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    Home > Active Ingredient News > Study of Nervous System > Pfizer's DMD gene therapy fordadistrogene movaparvovec was shelved by the FDA after the patient died

    Pfizer's DMD gene therapy fordadistrogene movaparvovec was shelved by the FDA after the patient died

    • Last Update: 2022-01-08
    • Source: Internet
    • Author: User
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    After the death of one patient in the Phase Ib study, the FDA shelved the clinical trial of Pfizer's Duchenne Muscular Dystrophy (DMD) drug candidate fordadistrogene movaparvovec
    .


    Pfizer said that during the review of the data with the external data monitoring committee (DMC), screening and dosing in the study have been suspended


    FDA Pfizer stated that screening and dosing in the study have been suspended during the review of the data in collaboration with the external data monitoring committee (DMC)


    DMD is a serious sex-linked genetic muscular dystrophy.
    It is caused by mutations in the dystrophin gene, leading to severe muscle atrophy and heart or respiratory failure at the age of 30
    .


    Male patients begin to have muscle weakness symptoms at about 4 years old, after which the symptoms begin to deteriorate rapidly


    fordadistrogene movaparvovec is an in vivo gene therapy, administered in a single dose, using a recombinant adeno-associated virus serotype 9 (rAAV9) capsid to deliver a shortened human dystrophin gene under the control of a human muscle-specific promoter
    .

    fordadistrogene movaparvovec is an in vivo gene therapy, administered in a single dose, using a recombinant adeno-associated virus serotype 9 (rAAV9) capsid to deliver a shortened human dystrophin gene under the control of a human muscle-specific promoter
    .


    fordadistrogene movaparvovec is an in vivo gene therapy, administered in a single dose, using a recombinant adeno-associated virus serotype 9 (rAAV9) capsid to deliver a shortened human dystrophin gene under the control of a human muscle-specific promoter


    Earlier this year, Pfizer was forced to modify the clinical trial protocol, restricting the participants of the ongoing CIFFREO trial because of the occurrence of three severe adverse events (SAE) of muscle weakness including two cases of myocarditis.

    .


    According to Pfizer, certain mutations in the dystrophin gene may be associated with a higher risk of SAE after treatment, with mutations affecting exons 9 to 13 (exon deletion, exon duplication, insertion or Point mutations) or the deletion of both exon 29 and exon 30 will be excluded from the clinical trial


     

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