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GSK files eltrombopag for chronic ITP (thrombocytopenia) with the FDA
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21 December 2007
www.pharmatimes.com
GlaxoSmithKline has submitted a New Drug Application to US regulators in a bid to get approval for Promacta, its experimental drug designed to treat a rare blood disorder.
The firm is asking the US Food and Drug Administration to give the green light to Promacta (eltrombopag), an oral platelet growth factor therapy being developed to treat chronic idiopathic thrombocytopenic purpura, or ITP. This is a rare disorder characterised by increased platelet destruction or inadequate platelet production in the blood, which causes an increased risk of bruising and bleeding.
People with chronic ITP often bleed from small blood vessels causing bruises, nosebleeds or even fatal gastrointestinal or intra-cerebral bleeds. Health officials estimate that between 50,000 and 100,000 individuals have the disorder in the USA.
The NDA submission is supported by "the largest database of clinical trial information on investigational therapies for chronic ITP patients", GSK noted. In a Phase III study, patients on the trial were randomised to either placebo (38 patients) or eltrombopag 50mg (76 patients) once daily for six weeks though the latter dose could be increased to 75mg in patients not responding after an initial three weeks of treatment. At the end of the trial, 59% of eltrombopag-treated patients and 16% on placebo treated patients achieved a platelet count of more than 50,000/microlitre.
Preliminary results from an continuing late-stage trial of Promacta were presented last week at the American Society of Hematology meeting in Atlanta. The treatment also showed it was well tolerated in the 94 patients taking part in the study, GlaxoSmithKline said.
Paolo Paoletti, senior vice president of the Oncology Medicine Development Centre at GSK, said that patients with chronic ITP do not have a treatment option that offers the convenience of an oral platelet growth factor. He added that the firm is hopeful that, if approved, Promacta will provide "a new, convenient and effective option for treating this difficult disease".
GSK plans to seek approval to sell Promacta under the tradename Revolade in Europe in 2008 and the European Medicines Agency's Committee for Orphan Medicinal Products adopted a positive opinion on the drug in June. Analysts believe it has the potential to be a $3 billion blockbuster.
Phase III data looks promising for GSK's eltrombopag
11 June 2007
GlaxoSmithKline has presented positive data from a late-stage study of its new platelet-boosting treatment Revolade, one of the drugs in the firm's pipeline that analysts believe can be a blockbuster.
Results from a pivotal international Phase III study, which were presented at the European Haematology Association congress in Vienna, showed that Revolade (eltrombopag), at 50-75mg once daily, resulted in a statistically significant increase in platelet counts and also reduced bleeding in adult patients with chronic idiopathic thrombocytopenia purpura.
ITP is a low platelet count disorder that leaves sufferers at risk of spontaneous bruising, mucosal bleeding and, in severe cases, intracranial haemorrhage. There are estimated to be 50,000-100,000 people diagnosed with chronic ITP in the USA alone and the predicted five-year mortality rates for ITP patients with persistent low platelet counts range from 2.2% for patients younger than 40 years of age, to 47.8% for those older than 60.
Patients on the trial were randomised to either placebo (38 patients) or eltrombopag 50mg (76 patients) once daily for six weeks though the Revolade dose could be increased to 75mg in patients not responding after an initial three weeks of treatment. At the end of the trial, 59% of eltrombopag-treated patients and 16% on placebo treated patients achieved a platelet count of more than 50,000/microlitre. Importantly, said GSK, there was a significantly lower incidence of bleeding events during treatment with Revolade compared to placebo.
Drew Provan of the Department of Haematology at the Royal London Hospital, UK, and an investigator on the trial said that options are particularly limited for those patients who fail to respond to primary treatment altogether and those who initially respond and then fail. Now, he added, "these trial results demonstrate that eltrombopag may represent the potential for a new oral therapy to reduce the number of bleeding episodes in ITP patients."
Revolade, which will be marketed as Promacta in the USA if approved, was discovered as a result of research collaboration between GSK and Ligand Pharmaceuticals, and several trials of the compound are ongoing.
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