U.S. FDA Accepts for Review CSL Behring’s Biologics License Application for Its Novel rVIII-SingleChain Therapy for Patients with Hemophilia A

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rVIII-SingleChain underscores CSL Behring’s deep commitment to developing and delivering specialty biotherapies that improve the well-being of patients with serious diseases

KING OF PRUSSIA, Pa. — 28 July 2015

CSL Behring announced today that the U.S. Food and Drug Administration has accepted for review the company’s Biologics License Application (BLA) for its novel investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment of hemophilia A. In the pivotal clinical trial, rVIII-SingleChain met all primary endpoints.

Hemophilia A is a congenital bleeding disorder characterized by deficient or defective factor VIII; nearly all affected patients are male. People with hemophilia A may experience prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. The condition affects approximately 1 in 6,000 male births.

“CSL Behring has one of the industry’s largest portfolios of biotherapies that improve the care and well-being of patients with a bleeding disorder,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and Director of R&D, CSL Limited. “Our scientific expertise and relationship with the bleeding disorders community led us to seek further advancements in the care and treatment of hemophilia. Today, we have the only recombinant single-chain factor VIII product in late-stage development for the management of hemophilia A, and we are excited to be one step closer to providing this innovative treatment to patients in the U.S.”

The BLA submission is based on the AFFINITY clinical development program, which includes a phase I/III open-label, multi-center trial examining safety and efficacy. The pharmacokinetics of rVIII-SingleChain compared with recombinant human antihemophilic factor VIII (octocog alfa) was also studied. Study design details for rVIII-SingleChain are available at clinicaltrials.gov.

Results from the phase I/III study were recently presented at the International Society on Thrombosis and Haemostasis (ISTH) congress in Toronto. Patients treating prophylactically had a median annualized bleeding rate (ABR) of 1.14 and a median annualized spontaneous bleeding rate (AsBR) of 0.00. The data also showed that, of 848 bleeds treated in the study, 94 percent were controlled with no more than two infusions of rVIII-SingleChain, with 81 percent controlled by one infusion. Moreover, hemostatic control of a bleeding event treated with rVIII-SingleChain was assessed by the investigator as excellent or good 94 percent of the time (835 assessed bleeding events).

The results presented included data on more than 14,000 exposure days in 146 patients on prophylaxis and 27 patients treating on demand for a bleeding event. In total, 120 patients were treated for more than 50 days of exposure; 52 had more than 100 days of exposure. In the prophylaxis group, 32 percent of patients were dosed twice weekly and 54 percent received treatment three times per week; the regimen was determined by the investigator. The most common adverse events were naso-pharyngitis, arthralgia, and headache. No inhibitors were reported.

About rVIII-SingleChain

Specifically designed for greater molecular stability, rVIII-SingleChain is the first and only single-chain factor VIII (FVIII) product in late-stage development for the treatment of hemophilia A. rVIII-SingleChain (also known as CSL627) has a strong affinity for von Willebrand factor, leading to greater stability and integrity of FVIII in circulation. For more information about CSL Behring’s recombinant products in development to treat hemophilia, visit http://www.cslbehring.com/products/bleeding-disorders/novel-recombinant-hemophilia-treatments.

About CSL Behring

The people and science of CSL Behring save lives around the world. We develop and deliver innovative specialty biotherapies, driven by our 100-year promise to help people with life-threatening conditions live full lives. With 14,000 employees and operations in 30 countries, CSL applies world-class R&D, high-quality manufacturing and patient-centered management.

CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a global biopharmaceutical company and a member of the CSL Group of companies. The parent company, CSL Limited (ASX:CSL), is headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.

For original article click here.

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CSL Behring Submits Biologics License Application for FDA Approval of Recombinant Fusion Protein Linking Coagulation Factor IX with Recombinant Albumin (rIX-FP) for Hemophilia B Patients

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KING OF PRUSSIA, Pa. — 16 December 2014

CSL Behring announced today it has submitted a biologics license application (BLA) to the United States Food and Drug Administration (FDA) for the marketing authorization of its long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). Once approved by the FDA, rIX-FP (Coagulation Factor IX {Recombinant}, Albumin Fusion Protein) will provide people with hemophilia B and their physicians a long-acting treatment option with dosing intervals up to 14 days.

“As we mentioned at our recent R&D investor briefing, submission of our BLA to the FDA for rIX-FP is a significant milestone for CSL Behring’s recombinant factor IX development program and moves us one step closer to bringing this innovative therapy to hemophilia B patients in the U.S.,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D Director, CSL Limited. “Our strong partnership with and commitment from the hemophilia community led us to develop rIX-FP based on novel recombinant albumin fusion technology. This technology has led to a long-acting treatment candidate that continues our legacy of improving the well-being of patients with bleeding disorders and other rare diseases.”

About PROLONG-9FP Clinical Development Program

CSL Behring’s BLA is based on the results from the PROLONG-9FP Phase II/III (patients ages 12 to 61 years) study. The Phase II/III pivotal study was an open-label, multicenter, safety, pharmacokinetic (PK) and efficacy study of rIX-FP in previously treated patients with severe hemophilia B (FIX ≤ 2%).

This study was designed to compare the change in frequency of spontaneous bleeding events between on-demand treatment and a weekly prophylaxis regimen in patients previously receiving only on-demand treatment; and the number of patients developing inhibitors against factor IX as primary outcome measures. The study evaluated multiple prophylaxis regimens, including 7-day and 14-day intervals. A sub-study evaluated the prevention and control of bleeding in patients with hemophilia B undergoing a surgical procedure.

Study design details for rIX-FP (CSL654) are available at www.clinicaltrials.gov.

About rIX-FP

CSL Behring engineered rIX-FP to extend the half-life of recombinant factor IX through genetic fusion with recombinant albumin. CSL Behring selected recombinant albumin as its recombinant genetic fusion partner for its coagulation factor proteins due to its long physiological half-life. In addition, recombinant albumin has been shown to have a good tolerability profile, low potential for immunogenic reactions and a well-known mechanism of clearance. The cleavable linker connecting recombinant factor IX and recombinant albumin has been specifically designed to preserve the native function of the coagulation factor in the fusion protein, while benefiting from recombinant albumin’s long physiological half-life.

In 2012, the FDA granted Orphan Drug Designation for rIX-FP for the treatment and prophylaxis of bleeding episodes in patients with hemophilia B. The designation includes routine prophylaxis treatment, control and prevention of bleeding episodes, and prevention and control of bleeding in perioperative settings. The FDA’s Orphan Drug Designation program provides orphan status to unique drugs and biologics defined as those intended for the safe and effective treatment or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Orphan designation qualifies the sponsor of the product for important tax credits, elimination of FDA license application fees and certain marketing incentives.

About Hemophilia B

Hemophilia B (congenital factor IX deficiency) is characterized by deficient or defective factor IX and affects approximately 1 in 25,000 to 50,000 people. Hemophilia B is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. Nearly all hemophilia B patients are male.

About CSL Behring

CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide.

CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a global biopharmaceutical company and a member of the CSL Group of companies. The parent company, CSL Limited (ASX:CSL), is headquartered in Melbourne, Australia. For more information, visit http://www.cslbehring.com.

CSL Behring Starts Pediatric Phase 3 Hemophilia Trial

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CSL Behring Enrolls First Patient in Global Pediatric Phase III Pivotal Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) to Treat Severe Hemophilia A

King of Prussia, PA — 02 May 2014

CSL Behring today announced that the first patient has been enrolled in the pivotal pediatric phase III study to evaluate the efficacy, safety and pharmacokinetics of its novel investigational recombinant factor VIII single chain (rVIII-SingleChain) for the treatment of previously treated children (up to age 11 years) with severe hemophilia A. The study site for this first enrollment is Malaysia.

A minimum of 25 previously treated subjects from six to 11 years of age and at least 25 subjects under six years of age who have undergone more than 50 exposure days with a previous factor VIII product are planned to be enrolled in this international, multicenter, open-label study. Subjects will be assigned to either an on-demand or prophylaxis treatment regimen for the treatment of bleeding episodes and will receive rVIII-SingleChain at a dose to be determined by the investigator. Hemostatic efficacy will be assessed by the subject or caregiver and the investigator, who will assess overall efficacy by a 4-point scale.

In an earlier study, rVIII-Single Chain showed improved pharmacokinetics over octocog alfa, the comparator, and demonstrated a safety and efficacy profile that supported advancement to late-stage clinical development. CSL Behring, in collaboration with its parent company CSL Limited, is developing rVIII-SingleChain for the treatment of hemophilia A as part of the AFFINITY clinical trial program.

About rVIII-SingleChain
Recombinant FVIII molecules currently available consist of a heavy and a light chain. Under certain conditions, these chains can dissociate, resulting in the formation of separated, or “dissociated,” rFVIII chains that are not hemostatically active. The CSL Behring rVIII-Single Chain uses a strong, covalent bond that connects the light and heavy chains, thereby creating a stable single chain rFVIII.

In-house CSL Behring studies have shown that the molecular integrity of rVIII-SingleChain is significantly increased using the single-chain design, resulting in a homogenous product that is more stable than currently available FVIII products. In addition, in-vitro studies have shown that rVIII-SingleChain demonstrates a strong affinity for von Willebrand factor (VWF), resulting in a faster and more efficient binding to VWF. The FVIII/VWF complex plays an important role in the physiological activity and clearance of FVIII and has been shown to have an influence on the presentation of FVIII to the immune system.

About Hemophilia
Hemophilia is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for people with hemophilia deficiency is to treat by replacement factor therapy.

About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide.

CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in the newborn. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical with headquarters in Melbourne, Australia. For more information, visit www.cslbehring.com.

###

Contact:
Sheila A. Burke
Director, Communications & Public Relations
Worldwide Commercial Operations
CSL Behring
O: 610-878-4209
Sheila.Burke at cslbehring.com

Original Press release can be found here.

National Outreach for Von Willebrand’s Disease

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NOW Conference video from Feb 2012, Phoenix, AZ

CSL Behring rIX-FP granted Orphan Drug Designation to treat hemophilia B

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Published on June 8, 2012 at 11:49 AM  by NewsMedical

CSL Behring announced today that the company has been granted Orphan Drug Designation by the United States Food and Drug Administration for its novel recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP). The Orphan Drug Designation is granted for the treatment and prophylaxis of bleeding episodes in patients with congenital factor IX deficiency (hemophilia B). The designation includes routine prophylaxis treatment, control and prevention of bleeding episodes, and prevention and control of bleeding in perioperative settings.

CSL Behring is developing this therapy in collaboration with its parent company CSL Limited (ASX: CSL).

“CSL Behring is pleased to have achieved this important regulatory milestone for our recombinant factor IX,” said Val Romberg, Senior Vice President, Research and Development at CSL Behring. “It represents yet another advance that our company is making in the area of recombinant factor development and is extremely encouraging.”

CSL Behring has engineered rIX-FP to extend the half-life of Factor IX through genetic fusion with recombinant albumin. Albumin has been chosen as the ideal recombinant genetic fusion partner for coagulation factor proteins due to its long physiological half-life. In addition, albumin has been shown to have a good tolerability profile, low potential for immunogenic reactions and a well-known mechanism of clearance compared to some other technologies. The cleavable linker connecting recombinant factor IX and recombinant albumin has been specifically designed to preserve the native function of the coagulation factor in the fusion protein, while benefiting from recombinant albumin’s long physiological half-life.

The FDA’s Orphan Drug Designation program provides orphan status to unique drugs and biologics, defined as those intended for the safe and effective treatment or prevention of rare diseases that affect fewer than 200,000 people in the U.S.

SOURCE CSL Behring

Do You Know the Signs and Symptoms of von Willebrand Disease (VWD)?

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Leading Health Information Source for Women Introduces Educational Materials About Most Common Bleeding Disorder

 RED BANK, N.J., April 18, 2012 /PRNewswire/ — Although von Willebrand disease (VWD) is the most common hereditary bleeding disorder in the United States, affecting up to 1 percent of the population, it is often misdiagnosed or under-diagnosed in women. HealthyWomen, the nation’s leading nonprofit health information source for women, has developed new educational materials they hope will shine a light on a disorder that, when undiagnosed and untreated, can severely impact the quality of life for women. 

Caused when von Willebrand factor, a protein in the blood that is necessary for clotting, is either missing or not working properly, VWD affects men and women equally. Yet, when undiagnosed and untreated in women, it puts them at a greater risk for life-threatening bleeding following childbirth and for undergoing unnecessary hysterectomies.  

“Undiagnosed bleeding disorders, like von Willebrand disease, can have real health consequences for women,” said Elizabeth Battaglino Cahill, RN, executive director of HealthyWomen. “The goal of these new materials, available on HealthyWomen.org, is to spark honest conversations among mothers, sisters and daughters about the signs and symptoms of VWD and to hopefully underscore the need for even more education about this condition.”

The five signs and symptoms of von Willebrand disease are:

  • Easy bruising
  • Frequent or prolonged nosebleeds
  • Heavy, prolonged menstruation
  • Prolonged bleeding following injury or surgery
  • Prolonged bleeding during dental procedures

“Because VWD is a hereditary disorder, many women dismiss tell-tale signs like easy bruising or heavy periods as normal because their mother or other female relatives experienced similar symptoms,” said Meera Chitlur, M.D., Director, Hemophilia Treatment Center and Hemostasis Program, Children’s Hospital of Michigan and who also works with the Foundation for Women & Girls with Blood Disorders (FWGBD), a non-profit advocacy and education organization dedicated to advancing physician and healthcare provider knowledge of the unique needs and challenges faced by women and adolescent girls with blood disorders.  “Unexplained bleeding or bruising is not normal and is something you should always discuss with your healthcare provider. If you are diagnosed with a bleeding disorder, treatments are available.”

The new materials include common questions about VWD answered by an expert, as well as real-life stories of women living with the condition. One of these women, Kristin Prior, 49, recalls her own diagnosis in 1996.

“While I had experienced many of the five signs and symptoms throughout early adulthood it wasn’t until I was 32 that I finally received my diagnosis of von Willebrand disease,” said Prior. “My hope is that women who are facing some of the same challenges that I did will read my story on HealthyWomen.org and realize that diagnosis and treatment can make a tremendous difference in their lives.”  

The von Willebrand disease educational resources were developed through an educational grant from CSL Behring, a world leader in developing and manufacturing safe and effective solutions to treat and manage bleeding disorders.

About HealthyWomen

HealthyWomen (HW) is the nation’s leading nonprofit health information source for women. For more than 20 years, women have been coming to HW for answers to their most pressing and personal health care questions. HW provides health information through a wide array of online content and print publications that are original, objective and reviewed and approved by medical experts. Its website, http://www.HealthyWomen.org®, was recognized by ForbesWoman as one of the “Top 100 Websites for Women” in 2010 and 2011 and was named the top women’s health website by Dr. Mehmet Oz in O, The Oprah Magazine. To learn more, visit www.HealthyWomen.org.

About the Foundation for Women & Girls with Blood Disorders

The Foundation for Women & Girls with Blood Disorders (FWGBD) is a non-profit advocacy and education organization dedicated to advancing physician and healthcare provider knowledge of the unique needs and challenges faced by women and adolescent girls with blood disorders.  Founded in 2010, the Foundation’s mission is to ensure that all women and adolescent girls with blood disorders are correctly diagnosed and optimally treated and managed at every life stage.  For more information, please visit www.fwgbd.org.

About CSL Behring

CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX: CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit http://www.cslbehring.com/.

Contact

Erin Graves, Director of Communications and New Media
HealthyWomen
(732) 978-4947
egraves@HealthyWomen.org

Lauren Abel
MCS Public Relations on behalf of HealthyWomen and CSL Behring
(800) 477-9626
laurena@mcspr.com

 

SOURCE HealthyWomen

For Original Article click here.

CSL Behring Commences Phase I Study With rVIIa-FP

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Phase I Study With rVIIa-FP, a Novel Therapy to Treat People With Hemophilia A and Hemophilia B Who Have Inhibitors

31 Mar 2012

KING OF PRUSSIA, PA, USA – March 30, 2012 – CSL Behring announced today the first in human dosing of recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP). The Phase I study will investigate in healthy volunteers the safety and pharmacokinetics of rVIIa-FP in comparison to placebo. CSL Behring, in collaboration with its parent company, CSL Limited (ASX: CSL.AX – News), is developing rVIIa-FP, a novel therapy to treat hemophilia A and hemophilia B patients who have inhibitors as part of the PROLONG 7- FP clinical study program. For more information about this study, please see www.clinicaltrials.gov.

CSL Behring’s albumin fusion technology uses albumin as the ideal recombinant genetic fusion partner for coagulation factor proteins due to its inherently long half-life, high potential for tolerability, known mechanism of clearance and low potential for immunogenic reactions. CSL Behring’s rVIIa albumin fusion protein is expected to exhibit a good tolerability profile and improved pharmacokinetics that may enable prophylaxis.

CSL Behring’s rVIIa-FP was previously granted Orphan Drug Designation by the European Commission and the United States Food and Drug Administration.

About Hemophilia
Hemophilia is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy. A complication in some patients is the development of inhibitory antibodies (inhibitors) to FVIII or FIX which render replacement therapy ineffective. This can occur in up to 25 percent of hemophilia A patients and approximately 5 percent of hemophilia B patients. One treatment option for these patients is recombinant activated factor VII (called a “bypassing agent”) which can be used to achieve hemostasis without the need for factor VIII or IX.

About the recombinant fusion protein linking coagulation factor VIIa with recombinant albumin (rVIIa-FP)
Preclinical studies have confirmed that CSL Behring’s rVIIa-FP has favorable pharmacokinetic properties compared with the existing recombinant FVIIa product. Significant increases in half-life have been observed across all animal species. The use of a bypassing agent with an extended half-life could offer significant benefit to those affected by hemophilia A or B with inhibitors and may offer patients the opportunity to be treated less frequently than with the currently available product.

CSL Behring’s clinical program intends to demonstrate that an extended half-life rVIIa-FP will result in a requirement for fewer doses while providing adequate therapeutic response in patients with hemophilia A and B with inhibitors.

About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX:CSL.AX – News), a biopharmaceutical company headquartered in Melbourne, Australia. For information: http://www.cslbehring.com.

SOURCE: CSL Behring

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