FDA RECALL – Warning for Port Needles

This is important information for those of you who use huber needles. These needles are not supposed to “CORE” when inserted.  Please continue to read and pass on to everyone in the bleeding disorder community.

Infusion Set Needles [Manufactured by Nipro for Exelint]: Recall

Exel/Exelint Huber needles,
Exel/Exelint Huber Infusion Sets
Exel/Exelint “Securetouch+” Safety Huber Infusion Sets

Audience: Hospital Risk Managers, Surgical Service personnel

[Posted 01/26/2010] FDA notified healthcare professionals of a Class I recall of Exel/Exelint Huber needles, Exel/Exelint Huber Infusion Sets and Exel/Exelint “Securetouch+” Safety Huber Infusion Sets, manufactured by Nipro Medical Corporation for Exelint International Corporation. Huber needles are used to access ports implanted under the skin of chronically ill patients for repeated access to veins for the withdrawal of blood and infusion of medication, nutritional solutions, blood products, and imaging solutions. 

The recall was initiated due to ‘coring’, the cutting or dislodging of silicone cores or slivers from the ports into which they are inserted. Coring may lead to decreased effectiveness of the port, infection, necrosis, swelling, embolization, or need for additional surgery to replace implanted port. 

Inspections conducted in October 2009 of Nipro facilities found that their needles “cored” in 60 to 72 percent of tests. The reason for this coring is related to design and manufacturing processes, which the FDA continues to investigate and will update the public if there are new developments.

Hospitals, clinics and patients who have needles from these lists should immediately stop using these affected products and return any unused products to Exelint International Corporation.

There are more than 2 million units impacted by this recall in distribution nationwide. Recalled needles were manufactured from January 2007 to August 2009. Units subject to recall have a lot number that begins with “07,” “08,” “09,” and one of the product codes or catalog numbers noted in the FDA news release.

Original Post

Hemophilia History: Plasma to Recombinate

From:  Hematology.org

Hemophilia: From Plasma to Recombinant Factors

By Jeanne M. Lusher, MD, Children’s Hospital of Michigan, Detroit, MI

Hemophilia is caused by the failure to produce certain proteins required for blood clotting: factor VIII (hemophilia A) or factor IX (hemophilia B). Because the genes encoding these factors are on the X chromosome, these diseases (termed “X-linked”) usually affect only men, who carry only one X chromosome. Women carrying the disease gene are “carriers” and can transmit the disease to their sons, but women are rarely affected because they also carry a normal X chromosome. Patients with severe hemophilia produce less than 1 percent of the normal amount of the affected clotting factor and are dependent on factor from intravenous infusions to treat or prevent bleeding episodes.

In the late 1950s and much of the 1960s, fresh frozen plasma (FFP) was the mainstay of treatment for hemophilia A and hemophilia B. Each bag of FFP contained only miniscule amounts of factor VIII and factor IX, thus large volumes of intravenously administered FFP were needed to stop bleeding episodes. Children were usually hospitalized for treatment of bleeding into a knee, an elbow, or other joint. Many adolescents were reluctant to tell their parents that they were bleeding, delaying treatment and gradually leading to chronic joint disease with crippling deformities.

A great advancement came in the mid-1960s with the discovery of a method for preparing factor VIII from FFP by allowing it to thaw in the cold (cryoprecipitated plasma). This preparation could be stored in frozen form as “cryoprecipitate.” This allowed intravenous administration of more factor VIII in a smaller volume, allowing outpatient treatment for bleeds and even elective surgery in persons with hemophilia A. This more concentrated form of clotting factor VIII rapidly became the preferred treatment for acute bleeding episodes in patients with hemophilia A.

By the late 1960s, scientists and manufacturers developed methods for separating factor VIII and factor IX from pooled plasma, resulting in neatly packaged bottles of freezedried (lyophilized) factor VIII or factor IX concentrates. Each bottle had a label indicating the amount of factor VIII or factor IX it contained, allowing more accurate dosing. By the early 1970s, the availability of these concentrates led to home treatment, greatly changing the lives of people with hemophilia.

However, there was a price to be paid for this newfound independence. Thousands of plasma donations were combined as starting material for one batch of plasma-derived factor VIII or factor IX concentrate, and by the early 1980s, human blood, plasma, and plasma-derived products were discovered to be transmitting potentially deadly blood-borne viruses, including hepatitis viruses and HIV. Manufacturers of plasma-derived clotting factor concentrates attempted to kill these viruses with dry heat, solvent-detergent treatment, and pasteurization, with varying degrees of success. By 1985, most patients with hemophilia in the U.S. had been switched to heat-treated concentrates, but many had already been infected with HIV and a large percentage of them succumbed to it. Great concern about the safety of plasma-derived products continued in the hemophilia community.

Eventually better screening methods for blood donors were developed, improving the safety of donated plasma. Screening of donors for the hepatitis B virus was already in place, and in 1989 the hepatitis C virus (HCV) was isolated, allowing HCV antibody testing of donors to begin in 1990. HIV was identified in 1984, and by 1985 a blood test for HIV antibodies was instituted in blood and plasma collection facilities.

The successful cloning of the factor VIII gene in 1984 was a major breakthrough, allowing production of recombinant human factor VIII (r factor VIII). Clinical trials in humans began three years later, and this was truly an exciting time, especially for families who had lost loved ones to AIDS. The boys and young men participating in the studies of r factor VIII were overheard saying, “This is so exciting, being on the cutting edge of science!” By 1992, two pharmaceutical companies had licensed r factor VIII products for use in hemophilia A. Cloning of factor IX was first reported in 1982, and a licensed r factor IX product (BeneFIX) became available for people with hemophilia B in 1997.

Other significant advances that have been made in recent years include treatment for patients with inhibitors (antibodies that inhibit or interfere with the function of factor VIII or factor IX) and prophylaxis (treatment to prevent disease). Inhibitor antibodies develop in approximately 30-35 percent of people with hemophilia A and 1-3 percent with hemophilia B. We now have a much better understanding of the causes (genetic, racial/ethnic, etc.) and natural history of these inhibitors, how to detect and measure them with greater certainty, and how best to eliminate them by manipulating the immune system. Scientists have also developed innovative treatment products for “bypassing” the inhibitor, such as recombinant activated factor VII (r factor VIIa), which was first licensed for use in hemophilia in 1997. Newer second-generation products to treat inhibitors are actively being developed, and while much progress has been made in treating inhibitors, this complication remains the greatest problem in the management of hemophilia today.

Now that we have safer clotting factor replacement products, preventive (prophylactic) treatment has gained acceptance as a means to preserve normal joint and musculoskeletal function in boys and young men with hemophilia. First described by Swedish physicians 40 years ago, then recommended by the (U.S.) National Hemophilia Foundation’s Medical and Scientific Advisory Council in 1994, and recently documented as effective in a multicenter controlled trial, increasing numbers of young boys with severe hemophilia A or B are being started on prophylaxis with r factor VIII or r factor IX. Because of these developments, the future for those with hemophilia looks much brighter than it was only a few decades ago.

This article was published in December 2008 as part of the special ASH anniversary brochure, 50 Years in Hematology: Research That Revolutionized Patient Care.

Bayer to Discontinue Phase II Clinical Trial for Long-Acting Recombinant Factor VIII

I’ve been posing quite a few times recently on clinical trials.  It’s not what I focus on but it’s what’s in the news.

Study will not be able to meet primary endpoint / Bayer remains committed to developing its long-acting recombinant factor VIII compounds; alternative candidates in development

Berlin, January 25, 2010 – Bayer Schering Pharma AG, Germany has announced that an independent Data Safety and Monitoring Board (DSMB) has completed an interim analysis of a phase II trial (the LIPLONG study) of the company’s long-acting recombinant factor VIII, BAY79-4980. The DSMB concluded that the study will not be able to achieve the predetermined efficacy endpoint (non-inferiority). No safety concerns were raised. As a result of the DSMB findings and recommendation, Bayer has decided to discontinue the study.

LIPLONG is a randomized, double-blind active comparator controlled study designed to demonstrate the non-inferiority of BAY79-4980 infused in hemophilia A patients once a week as compared with Kogenate® FS, antihemophilic factor (recombinant), which is infused three times per week.

“While we are disappointed with the outcome of the analysis, we remain committed to developing our long-acting recombinant factor VIII compounds,” said Kemal Malik, M.D., member of the Board of Management of Bayer Schering Pharma AG and Head of Global Development. “It is our goal to enable once weekly prophylaxis dosing as well as other factor therapies, such as BAY VII, a modified recombinant factor VII therapy for hemophilia A and B in patients with inhibitors.” Further analyses of the LIPLONG data will be carried out over the next months.

Furthermore, Bayer has sought scientific advice from regulatory agencies for the clinical development of a directly PEGylated recombinant factor VIII molecule, which in preclinical models has a doubled half-life. Proof of concept trials in humans are scheduled to start in late 2010. The company’s current projects in hemophilia also include the development of an improved full-length FVIII molecule.

About Bayer
The Bayer Group is a global enterprise with core competencies in the fields of health care, nutrition and high-tech materials. Bayer HealthCare, a subsidiary of Bayer AG, is one of the world’s leading, innovative companies in the healthcare and medical products industry and is based in Leverkusen, Germany. The company combines the global activities of the Animal Health, Bayer Schering Pharma, Consumer Care and Medical Care divisions. Bayer HealthCare’s aim is to discover and manufacture products that will improve human and animal health worldwide. Find more information at www.bayerhealthcare.com.

About Bayer Schering Pharma
Bayer Schering Pharma is a worldwide leading specialty pharmaceutical company. Its research and business activities are focused on the following areas: Diagnostic Imaging, General Medicine, Specialty Medicine and Women’s Healthcare. With innovative products, Bayer Schering Pharma aims for leading positions in specialized markets worldwide. Using new ideas, Bayer Schering Pharma aims to make a contribution to medical progress and strives to improve the quality of life. Find more information at www.bayerscheringpharma.de.

Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

For info on Bayer’s latest news, click here.

Ipsen and Inspiration Biopharmaceuticals enter into partnership to create a world leading hemophilia franchise

• Portfolio covers all major hemophilia markets and worldwide sales could exceed $1.0 billion by 2020

• Following the exclusive in-licensing of Ipsen’s OBI-1 alongside its own IB1001, Inspiration’s portfolio now includes two recombinant protein products ready to enter Phase III in 2010 and two earlier development compounds for the treatment of bleeding disorders

• Ipsen to acquire initial 20% stake in Inspiration for $85 million with the potential to increase ownership up to 47% through up to $174 million in additional milestone-based funding.

Ipsen (Euronext:  FR0010259150; IPN) and Inspiration Biopharmaceuticals, Inc. (Inspiration) yesterday announced that they have entered into a partnership to create a world leading hemophilia franchise. The partnership is designed to leverage combined expertise and resources to advance a broad portfolio of recombinant proteins, which address all major hemophilia disorders in a unique way by focusing on two significant unmet needs: wider access to treatment with coagulation factors and treatment for inhibitor complications. The two lead product candidates are scheduled to begin Phase III clinical testing in 2010 including Ipsen’s recombinant porcine factor VIII, OBI-1 (for the treatment of patients with acquired hemophilia and hemophilia A who have developed an inhibitory immune reaction to human forms of factor VIII), and Inspiration’s recombinant factor IX product, IB1001 (for the acute and preventative treatment of bleeding in patients with hemophilia B). Combined with Inspiration’s novel proprietary technology and an early-stage pipeline of additional hemophilia factors, this broad and unique portfolio would provide greater access to care and fulfill unmet needs for patients suffering from bleeding disorders.

For more information on IPSEN, click here.
For more information on INSPIRATION BIOPHARMACEUTICALS, INC., click here.

Inspiration also has an open letter to the community, the letter can be read here.

The current PhaseII study on IB1001  has an estimated completion date of April 2010. (the study started Jan 2009). For more information of this clinical trial, click here.

For the completed clinical trial of OBI-1, click here.

for information on US Clinical Trails in your state, click here.

Oral Medication for Hemophilia – Phase 2a Clinicla Trial

PTC has initiated a Phase 2a clinical trial of ataluren in patients with nonsense mutation hemophilia type A and B (nmHA and nmHB). The trial is a multi-center, open label, dose escalation study. The main goals of the trial are to determine whether treatment with ataluren can result in an increase in Factor VIII or IX levels and whether the drug can safely be given to people with severe hemophilia due to a nonsense mutation.

For Trial sites, here.
For FAQ, here.

For information from the US Government Clinical Trails website, here.

Below is the Eligibility information for the trial….

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

• Ability to provide written informed consent
• Age ≥18 years
• Presence of a nonsense mutation as the sole disease-causing mutation in the FVIII or FIX gene
• At least 20 prior treatments with FVIII or FIX concentrates
• Willingness and ability to comply with scheduled visits, drug administration plan, study restrictions, and study procedures

Exclusion Criteria:

• Known hypersensitivity to any of the ingredients or excipients of the study drug
• Any history of prior anti-FVIII/FIX inhibitors

Unable or unwilling to forego prophylactic FVIII/FIX concentrate use during the screening and on-study periods (Note: Patients are allowed use of FVIII/FIX concentrates for treatment of bleeding episodes while on study)

ABOUT PTC THERAPEUTICS

PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. PTC has developed proprietary technologies that it applies in its drug discovery activities and are the basis for collaborations with leading biopharmaceutical companies such as Celgene, Genzyme, Gilead, Merck, Pfizer and Roche. For additional information, visit the company’s web site at www.ptcbio.com.

Reliability of Fetal Sex Determination Using Maternal Plasma

Below is an abstract from the Obstetrics & Gynecology Journal – for other reviews look here, or here.

Obstetrics & Gynecology:

January 2010 – Volume 115 – Issue 1 – pp 117-126

doi: 10.1097/AOG.0b013e3181c3c938

Original Research

Scheffer, Peter G. MD; van der Schoot, C Ellen MD, PhD; Page-Christiaens, Godelieve C. M. L. MD, PhD; Bossers, Bernadette; van Erp, Femke; de Haas, Masja MD, PhD

Abstract

OBJECTIVE: To determine the diagnostic accuracy of noninvasive fetal sex determination in maternal plasma.

METHODS: All consecutive patients for whom fetal sex determination in maternal plasma was performed in our laboratory from 2003 up to 2009 were included in the study. Real-time polymerase chain reaction was performed for the SRY gene and multicopy DYS14 marker sequence. A stringent diagnostic algorithm was applied. In the case of a positive result for both Y chromosome–specific assays, a male-bearing pregnancy was reported. In the case of a negative result, the presence of fetal DNA was ascertained through the use of 24 biallelic insertion/deletion polymorphisms or paternally inherited blood group antigens. Only if the presence of fetal DNA was confirmed was a female-bearing pregnancy reported. Results were compared with the pregnancy outcomes.

RESULTS: A total of 201 women were tested. The median gestational age was 9 0/7 weeks (interquartile range 8 0/7 to 10 0/7 weeks). In 189 of 201 cases (94%), a test result was issued; in 10 cases, the presence of fetal DNA could not be confirmed; in two cases, an early miscarriage was observed. Pregnancy outcome was obtained in 197 cases, including 105 male-bearing and 81 female-bearing pregnancies and 11 miscarriages. Sensitivity and specificity of the test were 100% (95% confidence intervals 96.6–100% and 95.6–100%, respectively). In all 10 cases in which the presence of fetal DNA could not be confirmed, a female was born.

CONCLUSION: Noninvasive fetal sex determination in maternal plasma is highly reliable and clinically applicable.

LEVEL OF EVIDENCE: III

 

HFA Voices Blood Safety Concerns

WASHINGTON, Dec. 14 /PRNewswire-USNewswire/ –

The Hemophilia Federation of America (HFA) expresses its concern regarding the ongoing labor issues involving the Teamsters Local 929 and the American Red Cross of Pennsylvania /New Jersey. The national blood system depends on skilled workers to provide clean, safe blood to laboratories. The blood is distilled into products such as clotting factor for use in treating hemophilia and von Willebrand disease.

The Division of Blood Services at the American Red Cross forms an integral link in America’s blood supply. We would encourage the American Red Cross to re-commit itself to maintaining a safe, clean, and healthful presence inside America’s larger health infrastructure.

Blood collection at the Red Cross continues to be subject to a sixteen-year-old consent decree stemming from Federal Court action. The nature of this civil matter raises serious questions. The ongoing contract impasse and labor action serve as just the latest evidence of a pattern of behavior which seems to place cost ahead of the health and well-being of both workers and end-users of blood products.

When blood safety is threatened by the business cycle, it is of grave concern. The safety of the blood supply remains our highest priority. To this end, we call on all institutions serving users of blood products to place safety concerns ahead of cost. We, as consumers, saw first-hand the dire consequences of allowing the shadow of cost weigh too heavily on public health outcomes, as some ten-thousand people with hemophilia contracted HIV/AIDS and/or Hepatitis C.

The Hemophilia Federation of America calls on all of the parties to re-double their efforts and move towards a speedy and equitable end to this current labor stalemate. It is our belief that doing so is in the best interest of all parties concerned.

The Hemophilia Federation of America is a national 501(c)(3) organization consisting of 30 member organizations and numerous individual members who offer assistance and grassroots advocacy on behalf of the bleeding disorders community. Incorporated in 1994, the HFA provides programs and services to improve the quality of life for persons with hemophilia and von Willebrand disease (VWD).

For more information, visit our website at www.hemophiliafed.org or call 1-800-230-9797.

SOURCE Hemophilia Federation of America

Octapharma USA Announces FDA Approval of wilate(R) – the First Replacement Therapy Developed Specifically for von Willebrand Disease

HOBOKEN, N.J., Dec. 7 /PRNewswire/ — Octapharma USA today announced the U.S. Food and Drug Administration has approved wilate® for the treatment of spontaneous and trauma-induced bleeding episodes in patients with all types of von Willebrand disease (VWD). Wilate® is a newly developed, high-purity, double virus inactivated von Willebrand Factor/Coagulation Factor VIII Concentrate (Human) that demonstrated efficacy for all types of VWD, including pediatric patients, in four prospective clinical trials utilizing both objective and subjective criteria.

Wilate® is indicated for the treatment of spontaneous and trauma-induced bleeding episodes in patients with severe VWD as well as patients with mild or moderate VWD in whom the use of desmopressin is known or suspected to be ineffective or contraindicated.(1) Wilate® is the first double virus inactivated VWF/FVIII (von Willebrand Factor / Factor FVIII), high-purity concentrate, utilizing the solvent/detergent (S/D) process and a special terminal dry-heating (TDH) system. The selected purification processes isolates the VWF/FVIII complex under highly protein-protecting conditions, resulting in a 1:1 ratio of VWF:RCo (ristocetin cofactor) and FVIII activities that is similar to normal plasma. No albumin is added as a stabilizer. Wilate® is exclusively derived from large pools of human plasma collected in U.S. FDA approved plasma donation centers.

The FDA approval of wilate® marks the entrance of Octapharma USA into the U.S. blood coagulation market, with product availability scheduled for early 2010. Octapharma USA is the rapidly growing U.S. division of Octapharma AG, one of the largest plasma products manufacturers in the world.

VWD is the most common bleeding disorder, which is found in approximately 1% to 2% of the U.S. population, according to the Centers for Disease Control and Prevention.(2) The illness is a result of the body’s inability to make von Willebrand Factor, the human protein that helps clot blood.
“The FDA approval of wilate® makes this the first and only replacement therapy developed and manufactured specifically for VWD. Its unique viral attenuation steps and it’s close to 1:1 ratio of FVIII and VWF will provide a next generation treatment option for patients with von Willebrand disease,” said Craig Kessler, M.D., Georgetown University Hospital, Professor of Medicine and Pathology and Director of the Division of Coagulation.

Four prospective clinical trials have demonstrated the safety, tolerability and hemostatic efficacy of wilate® in the treatment of acute bleeding episodes and prophylaxis in patients with various types of VWD. Using objective criteria, wilate® was observed in 1,068 bleeding episodes and determined to be successful between 84% and 93% of the time with results varying dependent on patient type.(3)

“Octapharma’s worldwide commitment to coagulation disorders dates back to Octapharma Group’s formation 25 years ago,” said Octapharma USA President Flemming Nielsen. “We are thrilled that U.S. patients will now have access to wilate® following its significant success in Europe as a next generation therapy. Octapharma is committed to providing the U.S. market with life-enhancing therapies.”

Since the mid-1980s, the requirements for the viral safety of plasma preparations have constantly been made increasingly stringent, requiring demonstrated virus elimination/inactivation.(4) (5) Several viral inactivation steps have enhanced the safety of coagulation products, but S/D inactivation is the current gold standard for safety from highly infectious enveloped viruses.(6) Octapharma was the first manufacturer to apply the S/D inactivation to a large-scale production of plasma derivatives. The wilate® manufacturing process provides two independent and effective virus inactivation procedures, namely S/D treatment in bulk and TDH treatment of the lyophilized product in final container. In addition, the ion-exchange chromatography step utilized during wilate® manufacturing contributes to the viral safety.

Plasma contains VWF and FVIII at very low concentration. The wilate® manufacturing process is designed to enrich the proportion of VWF/FVIII complex. Accompanying plasma proteins that may give rise to clinical side-effects, as well as proteases that could impair the stability of coagulation factors and degrade their natural structure and functionality, are efficiently removed during production.
Octapharma AG
Headquartered in Lachen, Switzerland, Octapharma is one of the largest plasma products manufacturers in the world and has been committed to patient care and medical innovation for over 25 years. Octapharma’s core business is the development, production and sale of high quality human protein therapies from both human plasma and human cell lines, including immune globulin intravenous (IGIV). In the U.S., Octapharma’s IGIV product, octagam® (immune globulin intravenous [human] 5%), is used to treat disorders of the immune system, and Octapharma’s Albumin (Human) is indicated for the restoration and maintenance of circulating blood volume. Octapharma employs over 3,000 people and has biopharmaceutical experience in 80 countries worldwide, including the United States, where Octapharma USA is headquartered in Hoboken, N.J. Octapharma operates two state-of-the-art production sites licensed by the U.S. Food and Drug Administration, providing the highest level of production flexibility and minimizing product shortages. For more information, please visit www.octapharma.com.

Forward-looking statements
This news release contains forward-looking statements, which include known and unknown risks, uncertainties and other factors not under the company’s control. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. These factors include results of current or pending research and development activities and actions by the FDA or other regulatory authorities.

References
(1) Mollison.P.L., Engelfriet C.P., Contreras M.: Some unfavourable effects of transfusion; in Klein H.G., Anstee D.J. (eds): Mollison’s Blood Transfusion in Clinical Medicine. Blackwell Publishing, 2005, pp 666-700.
(2) Centers for Disease Control and Prevention. Bleeding disorders. Available at www.cdc.gov/ncbddd/hbd/hemophilia.htm. Accessed on November 22, 2009.
(3) Wilate® Approved Complete Prescribing Information, December 2009.
(4) Note for guidance on virus validation studies: The design, contribution and interpretation of studies validating the inactivation and removal of viruses CPMP/BWP/268/95 February 1996.
(5) Note for guidance on plasma-derived products CPMP/BWP/269/95, rev.2. July 1998.
(6) Farrugia A . Guide for the assessment of clotting factor concentrates for the treatment of Hemophilia. 2003, WFH.

SOURCE Octapharma USA
FOR SPC below is valid in these countries:
RMS: DE. CMS: AT, BE, DK, FI, LU, NO, PT, SE.
SPC :  http://www.octapharma.com/documents/spc/181Wilspc_18int_191108_MRP.pdf

from EarthTimes

Biovitrum Advances Novel Factor VIII Long-Acting Hemophilia A Therapy into Clinical Trials

Stockholm, Sweden – Dec 15, 2009 – Biovitrum AB (publ) (STO: BVT) today announced that the first patient was dosed in a phase I/IIa study of its long-acting fully-recombinant Factor VIII Fc fusion (rFVIIIFc) protein. The phase I/IIa open-label study will assess the safety, tolerability and pharmacokinetics of rFVIIIFc in severe, previously-treated, hemophilia A patients. The rFVIIIFc program and international study are partnered with Biogen Idec (NASDAQ: BIIB).

  

Hemophilia A patients require frequent Factor VIII injections, which create a significant burden for these individuals. The rFVIIIFc molecule is being investigated for the potential to prolong protection from bleeding and reduce the frequency of injections for both prophylaxis and on-demand therapy in Hemophilia A. Preclinical studies showed improved half-life of rFVIIIFc, which is based on Biogen Idec’s monomeric Fc-fusion technology (recently presented 7 December 2009 at the American Society of Hematology conference). 

 

“We are excited about bringing rFVIIIFc into the clinical stage together with Biogen Idec and, thereby adding another significant collaboration project to the ongoing recombinant Factor IX Fc fusion (rFIXFc)  clinical program. The innovative rFVIIIFc program holds great potential in offering true value to hemophilia A patients, and is thus a prioritized therapeutic and business area within Biovitrum,” said Peter Edman, CSO of Biovitrum.

 

About Hemophilia A

Hemophilia A is a rare, inherited disorder in which the ability of a person’s blood to clot is impaired. Hemophilia A occurs in about 1 in 10,000 male births annually and is caused by having substantially reduced or no factor VIII protein, which is needed for normal blood clotting. People with hemophilia A therefore need injections of factor VIII to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening hemorrhages. Prophylaxis treatment with infusions three times per week or every second day to maintain a sufficient circulating level of coagulation factor is being increasingly used, and long-term studies demonstrate that such regimens increase the patient’s life expectancy and greatly reduce if not eliminate progressive joint deterioration. The current global market for recombinant Factor VIII products is over 4 BUSD annually.

 

About Biovitrum

Biovitrum is an international pharmaceutical company that markets specialist pharmaceuticals in several regions. Using its expertise and experience Biovitrum takes scientific innovation to patients with significant unmet medical need. Research expertise and capabilities are focused on development and production of biotechnology therapeutics within our prioritized areas of hemophilia, inflammation/autoimmune diseases, cancer supportive care and malabsorption. The company has revenues of approximately SEK 1.2 billion and around 400 employees. The company head office is located in Sweden and it is listed on the Stockholm OMX Nordic Exchange. For more information please visit www.biovitrum.com.

 

 

 

 

For more information please contact:

 

Biovitrum AB (publ)

Erik Kinnman, EVP Investor Relations

Phone: +46 73 422 15 40

erik.kinnman@biovitrum.com

 

 

Peter Edman, CSO

Phone: +46 8 697 21 77

 

 

Biovitrum AB (publ) may be required to disclose the information provided herein pursuant to the Swedish Securities Markets Act. The information was provided for public release on December 15, 2009 at 08:30 a.m. CET.

for the Pdf of this this press release click here.

XANODYNE ANNOUNCES FDA APPROVAL OF LYSTEDA™ (tranexamic acid) FOR TREATMENT OF WOMEN WITH HEAVY MENSTRUAL BLEEDING

.-.-.-.-.-.-.-.-.-.-.   Xanodyne Press Release   .-.-.-.-.-.-.-.-.-.

First-in-class non-hormonal, oral therapy indicated specifically for cyclic HMB

NEWPORT, K.Y. (Nov. 16, 2009) — Xanodyne Pharmaceuticals, Inc., an integrated specialty pharmaceutical company with both development and commercial capabilities focused on women’s healthcare and pain management, announced today that Lysteda™ (tranexamic acid) oral tablets has received U.S. Food and Drug Administration (FDA) approval for treatment of women suffering from cyclic heavy menstrual bleeding (HMB), also known as menorrhagia. Lysteda was approved following a Priority Review by FDA and is a first-in-class non-hormonal, oral therapeutic agent indicated specifically for this condition.

“We are very pleased with the U.S. approval of Lysteda, which represents an important new non-hormonal treatment option for women suffering from heavy menstrual bleeding,” said Dr. Gary A. Shangold, Chief Medical Officer, Xanodyne. “Lysteda is a significant addition to Xanodyne’s emerging product portfolio and represents the company’s second FDA drug approval within five months.”

Heavy menstrual bleeding is a persistent and recurrent medical condition that is one of the most common complaints encountered by gynecologists and primary care physicians. In the United States, this medical condition affects about 10 percent of women of reproductive age. It is described as regular intervals of menstruation with excessive volume that may exceed 80 milliliters of blood loss per menstrual cycle. HMB is often associated with a disruption in daily routines leading to a significant decrease in health-related quality of life and time lost from work or school.

An abnormally high rate of clot breakdown (fibrinolysis) in the uterus has been associated with heavy menstrual bleeding. As an anti-fibrinolytic, Lysteda works to reduce this excessive activity, thereby helping to support one of the important natural mechanisms by which menstrual blood flow normally stops each month. Until today, no pharmacotherapy which works through this mechanism has ever been approved by the FDA specifically for the treatment of this condition.

“Heavy menstrual bleeding is a very common gynecologic problem,” said Dr. Daniel R. Mishell Jr., The Lyle G. McNeile Professor and former Chairman of Obstetrics and Gynecology at the Keck School of Medicine, University of Southern California. “This disorder impairs women’s activity and quality of life. The FDA’s approval of Lysteda to treat heavy menstrual bleeding provides clinicians with a new therapeutic option to help women with heavy bleeding.”

In the clinical trials which were the basis for Lysteda’s approval, there was a statistically significant reduction in menstrual blood loss in women who received Lysteda, compared with those taking an inactive tablet (placebo). 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. Concomitant use of hormonal contraceptives and Lysteda may further exacerbate the increased risk of blood clots, stroke, or heart attack known to be associated with hormonal contraceptives. Therefore, though there are no clinical trial data on the risk of thrombotic events with the concomitant use with hormonal contraceptives, 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.

About Xanodyne

Xanodyne Pharmaceuticals, Inc., which commenced operations in 2001, is an integrated specialty pharmaceutical company with both development and commercial capabilities focused on women’s healthcare and pain management. Xanodyne markets a portfolio of products consisting of prescription pharmaceuticals and a line of prenatal vitamins. Additionally, Xanodyne is advancing a late stage pipeline of product candidates targeted at significant potential markets in Xanodyne’s focus areas.

Investor/Media Contact: Dr. Gary Shangold (908) 604-2354
Source: Xanodyne Pharmaceuticals, Inc.

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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. (Were they even looking for vWD – von Willebrand Disease?)

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.

For full perscribing information, look here.