NEWS

ASMD indications approved by FDA in Xenpozyme

Release time:

2022-09-09 11:18

 

 

US Food and Drug Administration ApprovedXenpozyme (Olipudasealfa)for patients with acid sphingomyelinase deficiency(ASMD)intravenous infusion of pediatric and adult patients.

Xenpozymeis a treatment for acid sphingomyelinase deficiency(ASMD)the patient's medication,ASMDIs a genetic disease, historically known as Nieman-pick diseaseA,A/BandBType. Nieman-There are three types of pick disease (A,BandC), with different genetic causes and different symptoms.Xenpozymefor treatmentA/BType orBtype of patients. It aims to treat unrelated brainASMDSymptoms.

Due to genetic mutations, peopleA,A/BandBTypeASMD's patients lack a functional enzyme, acid sphingomyelinase, which is found in lysosomes (the part of the body's cells that breaks down nutrients and other substances) and is necessary to break down certain fats. The resulting fat accumulation changes the way cells work and causes them to die, affecting the normal function of tissues and organs, including the liver, spleen, lungs, heart and brain.

Xenpozymeactive substance inWas puda alpha.It's a copy of normal acid sphingomyelinase. It is expected to replace the defective enzyme in patients, thereby reducing the accumulation of fat in lysosomes and alleviating some disease symptoms. However, because the drug cannot cross the blood-brain barrier, which separates blood from brain tissue, it is not expected to improve symptoms affecting the brain.

XenpozymeFast track, breakthrough therapy and priority review designations were obtained. It also received the Orphan Drug designation, which provides incentives to help and encourage the development of drugs for rare diseases.FDASponsors were issued rare pediatric disease priority review vouchers to encourage the development of new drugs and biologics for the prevention and treatment of rare diseases in children.

 

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