Baxter Announces FDA Approval Of Ceprotin For Severe Congential Protein C Deficiency

DEERFIELD, Ill., March 30 /PRNewswire-FirstCall/ -- Baxter International Inc. announced the U.S. Food and Drug Administration (FDA) has approved CEPROTIN [Protein C Concentrate (Human)], a plasma-derived Protein C concentrate for use as replacement therapy in patients with life-threatening blood-clotting complications related to severe congenital Protein C deficiency.

Severe congenital Protein C deficiency results in a hypercoagulable state, meaning there is an abnormal tendency for blood clotting. This can cause severe, often life-threatening blood clots in small blood vessels, which if left untreated could result in blindness, severe brain damage, multi-organ failure and death. The disease manifests in children very early in life, often in utero or in the first few days of life. Incidence of severe congenital Protein C deficiency has been estimated to be one to two for every million births. Currently, there are fewer than 20 known cases of severe congenital Protein C deficiency in the United States. FDA has granted CEPROTIN orphan drug status.

CEPROTIN is the first FDA approved therapy for patients with severe congenital Protein C deficiency. It is indicated for patients with severe congenital Protein C deficiency for the prevention and treatment of venous thrombosis and purpura fulminans (PF) (a severe skin and systematic blood clotting disorder). CEPROTIN is indicated as a replacement therapy for pediatric and adult patients.

"Today's approval helps ensure that this critical therapy will continue to be available to patients," stated Larry Guiheen, president, Baxter BioPharmaceuticals. "Baxter has a long-standing history of developing therapeutics for rare coagulation disorders. As part of that commitment, we have made CEPROTIN available to patients, most of them children, for compassionate use and through our clinical trials since 1998. We are pleased to make this therapy available to patients with this extremely rare condition."

Baxter is committed to working closely with patients, family members and physicians to assist patients in need to gain access to CEPROTIN, and to answer any questions regarding the therapy. Starting April 2, people can call 1-888-CEPROTIN (1-888-237-7684) from 7:00 a.m. to 6:00 p.m., Central Standard Time, Monday through Friday, for more information about CEPROTIN.

"Importantly, the FDA's approval of CEPROTIN is a result of a successful patient-focused clinical trial and compassionate use program in the United States, and more than five years of use in Europe," stated Dr. Edward Gomperts, Children's Hospital Los Angeles. "This is a significant day for patients with severe congenital Protein C deficiency and their families, as this offers a much needed treatment option for these patients."

CEPROTIN Clinical Data

CEPROTIN's approval is based on data from a pivotal multi-center, open- label, non-randomized, Phase II/III study, which evaluated the safety and efficacy of CEPROTIN in subjects with severe congenital Protein C deficiency for the (on-demand) treatment of acute thrombotic episodes, such as PF, warfarin-induced skin necrosis (WISN), and other thromboembolic events, and for short-term or long-term prophylaxis. The study included 18 patients (nine male and nine female) ranging in age from newborn to 25.7 years. CEPROTIN was demonstrated to be effective in 94 percent of the episodes of PF. In the remaining six percent of patients, the treatment was found "effective with complications" because they required a dosage adjustment. Inadequate data were available for the treatment of WISN.

When compared with the efficacy ratings for 21 episodes of PF (historical control group), subjects with severe congenital Protein C deficiency were more effectively treated with CEPROTIN than those treated with modalities such as fresh frozen plasma or conventional anticoagulants. CEPROTIN also demonstrated effectiveness in reducing the size and number of skin lesions in patients. Treatment with CEPROTIN healed non-necrotic skin lesions after a median of four days, and necrotic skin lesions were healed after a median of 11 days.

Seven patients took CEPROTIN as a preventive measure before surgery or anticoagulation therapy and had no associated blood clotting complications. Eight patients who were given CEPROTIN as a long term preventive measure, again did not experience any blood clotting complications.

In the clinical development period, no serious adverse reactions related to CEPROTIN were reported. The most common adverse reactions observed in clinical trials were rash, itching, and lightheadedness.

About CEPROTIN

CEPROTIN [Protein C Concentrate (Human)] is indicated for patients with severe congenital Protein C deficiency for the prevention and treatment of venous thrombosis and purpura fulminans. CEPROTIN is indicated as a replacement therapy for pediatric and adult patients.

Important Safety Information

The most serious and common adverse reactions observed in clinical trials were rash, itching and lightheadedness.

The following adverse reactions have been identified during postapproval use of CEPROTIN: hemothorax, hypotension, hyperhydrosis, fever and restlessness.

Patients on low sodium/renal impairment diet should be informed that the quantity of sodium in the maximum daily dose of CEPROTIN exceeds 200mg.

CEPROTIN contains trace amounts of heparin, which may lead to Heparin- induced Thrombocytopenia.

As with all plasma-derived therapeutics, the potential to transmit infectious agents (e.g. viruses) cannot be totally eliminated.

Please review the CEPROTIN Prescribing Information for full prescribing details.

About Baxter

Baxter International Inc., through its subsidiaries, assists healthcare professionals and their patients with treatment of complex medical conditions, including hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other indications. The company applies its expertise in medical devices, pharmaceuticals, and biotechnology to make a meaningful difference in patients' lives.