch/report.htm.

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FDA Approves RiaSTAP for Treatment of Bleeding in Patients with Rare Genetic Defect

The U.S. Food and Drug Administration today licensed RiaSTAP, an orphan drug for the treatment of bleeding in patients with a rare genetic defect known as congenital fibrinogen deficiency. Without treatment, these patients are at risk of potentially life-threatening bleeding.

People with congenital fibrinogen deficiency are unable to make sufficient amounts of fibrinogen, which plays an important role in blood coagulation by helping to form blood clots and prevent bleeding. Fibrinogen is manufactured in the liver and circulates in the blood plasma in a normal concentration of 250-400 mg/dL.

"This product offers much-needed treatment for the small number of patients with congenital fibrinogen deficiency," said Jesse Goodman, M.D., M.P.H., director of the FDA’s Center for Biologics Evaluation and Research. "If bleeding occurs in the brain or other organs and is left untreated, it may lead to blood loss, organ damage and death.”

Fibrinogen deficiency affects only 150 to 300 people in the United States and is usually diagnosed at birth when newborns bleed from their umbilical cord site. Children with the defect need to curtail activities because of risk of bleeding from minor trauma.

RiaSTAP is an intravenous fibrinogen concentrate made from the plasma of healthy human blood donors. The product is indicated for patients who have no fibrinogen or low levels of the substance, an abnormality known as afibrinogenemia, or for those patients whose fibrinogen levels are below 50 mg/dL, an abnormality known as hypofibrinogememia. The product is not indicated for patients with dysfibrinogenemia, who may have normal fibrinogen levels but defective fibrinogen function. Patients such as these are at risk for both bleeding and clotting complications.

The licensing of RiaSTAP was supported by a study of 15 patients with afibrinogenemia who achieved the target level of fibrinogen expected to prevent bleeding after they received 70 mg/kg of the drug. In addition, plasma from 14 of the 15 patients showed increased maximum clot firmness, a surrogate marker likely to predict clinical benefit. Fever and headache were the most common adverse reactions.

Clinical benefit will be further verified in a postmarketing study which will include both afibrinogenemic and hypofibrinogenemic patients.

Orphan drugs are drugs or biologics intended for use in a rare disease or condition. Manufacturers are qualified to receive certain government benefits in exchange for developing such products. RiaSTAP [Fibrinogen Concentrate (Human)] was developed under the FDA’s accelerated approval regulations.

The drug is manufactured by CSL Behring, Marburg, Germany.

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