Opaxio

Application withdrawn

The application for this medicine has been withdrawn

Overview

On 21 September 2009, CTI Life Sciences Ltd officially notified the Committee for Medicinal Products for Human Use (CHMP) that it wishes to withdraw its application for a marketing authorisation for Opaxio, for the first-line treatment of patients with advanced non-small cell lung cancer who have an ECOG performance status of 2.

What is Opaxio?

Opaxio is a powder that is made up into a solution for infusion (drip into a vein). It contains the active substance paclitaxel poliglumex.

What was Opaxio expected to be used for?

Opaxio was expected to be used in patients with advanced non-small cell lung cancer who have an ECOG performance status of 2. The ECOG performance status is a measure of how ill a cancer patient is. A performance status of 2 means that the patient can take care of him or herself without help but is too ill to do any work.

How is Opaxio expected to work?

The active substance in Opaxio, paclitaxel poliglumex, is expected to be converted into paclitaxel in the cancer cells. Paclitaxel works by blocking the ability of cancer cells to break down the internal 'skeleton' that allows them to divide and multiply. With their skeleton still in place, the cells cannot divide and they eventually die.

Paclitaxel is an anticancer medicine which has been available in the European Union since 1993. In Opaxio, paclitaxel is attached to poliglumex to make it more soluble.

What documentation did the company present to support its application to the CHMP?

The effects of Opaxio were first tested in experimental models before being studied in humans. The company presented results of one main study involving 477 patients with advanced non-small cell lung cancer and a performance status of 2. In this study, Opaxio was compared with gemcitabine or vinorelbine (two other anticancer medicines). The main measure of effectiveness was how long the patients lived.

How far into the evaluation was the application when it was withdrawn?

The application was at day 180 when the company withdrew. After the CHMP had assessed the responses from the company to a list of questions, there were still some unresolved issues. The CHMP normally takes up to 210 days to evaluate a new application. Based on the review of the initial documentation, the CHMP prepares a list of questions at day 120, which is sent to the company. Once the company has supplied responses to the questions, the CHMP reviews them and may, before giving an opinion, ask any remaining questions at day 180. Following the CHMP's opinion, it usually takes around two months for the European Commission to issue a decision on this opinion.

What was the recommendation of the CHMP at that time?

Based on the review of the data and the company's response to the CHMP list of questions, at the time of the withdrawal, the CHMP had some concerns and was of the provisional opinion that Opaxio could not have been approved for the first-line treatment of patients with advanced non-small cell lung cancer who have an ECOG performance status of 2.

What were the main concerns of the CHMP?

The CHMP noted that the main study did not show that Opaxio was effective in patients with advanced non-small cell lung cancer who have an ECOG performance status of 2. The company's view that the study showed that Opaxio was as least as good as the comparators was not accepted by the Committee because it was not clear that the comparators themselves were effective in the type of patients involved in the main study. In addition, the studies did not show that Opaxio was more effective than the two comparators.

The CHMP also had concerns about the side effects of the medicine, especially neuropathy (damage to the nerves) and unexplained deaths. There were also concerns about impurities in the medicine and about the way paclitaxel is released and distributed in the body when Opaxio is given.

What were the reasons given by the company for withdrawing the application?

The letter from the company notifying the CHMP of the withdrawal of the application is available under the tab 'All documents'.

What are the consequences of the withdrawal for patients in clinical trials or compassionate use programmes using Opaxio?

The company informed the CHMP that the withdrawal has no impact on patients in the European Union (EU) as there are no ongoing clinical trials or compassionate use programmes in the EU. However, outside the EU, the company is continuing to study Opaxio in several cancers including ovarian, oesophageal and non-small cell lung cancer.

Questions and answers on the withdrawal of the marketing authorisation application for Opaxio (paclitaxel poliglumex)

Reference Number: EMEA/654674/2009

Key facts

• Name of medicine:
  Opaxio
• Therapeutic area (MeSH):
  Carcinoma, Non-Small-Cell Lung
• EMA product number:
  EMEA/H/C/000994
• Marketing authorisation applicant:
  CTI Life Sciences Ltd
• Withdrawal of application:
  21/09/2009

All Documents

Withdrawal assessment report for Opaxio

AdoptedReference Number: EMA/CHMP/731026/2009

Withdrawal letter : Opaxio