What is Brineura - Cerliponase Alfa used for and what is it used for?
Brineura is a medicine for the treatment of type 2 neuronal lipofuscinosis ceroid (CLN2), an inherited disorder in children that leads to progressive brain damage.
Because the number of patients with CLN2 is low, the disease is considered 'rare' and Brineura was designated an 'orphan medicine' (a medicine used in rare diseases) on 13 March 2013.
Brineura contains the active ingredient cerliponase alfa.
How is Brineura - Cerliponase Alfa used?
Brineura is infused directly into the brain. Before the initial infusion, the patient will have to undergo surgery to implant a device, which goes from the outside of the skull through the fluid cavity to the brain, where the medicine is released.
Infusions are performed once every two weeks by a healthcare professional experienced in administering medicines in the brain. To reduce the risk of infusion reactions, other medicines may be given to patients before or during treatment with Brineura or the infusion may be slowed. Treatment can continue until the patient benefits.
Brineura can only be obtained with a prescription. For more information, see the package leaflet.
How does Brineura - Cerliponase Alfa work?
Patients with CLN2 lack an enzyme necessary for normal brain development called tripeptidyl-peptidase 1 (TPP1). The active ingredient in Brineura, cerliponase alfa, is a copy of TPP1 and is used to replace the missing enzyme.
The medicine is infused directly into the brain in order to bypass the blood-brain barrier, a protective barrier that separates blood flow from the brain and prevents substances such as medicines from entering the brain tissue.
What benefit has Brineura - Cerliponase Alfa shown during the studies?
In the first studies it was highlighted that Brineura reduces the speed with which the disease progresses measured according to a standard evaluation scale.
In a study of 23 children (average age 4 years) treated with Brineura for almost a year, 20 of them (87%) did not show the 2-point loss in motor and linguistic abilities historically observed in patients not receiving treatment.
The evaluation was performed by doctors who assigned patients separate scores for motor and language skills (where 0 means more severe and 3 is normal). A patient's final score corresponded to the sum of the two scores.
In a follow-up study, Brineura's benefits lasted for another year; the results showed that the disease could be slowed in most patients. This study is still ongoing.
What are the risks associated with Brineura - Cerliponase Alfa?
The most common side effects of Brineura (which may affect more than 1 in 5 people) are fever, low levels of CSF protein (brain fluid and spinal cord), ECG abnormalities (an examination of cardiac activity), vomiting, upper respiratory tract infections (nose and throat infections) and hypersensitivity (allergic) reactions. For the full list of all side effects reported with Brineura, see the package leaflet.
Brineura should not be used in patients who have experienced life-threatening (allergic) hypersensitivity reactions with Brineura and whose symptoms have recurred when the medicine has been administered again. Furthermore, it should not be given to patients who have been implanted with a shunt to drain excess fluid from the brain. Finally, Brineura should not be given to patients if there are problems related to the device, such as leakage or infection.
Why has Brineura - Cerliponase Alfa been approved?
The available data show that Brineura contributes to slowing the loss of motor and language skills in patients with CLN2, a disease for which there are no other treatments.
Regarding security, the data does not reveal any unacceptable problems. The Agency's Committee for Medicinal Products for Human Use (CHMP) therefore concluded that Brineura's benefits are greater than its risks and recommended that it be approved for use in the EU.
Brineura was authorized in "exceptional circumstances" because it was not possible to obtain complete information on Brineura due to the rarity of the disease. Every year the European Medicines Agency will review the new information available and this summary will be updated accordingly.
What information is still awaited for Brineura?
As Brineura has been authorized in exceptional circumstances, the company that markets it will provide additional data from studies on the safety of the medicine, including the risk of allergic reactions when used over the long term, and on its long-term efficacy in slowing down or halting the deterioration of motor and linguistic skills. The studies will be attended by children under the age of two, for whom there are currently no data.
What measures are being taken to ensure the safe and effective use of Brineura - Cerliponase Alfa?
The company that markets Brineura will ensure that all health professionals who should use the medicine are provided with training material on how to use it and how to prevent device-related problems, such as infection or obstruction.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Brineura have also been included in the summary of product characteristics and the package leaflet.
More information on Brineura - Cerliponase Alfa
The full EPAR for Brineura can be found on the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information on Brineura therapy, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
The summary of the opinion of the Committee for Orphan Medicinal Products related to Brineura is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
