US FDA approved new medicine Brineura that contains active pharmaceutical ingredient cerliponase alfa for the treatment of specific type of batten disease. This first drug approved by US FDA to reduce the severity of symptoms like impaired walking ability in pediatric patients having age less than 3 years or more suffering from late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.
Batten disease is extremely rare kind of disease and characterized by fatal autosomal recessive neuro-degenerative disorder. It is kind of neuro-degenerative disorder and placed in a group of disorders termed as neuronal ceroid lipofuscinoses (NCLs). So far 20 genes have been identified that have linked with batten disease out of which type 3 (CLN3) has high incidence rate.
Major sign and symptoms of batten disease include vision difficulties, mood changes, variation in behavior, mental retardation, speech difficulties, seizures and poor circulation of blood in extremities. It has been observed that in females symptoms develops later than in males but their survival life span is shorter than males. Batten disease is simply a genetic disorder so, people having batten disease in their family history are more prone to batten disease.
Brineura/cerliponase alfa Side effectsAccording to data provided by US FDA drug exhibit following side effects during clinical trials
- Changes in ECG waves
- Decreased heart rate
- Increase in CSF protein
- Infection at injection site
Brineura/cerliponase alfa Uses or Patient Information
This first drug approved by US FDA for the treatment of specific form of batten and to slow down the loss of walking ability (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.
Brineura/cerliponase alfa Dosage
This drug is formulated as intraventricular injection administered via intraventricular administration kit. This kit includes
- Two 20-mL syringes
- Two syringe needles (21 ga, 25.4 mm)
- One extension line
- One infusion set with 0.2 micron inline filter
- One port needle (22 ga, 16 mm)
Standard dosage frequency of Brineura is 300mg by intraventricular infusion once every other week in patients having age 3 years or more. Drug Safety profile has not yet evaluated in patents having age less than 3 years.