FDA approves first treatment for a form of Batten disease stat : U S FDA

Its active ingredient (cerliponase alfa) is a recombinant form of human TPP1, the enzyme deficient in patients with CLN2 disease. The U.S. Food and addiction Administration today approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease. CLN2 disease affects essential motor skills, such as sitting and walking. CLN2 disease is a rare inherited disorder that primarily affects the nervous system. "Approving the first drug for the treatment of this form of Batten disease is an important advance for patients suffering with this condition."CLN2 disease is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), collectively referred to as Batten disease.




(bit.ly/2oQ3x55)CLN2 disease is one of a group of disorders collectively referred to as Batten disease. April 27 The United States Food and addiction Administration said on Thursday it had approved Biomarin Pharmaceutical Inc's drug to treat a type of Batten disease, a rare disorder that mainly affects the nervous system and is ultimately fatal. The drug, Brineura, is an enzyme replacement therapy designed to slow loss of walking ability in patients, three years or older, with CLN2 disease. (Reporting by Akankshita Mukhopadhyay in Bengaluru; Editing by Shounak Dasgupta)


The addiction, Brineura, is an enzyme replacement therapy designed to slow loss of walking ability in patients, three years or older, with CLN2 disease. The company said every year about 20 children are born in the U.S. with CLN2 disease, which is one of a group of disorders collectively referred to as Batten disease. The FDA said it had issued Biomarin a rare pediatric disease priority review voucher, which would help a drug to get rapid approval. (Adds details)April 27 (Reuters) - The U.S. Food and Drug Administration said on Thursday it had approved Biomarin Pharmaceutical Inc's drug to treat a type of Batten disease, an extremely rare disorder that mainly impacts the nervous system. Biomarin said Brineura is expected to be available in the United States by early June and the company will begin promotion of the drug immediately.


collected by :Lucy William

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