Cell Therapeutics, Inc. (CTI), a biopharmaceutical company, announced that the National Institute for Health and Care Excellence (NICE), a non-departmental public body of the Department of Health in the United Kingdom, issued second draft guidance on Pixuvri (pixantrone) as a monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive B-cell non-Hodgkin lymphoma (aggressive B-cell NHL).
NICE's independent Appraisal Committee met on September 11, 2013, to consider the cost effectiveness of pixantrone taking into consideration CTI's initial patient access scheme that was approved by the Department of Health in July 2013. The result is a second appraisal consultation document, or ACD, whereby the Committee concluded that this scheme does not overcome the uncertainties in the evidence for pixantrone's (Pixuvri) clinical effectiveness and once again requests that consultees, including CTI, healthcare professionals and members of the public, comment on the draft guidance via the NICE website. The ACD consultation will close on November 4, 2013, and any comments received will be considered by the NICE appraisal committee to enable them to develop the next stage of guidance. It should be noted that this is not NICE's final guidance on pixantrone and that a third Appraisal Committee meeting is expected to be held on November 13, 2013, where subject to approval, CTI hopes the Committee will consider an 'enhanced' patient access scheme to demonstrate the cost effectiveness of pixantrone for use by the NHS in the UK.
In May 2012, the European Commission (EC) granted conditional marketing authorization in the European Union (EU) for Pixuvri as a monotherapy for adult patients with multiply relapsed or refractory aggressive B-cell NHL based on the results of the EXTEND, or PIX301, pivotal randomized phase 3 clinical trial. Pixuvri was made available to patients in eight countries in the European Union in the fourth quarter of 2012, and some patients in other countries have already started to receive the treatment. Prior to the approval of Pixuvri in the EU, there were no approved agents or standard of care in this stage of the disease. The PIX301 trial was designed utilizing agents in the comparator arm that have anti-tumor activity in relapsed disease and are typically employed as palliative therapy for these patients.
"We look forward to collaborating with NICE during the final stage of the process as we seek to bring this new approved therapy to patients in the UK with aggressive non-Hodgkin lymphoma in the 3rd line salvage setting," said James A. Bianco, president and CEO of CTI.
Pixuvri is a novel aza-anthracenedione with unique structural and physiochemical properties. Unlike related compounds, Pixuvri forms stable DNA adducts and in preclinical models has superior anti-lymphoma activity compared to related compounds. Pixuvri was structurally designed so that it cannot bind iron and perpetuate oxygen radical production or form a long-lived hydroxyl metabolite -- both of which are the putative mechanisms for anthracycline induced acute and chronic cardiotoxicity. These novel pharmacologic properties allow Pixuvri to be administered to patients with near maximal lifetime exposure to anthracyclines without unacceptable rates of cardiotoxicity.
In May 2012, the European Commission (EC) granted conditional marketing authorization for Pixuvri as a monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive NHL. The benefit of Pixuvri treatment has not been established in patients when used as fifth line or greater chemotherapy in patients who are refractory to last therapy. The Summary of Product Characteristics (SmPC) has the full prescribing information, including the safety and efficacy profile of Pixuvri in the approved indication. The SmPC is available at www.Pixuvri.eu. Pixuvri does not have marketing approval in the United States.
Similar to accelerated approval regulations in the United States, conditional marketing authorizations are granted in the EU to medicinal products with a positive benefit/risk assessment that address unmet medical needs and whose availability would result in a significant public health benefit. A conditional marketing authorization is renewable annually. Under the provisions of the conditional marketing authorization for Pixuvri, CTI will be required to complete a post-marketing study aimed at confirming the clinical benefit previously observed.
The European Medicines Agency's (the EMA) Committee for Medicinal Products for Human Use has accepted PIX306, CTI's ongoing randomized controlled phase 3 clinical trial, which compares Pixuvri-rituximab to gemcitabine-rituximab in patients who have relapsed after one to three prior regimens for aggressive B-cell NHL and who are not eligible for autologous stem cell transplant. As a condition of approval, CTI has agreed to have available the PIX306 clinical trial results by June 2015.
CTI is a biopharmaceutical company committed to the development and commercialization of an integrated portfolio of oncology products aimed at making cancer more treatable. \
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