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CTI BioPharma has withdrawn its application for marketing authorization of pacritinib (Enpaxiq) in the European Union, according to the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
The company was seeking approval for pacritinib, a JAK2/FLT3 inhibitor, to treat splenomegaly or symptoms of myelofibrosis (MF) in adults with primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF.
When the application was withdrawn, the CHMP was of the provisional opinion that pacritinib could not have been approved for this indication.
Last year, CTI BioPharma withdrew its application for approval of pacritinib in the US.
Issues preventing approval
The CHMP said it had a number of concerns related to the PERSIST-1 trial, which was used to support the application for approval in the European Union. In this trial, researchers compared pacritinib to best available therapy, excluding JAK inhibitors, in patients with MF.
The CHMP said the reduction in spleen size, which was the main efficacy outcome in the study, appeared to be lower with pacritinib than with another medicine of its class, with no improvement in symptom scores.
In addition, the incidence of thrombocytopenia was higher in patients treated with pacritinib.
And more deaths occurred in patients taking pacritinib than in those receiving best available therapy, including deaths due to bleeding and adverse effects on the heart.
The CHMP also said it needs more information about the starting materials used in the manufacture of pacritinib and how the drug acts on target proteins.
Given these concerns, the CHMP was of the opinion that pacritinib’s benefits had not been shown to outweigh its risks.
CTI BioPharma said it could address the CHMP’s concerns by providing data from a second study of pacritinib, PERSIST-2.
However, there was not enough time in the current application procedure to provide the data, so the company decided to withdraw the application.
CTI BioPharma said it intends to integrate data from PERSIST-2 into its current dossier before approaching the European Medicines Agency to discuss a new application.
The company also said the withdrawal of its application will not affect patients currently enrolled in clinical trials of pacritinib or compassionate use programs for the drug.
Previous withdrawal, clinical hold
CTI BioPharma withdrew its application for approval of pacritinib in the US after the US Food and Drug Administration (FDA) placed a full clinical hold on trials of the drug.
The FDA placed the hold on pacritinib trials in February 2016 after results from PERSIST-1 and PERSIST-2 showed excess mortality in patients who received pacritinib.
The FDA lifted the hold in January 2017 after CTI BioPharma agreed to conduct dose-exploration studies for pacritinib, submit final study reports and data sets for PERSIST-1 and PERSIST-2, and make modifications to protocols and study-related documents. 
CTI BioPharma has withdrawn its application for marketing authorization of pacritinib (Enpaxiq) in the European Union, according to the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
The company was seeking approval for pacritinib, a JAK2/FLT3 inhibitor, to treat splenomegaly or symptoms of myelofibrosis (MF) in adults with primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF.
When the application was withdrawn, the CHMP was of the provisional opinion that pacritinib could not have been approved for this indication.
Last year, CTI BioPharma withdrew its application for approval of pacritinib in the US.
Issues preventing approval
The CHMP said it had a number of concerns related to the PERSIST-1 trial, which was used to support the application for approval in the European Union. In this trial, researchers compared pacritinib to best available therapy, excluding JAK inhibitors, in patients with MF.
The CHMP said the reduction in spleen size, which was the main efficacy outcome in the study, appeared to be lower with pacritinib than with another medicine of its class, with no improvement in symptom scores.
In addition, the incidence of thrombocytopenia was higher in patients treated with pacritinib.
And more deaths occurred in patients taking pacritinib than in those receiving best available therapy, including deaths due to bleeding and adverse effects on the heart.
The CHMP also said it needs more information about the starting materials used in the manufacture of pacritinib and how the drug acts on target proteins.
Given these concerns, the CHMP was of the opinion that pacritinib’s benefits had not been shown to outweigh its risks.
CTI BioPharma said it could address the CHMP’s concerns by providing data from a second study of pacritinib, PERSIST-2.
However, there was not enough time in the current application procedure to provide the data, so the company decided to withdraw the application.
CTI BioPharma said it intends to integrate data from PERSIST-2 into its current dossier before approaching the European Medicines Agency to discuss a new application.
The company also said the withdrawal of its application will not affect patients currently enrolled in clinical trials of pacritinib or compassionate use programs for the drug.
Previous withdrawal, clinical hold
CTI BioPharma withdrew its application for approval of pacritinib in the US after the US Food and Drug Administration (FDA) placed a full clinical hold on trials of the drug.
The FDA placed the hold on pacritinib trials in February 2016 after results from PERSIST-1 and PERSIST-2 showed excess mortality in patients who received pacritinib.
The FDA lifted the hold in January 2017 after CTI BioPharma agreed to conduct dose-exploration studies for pacritinib, submit final study reports and data sets for PERSIST-1 and PERSIST-2, and make modifications to protocols and study-related documents.
CTI BioPharma has withdrawn its application for marketing authorization of pacritinib (Enpaxiq) in the European Union, according to the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
The company was seeking approval for pacritinib, a JAK2/FLT3 inhibitor, to treat splenomegaly or symptoms of myelofibrosis (MF) in adults with primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF.
When the application was withdrawn, the CHMP was of the provisional opinion that pacritinib could not have been approved for this indication.
Last year, CTI BioPharma withdrew its application for approval of pacritinib in the US.
Issues preventing approval
The CHMP said it had a number of concerns related to the PERSIST-1 trial, which was used to support the application for approval in the European Union. In this trial, researchers compared pacritinib to best available therapy, excluding JAK inhibitors, in patients with MF.
The CHMP said the reduction in spleen size, which was the main efficacy outcome in the study, appeared to be lower with pacritinib than with another medicine of its class, with no improvement in symptom scores.
In addition, the incidence of thrombocytopenia was higher in patients treated with pacritinib.
And more deaths occurred in patients taking pacritinib than in those receiving best available therapy, including deaths due to bleeding and adverse effects on the heart.
The CHMP also said it needs more information about the starting materials used in the manufacture of pacritinib and how the drug acts on target proteins.
Given these concerns, the CHMP was of the opinion that pacritinib’s benefits had not been shown to outweigh its risks.
CTI BioPharma said it could address the CHMP’s concerns by providing data from a second study of pacritinib, PERSIST-2.
However, there was not enough time in the current application procedure to provide the data, so the company decided to withdraw the application.
CTI BioPharma said it intends to integrate data from PERSIST-2 into its current dossier before approaching the European Medicines Agency to discuss a new application.
The company also said the withdrawal of its application will not affect patients currently enrolled in clinical trials of pacritinib or compassionate use programs for the drug.
Previous withdrawal, clinical hold
CTI BioPharma withdrew its application for approval of pacritinib in the US after the US Food and Drug Administration (FDA) placed a full clinical hold on trials of the drug.
The FDA placed the hold on pacritinib trials in February 2016 after results from PERSIST-1 and PERSIST-2 showed excess mortality in patients who received pacritinib.
The FDA lifted the hold in January 2017 after CTI BioPharma agreed to conduct dose-exploration studies for pacritinib, submit final study reports and data sets for PERSIST-1 and PERSIST-2, and make modifications to protocols and study-related documents.