Tracleer and PPH News
FDA Approves Expanded Label Indication and Satisfaction of Subpart H Accelerated Approval Commitment for Remodulin (Treprostinil Sodium) Injection
SILVER SPRING, M.D., and RESEARCH TRIANGLE PARK, N.C. -- March 21, 2006 -- United Therapeutics Corporation announced today that the United States Food and Drug Administration (FDA) has approved a supplemental New Drug Application (sNDA) for Remodulin(R) (treprostinil sodium) Injection. The sNDA was filed by United Therapeutics in satisfaction of the FDA's Subpart H accelerated approval requirement for a Phase 4 post-marketing study to confirm the clinical benefit of Remodulin. The initial approvals of both subcutaneous and intravenous use of Remodulin were contingent upon United Therapeutics' completion of that study.
The Phase 4 study, which was successfully completed last year, involved the transition of patients from Flolan to either Remodulin or placebo. In the trial, 13 of 14 patients (93%) randomized to Remodulin were able to successfully transition from Flolan and complete the study without the need to re-institute Flolan therapy, compared to only 1 of 8 patients (13%) randomized to placebo (P =.0002).
"We are pleased to have successfully completed our Subpart H accelerated approval efficacy commitment," said Roger A. Jeffs, PhD, President and Chief Operating Officer of United Therapeutics. "This study confirms the clinical benefit of Remodulin in pulmonary arterial hypertension patients, provides expanded labeling specific to Flolan transition, and removes Remodulin's conditional approval status."
Based on these positive Phase 4 study results, the FDA has expanded Remodulin's labeling to include the following: "Remodulin is indicated to diminish the rate of clinical deterioration in patients requiring transition from Flolan; the risks and benefits of each drug should be carefully considered prior to transition." Previously, Remodulin had been approved as a continuous subcutaneous infusion or intravenous infusion (for those not able to tolerate a subcutaneous infusion) for the treatment of pulmonary arterial hypertension in patients with NYHA Class II-IV symptoms to diminish symptoms associated with exercise.
In clinical trials, the most common side effects reported with subcutaneous Remodulin therapy included infusion site pain (85%) and infusion site reaction (83%). Infusion site symptoms were sometimes severe (39%), and could require prescription narcotics (32%), or could lead to discontinuation of Remodulin (7%). Other adverse events included headache (27%), diarrhea (25%), nausea (22%), rash (14%), jaw pain (13%), vasodilatation (11%), dizziness (9%), edema (9%), pruritus (8%) and hypotension (4%).
Among patients (n=38) treated for 12-weeks with intravenous Remodulin in an open- label study, two patients experienced either line infections or sepsis. Other events potentially related to the mode of infusion include arm swelling, paresthesias, hematoma and pain.
Remodulin is a potent pulmonary and systemic vasodilator and should be used only by clinicians experienced in the diagnosis and treatment of pulmonary arterial hypertension. Reduction in blood pressure caused by Remodulin may be exacerbated by drugs that by themselves alter blood pressure, such as diuretics, antihypertensive agents, or vasodilators. Abrupt cessation or sudden large reductions in dosage of Remodulin may result in worsening of pulmonary arterial hypertension symptoms and should be avoided.
SOURCE: United Therapeutics Corporation
What Is Pulmonary Arterial Hypertension?
Pulmonary (PULL-mun-ary) arterial hypertension (PAH) is continuous high blood pressure in the pulmonary artery. The average blood pressure in a normal pulmonary artery is about 14 mmHg when the person is resting. In PAH, the average is usually greater than 25 mmHg.
PAH is a serious condition for which there are treatments but no cure. Treatment benefits many patients.
The pulmonary arteries are the blood vessels that carry oxygen-poor blood from the right ventricle (VEN-trih-kul) in the heart to the small arteries in the lungs. In PAH, three types of changes may occur in the pulmonary arteries:
The muscles within the walls of the arteries may tighten up. This makes the inside of the arteries narrower.
The walls of the pulmonary arteries may thicken as the amount of muscle increases in some arteries. Scar tissue may form in the walls of arteries. As the walls thicken and scar, the arteries become increasingly narrow.
Tiny blood clots may form within the smaller arteries, causing blockages.
There is less room for the blood to flow through these narrower arteries. The arteries may also stiffen. Over time, some of the arteries may become completely blocked.
The narrowing of the pulmonary arteries causes the right side of heart to work harder to pump blood through the lungs. Over time, the heart muscle weakens and loses its ability to pump enough blood for the body's needs. This is called right heart failure. Heart failure is the most common cause of death in people with PAH.
There are two types of PAH:
Primary pulmonary arterial hypertension (PPAH) is inherited or occurs for no known reason.
Secondary pulmonary arterial hypertension (SPAH) either is caused by or occurs because of another condition. The conditions include chronic heart or lung disease, blood clots in the lungs, or a disease like scleroderma (skler-o-DER-ma).
About 300 new cases of PPAH are diagnosed in the United States each year. SPAH is much more common.
Doctors have learned a lot about PAH in recent years. More treatments are now available. Researchers are also studying several promising new treatments that may prolong lives as well as improve the quality of life for people living with PAH.
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