FDA Updates for the Week of Feb. 12, 2024: New Approvals, an Extended Review and Goal Dates Set
February 17th 2024The FDA approves Eohilia for eosinophilic esophagitis and Onivyde for metastatic pancreatic cancer but extends review of gene therapy for rare immune disorder. The agency also set goal dates for three products: full approval for Elevidys, Augtyro for solid tumors with NTRK gene fusions and seladelpar as for rare liver disease.
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FDA Sets Date for Full Approval, Broader Indication for Elevidys
February 16th 2024The FDA has set a PDUFA date of June 21, 2024, to convert the accelerated approval to full approval and expand the labeling of the gene therapy Elevidys beyond the current use in children aged 4 and 5 with Duchenne muscular dystrophy.
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FDA Updates for the Week of Feb. 5, 2024: Review Dates and Advisory Committee Meetings Scheduled
February 10th 2024The FDA has set PDUFA dates for several products: Arexvy to prevent RSV in 50 to the 59 population, for acoramidis in heart failure indication, and for an engineered tissue for vascular replacement. The agency has also scheduled an advisory committee meeting for Abecma in supplemental multiple myeloma indication. Additionally, Amneal has resubmitted its application for novel Parkinson’s therapy.
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10 State Medicaid Programs Include Pheburane for Urea Cycle Disorders
January 23rd 2024Current treatments for urea cycle disorders, which causes ammonia to build up in the blood, have a bitter taste and smell. Pheburane was developed with a special coating that masks the taste of sodium phenylbutyrate. It has a list price of $4,375 per bottle.
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Important Vaccines Approved in 2023, But Vaccine Hesitancy Persists
January 19th 2024Last year saw the approval of several firsts, including the first vaccines to prevent respiratory syncytial virus in older adults and infants and a first vaccine to prevent the mosquito-borne virus chikungunya.
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