The FDA just approved the first drug capable of treating a rare form of Batten disease. Called Brineura, this recently approved drug can help treat late infantile neuronal ceroid lipofuscinosis type 2 ( CLN2). More exactly, it will treat one of the symptoms of this condition, the TPP1 deficiency.
CLN2 along with other neuronal ceroid lipofuscinoses diseases are known as Batten diseases. The CLN2 is an inherited condition that affects the nervous system. In the late infantile form, the symptoms appear at the age of 3. The first sign of the disease includes recurrent seizures, language delay, and ataxia.
Children who suffer from CLN2 can develop vision loss and muscle twitches. The disease affects motor skills like walking and sitting. Most of the individuals who suffer from this condition require a wheelchair. These children usually don’t survive past the age of 12. Batten disease is rare as it occurs in 4 of every 100,000 children in the US.
FDA Approved A Drug That Could Help Treat Batten Disease
The drug that was just approved is an enzyme replacement therapy. It has an active ingredient that replaces the TPP1 enzyme as patients that suffer from CLN2 lack it. Brineura is administered in the CSF (cerebrospinal fluid) by infusion with help from an implanted catheter and reservoir in the head. This drug needs to be administered under sterile conditions so that the risk of infection is as low as possible.
The treatment should be administered by a health care specialist that knows how to manage this drug. Brineura’s recommended dose is 300 mg once a week. The administration of the treatment can last for over 4 hours. Although it seems like a hard and complicated process, it could lower these children’s symptoms and risk of death.
Like any other drug, this Batten disease treatment has a few side effects. Some of these adverse reactions include ECG abnormalities or an increased number of white blood cells. They also number vomiting, headaches, low blood pressure, and in some cases, even seizures.
This drug was only tested on children with Batten disease that were older than 3. Younger kids should not take this treatment until further studies show it is safe.
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