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FDA Approves Merck’s ENFLONSIA™ to Prevent RSV in Infants

The Pharma Data

FDA Approves Merck’s ENFLONSIA™ to Protect Infants from Severe RSV Illness Merck operating as MSD outside the United States and Canada, has received a significant regulatory milestone with the U.S. Food and Drug Administration (FDA) granting approval for ENFLONSIA™ (clesrovimab-cfor).

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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease 52
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Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

Muldoon, a baby boy born without the ability to process dietary protein properly, had become the first person to be treated with a customized gene editing therapy. wasn’t the first patient to receive base editing therapy. He needed a personalized, one-of-a-kind therapy. This unprecedented feat required diagnosing K.J.’s

Treatment 133
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FDA Grants Accelerated Approval to Lynozyfic™ for Relapsed or Refractory Multiple Myeloma

The Pharma Data

This accelerated pathway reflects the FDA’s recognition of an urgent medical need in advanced-stage multiple myeloma and is based on early clinical indicators such as objective response rate (ORR) and durability of response (DoR). Yancopoulos, President and Chief Scientific Officer of Regeneron.

FDA 40
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The Long Road to End Tuberculosis

Codon

The experience showed me that new therapies are needed not only to meet the targets laid out by the End TB Strategy but also to prevent drug resistance from negating the effectiveness of current therapeutics. Selman Waksman (middle) in his laboratory, sorting through soil samples with two unidentified assistants.

Vaccine 115
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A Visual Guide to Genome Editors

Codon

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. Such off-target cuts are obviously a big concern when developing human therapies, because they can cause mutations that lead to cancer or other diseases.

DNA 83
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FDA Grants Priority Review for WINREVAIR Label Update Based on ZENITH Trial

The Pharma Data

Importantly, the treatment effect emerged early in the course of therapy and continued to increase over time. Despite existing therapies, many PAH patients remain at substantial risk. Patients were randomized 1:1 to receive either WINREVAIR or placebo, both in combination with maximum-tolerated background therapy. Source link

Trials 52