US health regulators expanded approval of Novartis’ Promacta to treat children with a rare blood disorder.
Novartis said that the US Food and Drug Administration (FDA) approved Promacta (eltrombopag) for the treatment of children six years and older with chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. The drug has been approved in the US for treatment of adult patients with this condition since 2008.
Novartis gained access to the drug through its recent asset exchange with GlaxoSmithKline.
ITP is a blood disorder characterized by blood that does not clot as it should, due to a low number of platelets. Patients with ITP often develop purple bruises or tiny red or purple dots on the skin, and also may have nosebleeds, bleeding from the gums during dental work, or other bleeding that is hard to stop. In most cases, an autoimmune response is thought to cause ITP in which a person’s immune system attacks and destroys its own platelets. Two types of ITP exist. These are acute and chronic. Acute ITP typically occurs in children, often after a viral infection, and generally lasts less than six months. After six to twelve months, the platelet count returns to normal and treatment may not be needed. However, up to 30 percent of teenagers and children with ITP experience persistent disease for more than six months and are diagnosed with chronic ITP (cITP). ITP affects as many as 5 in 100,000 children each year.
“Young patients with chronic ITP who have either an insufficient response to or side effects from standard therapies have limited treatment options, making this FDA approval of eltrombopag for children six years and older particularly important,” said James B. Bussel, MD, a professor of pediatrics, of pediatrics in obstetrics and gynecology and of pediatrics in medicine at Weill Cornell Medical College, and lead study investigator of the PETIT study. “Through the eltrombopag studies, one of which is the largest randomized trial ever performed in children with chronic ITP, we discovered that Promacta – a treatment that can be taken once daily by mouth and shown to be well tolerated – can manage this disorder and help these young patients.”
The most commonly available and used therapies for ITP include corticosteroids and intravenous immunoglobulins (IVIG), however, these are associated with side effects that are often difficult to tolerate in pediatric setting.
Promacta’s expanded approval was based on data from the PETIT and PETIT2 clinical trials. In the trials, Promacta significantly increased sustained platelet counts among some pediatric patients with chronic ITP. Additionally, some patients taking concomitant ITP medications were able to reduce or discontinue their use of these drugs, primarily corticosteroids.
Promacta is a once-daily oral thrombopoietin (TPO) receptor agonist that works by inducing simulation and differentiation of megakaryocytes, the large cells found in bone marrow, from bone marrow stem cells, in order to increase platelet production.
“Today’s FDA approval of Promacta for children with chronic ITP, a rare and potentially serious blood disorder, gives new hope to patients and their families,” said Bruno Strigini, President, Novartis Oncology. “All patients are important, but when we can help children, we are especially gratified. This approval underscores our expertise in benign hematologic disease and our commitment to provide treatments for rare diseases.”
Last updated: 6/12/15; 11:45am EST