Ceprotin: Withdrawal of the application to change the marketing authorisation

Ceprotin is a medicine that is used to treat patients with severe congenital (hereditary) protein C deficiency and for the short-term prevention of blood clotting in patients with severe congenital protein C deficiency.

Ceprotin contains the active substance human protein C. It has been authorised in the EU since July 2001.

Ceprotin was also expected to be used in patients with 'acquired' protein C deficiency. 'Acquired' means that the disease is not inherited but develops during the patient's life and may occur for different reasons such as severe infection, liver disease or vitamin K deficiency.

Ceprotin contains human protein C, extracted and purified from human plasma (the liquid part of the blood). In the body, protein C controls the generation of thrombin, one of the proteins involved in blood clotting. Protein C slows down the production of thrombin, and therefore slows down clotting. An injection of Ceprotin produces an immediate but temporary increase in levels of protein C, controlling clotting problems in protein C-deficient patients.

The applicant presented data from 2 main studies involving a total of 52 children with protein C deficiency due to severe infection, where Ceprotin was compared with placebo (a dummy treatment). Results from a retrospective study based on medical records of 94 children with congenital or acquired protein C deficiency were also provided.

The application was withdrawn while CHMP was still evaluating the initial documentation provided by the company.

As the CHMP was evaluating the initial documentation provided by the company, it had not yet made any recommendations.

In its letter notifying the Agency of the withdrawal of application, the company stated that it withdrew the application because in its preliminary assessment the CHMP was requesting additional clinical data to support the claim that Ceprotin is also effective in patients with acquired protein C deficiency.

The company informed the CHMP that there are no consequences for patients in clinical trials or compassionate use programmes.

If you are in a clinical trial or compassionate use programme and need more information about your treatment, contact the doctor who is giving it to you.

There are no consequences on the use of Ceprotin in its authorised indication.