FDA Approves Rhophylac® for the Treatment of Immune Thrombocytopenic Purpura (ITP)
New indication can help 200,000 Americans suffering from platelet disorder
King of Prussia, PA — 02 April 2007
CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for an additional indication for Rhophylac Rh0(D) (Immune Globulin Intravenous [Human]), an anti-D Rh immunoglobulin (Ig). The additional indication is for the treatment of immune thrombocytopenic purpura (ITP). Administered intravenously, Rhophylac is indicated to raise platelet counts in Rh0(D)-positive, non-splenectomized adult patients with chronic ITP. Having established efficacy and safety in over 10 years of use for other indications, Rhophylac offers high value pricing to help healthcare practitioners manage costs.
“Through the years, CSL Behring has made a significant commitment to patients with rare and serious disorders,” said Paul Perreault, Executive Vice President of Worldwide Commercial Operations, CSL Behring, which manufactures and markets Rhophylac. “In an environment of rising health care costs, Rhophylac provides the right balance of value and cost, while continuing the company’s long tradition of providing patients with safe and effective therapies.”
In an open-label, single-arm, multi-center trial, Rhophylac proved safe and effective in the treatment for ITP. In the trial, 98 Rh0(D)-positive adult chronic ITP patients with platelet counts of <30 x 109/L were treated with Rhophylac in a single intravenous dose of 250 IU (50 mcg)/ kg body weight. The primary efficacy endpoint was the response rate as defined by two criteria: a rise in platelet count greater than 20x109/L from baseline, and achievement of a platelet count of at least 30x109/L by Day 15. In various subsets of the intent-to-treat population (ITT), defined by baseline platelet counts, primary response rates ranging from 39 to 89 percent (66 percent overall) were achieved. The median time to response was three (3) days and median duration of response was 22 days (secondary endpoints).
Other secondary endpoints included: response rate, defined by an increase in platelet counts to >50x109/L by Day 15, as well as regression of hemorrhage. Response rates for an increase in platelet counts to >50x109/L ranged from 26 to 100 percent in the subsets of the ITT population. The overall regression of hemorrhage achieved was 88 percent in 50 patients with bleeding at baseline.
Highly Advanced Manufacturing Process
Rhophylac is manufactured using CSL Behring’s unique ChromaPlus™ process, yielding a safe, highly pure, and stable product. The process includes: solvent/detergent treatment to inactivate enveloped viruses (e.g., HIV, HCV, HBV); nanofiltration using a 15- nanometer virus filter to effectively remove enveloped and non-enveloped viruses (including models for HAV and human parvovirus B19); and ion-exchange chromatography to purify the final product by separating unwanted proteins.
Coupled with careful donor selection and screening, the ChromaPlus process significantly reduces the risk of viral transmission. In clinical studies, there was no evidence of viral transmission with Rhophylac.
Over the past 11 years, more than 1.5 million doses of Rhophylac have been administered worldwide to prevent Hemolytic Disease of the Newborn (HDN)—a condition that can affect a newborn from an Rh-incompatible pregnancy—and to treat Rh-negative individuals in the event of an incompatible blood transfusion. In 2004, Rhophylac received approval from the FDA for the suppression of Rh isoimmunization in pregnancy and obstetric conditions and following an incompatible transfusion.
“Our vast experience in preventing HDN has firmly established Rhophylac as a safe and effective product that physicians have come to rely on,” said Robert Lefebvre, Vice President and General Manager, U.S. Commercial Operations, CSL Behring.
Important Safety Information
Rhophylac is indicated to raise platelet counts in Rh0(D)-positive, non-splenectomized adult patients with chronic immune thrombocytopenic purpura (ITP). For the treatment of ITP, Rhophylac must be administered IV.
Rhophylac is indicated for suppression of rhesus (Rh) isoimmunization in:
- Pregnancy and obstetric conditions in non-sensitized, Rho(D)-negative women with an Rh-incompatible pregnancy, including routine antepartum and postpartum Rh prophylaxis and Rh prophylaxis in cases of obstetric complications, invasive procedures during pregnancy, or obstetric manipulative procedures.
- Incompatible transfusions in Rho(D)-negative individuals transfused with blood components containing Rho(D)-positive red blood cells.
For suppression of Rh isommunization, Rhophylac can be administered IM or IV.
Rhophylac is contraindicated in individuals with known anaphylactic or severe systemic reaction to human immune globulin products.
Allergic or hypersensitivity reactions may occur with Rhophylac; early signs of hypersensitivity include generalized urticaria, chest tightness, wheezing, hypotension, and anaphylaxis. Individuals with selective IgA deficiency can develop antibodies to IgA and may develop severe hypersensitivity and anaphylactic reactions. For these individuals, weigh the expected benefits of treatment against potential risks.
Rhophylac is derived from human plasma. As with all plasma-derived products, the risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.
Immune Thrombocytopenic Purpura: ITP patients being treated with Rhophylac should be monitored for signs and symptoms of intravascular hemolysis, including back pain, shaking chills, fever, and hemoglobinuria. Potentially serious complications of intravascular hemolysis include clinically compromising anemia, acute renal insufficiency, and, very rarely, disseminated intravascular coagulation, and death.
In patients with preexisting anemia, weigh the benefits of Rhophylac vs the potential risk of increasing the severity of the anemia.
The most common adverse reactions in ITP treatment with Rhophylac are chills, fever, increased bilirubin, and headache.
Suppression of Rh Isoimmunization: For postpartum use following an Rh-incompatible pregnancy, Rhophylac should not be given to the newborn infant.
The most common adverse reactions in suppression of Rh isoimmunization with Rhophylac are nausea, dizziness, headache, injection-site pain, and malaise.
Immune Thrombocytopenic Purpura, or ITP, is an autoimmune disease in which the immune system attacks and destroys the body's own platelets, the cells that prevent bleeding in blood vessels and facilitate clotting. There are two forms of ITP: acute ITP, which resolves within six months, and chronic ITP, which most often occurs in adults and by definition lasts six months or longer. The annual incidence of ITP is 100 to115 in every one million people. In the U.S., approximately 200,000 people have the disorder.
ITP is characterized by a low number of platelets (<30 x 109/L), usually caused by the body’s production of substances (antibodies) that coat the platelets and signal their elimination from the blood. Diagnosis of ITP is often made by excluding other possible causes of the low platelet count and bleeding. People with the disorder often have purple bruises on the skin called purpura, a sign that bleeding has occurred in small blood vessels under the skin. They can also have petechiae, small red splotches on the skin that resemble a rash.
For more information on ITP, please visit www.Rhophylac4ITP.com or contact the Platelet Disorders Support Association (www.pdsa.org), the leading ITP patient advocate group in the U.S.
About CSL Behring (formerly ZLB Behring)
CSL Behring is a global leader in the plasma protein biotherapeutics industry. Dedicated to improving the quality of life for patients throughout the world, CSL Behring provides safe and effective plasma-derived and recombinant products and offers patients a wide range of related services. The company’s broad portfolio of life-saving therapeutics is used in the treatment of individuals with hemophilia and other bleeding disorders, immune deficiency disorders and inherited emphysema; for the prevention of hemolytic diseases for the newborn; in cardiac surgery patients; and in shock and burn victims. Additionally, CSL Behring operates one of the world’s largest, fully owned plasma collection networks. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company that operates worldwide from its headquarters in Melbourne, Australia. For more information, please visit www.CSLBehring.com.
Sheila A. Burke
Director, Public Relations & Communications
Worldwide Commercial Operations