Iptacopan for PNH, Casgevy and Lyfgenia for Sickle Cell Disease, SGT-003 in Duchenne Muscular Dystrophy, New Fingerstick Blood Collection Device

New FDA Approvals

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Here are the highlights:

1. Iptacopan (Fabhalta) for PNH: The FDA approved iptacopan (Fabhalta), the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH), a condition characterized by the destruction of red blood cells. This approval, based on the APPLY-PNH and APPOINT-PNH studies, marks a significant advancement over existing infusion-based treatments, with most patients experiencing increased hemoglobin levels without the need for blood transfusions.

2. Casgevy and Lyfgenia for Sickle Cell Disease: The FDA approved two gene therapies, Casgevy and Lyfgenia, for sickle cell disease treatment. Casgevy, using CRISPR/Cas9 technology for a one-time stem cell transplant, showed significant effectiveness in reducing severe vaso-occlusive crises in trials. Lyfgenia, employing lentiviral vector gene addition, demonstrated a high rate of resolution in severe vaso-occlusive events, marking a substantial progression in treating this condition.

3. SGT-003 for Duchenne Muscular Dystrophy: SGT-003, a gene therapy for Duchenne muscular dystrophy (DMD), received FDA fast track designation. Utilizing a novel capsid to deliver microdystrophin, this one-time intravenous therapy aims to address the underlying cause of DMD. It follows the accelerated approval of another gene therapy, Elevidys, also for DMD, highlighting advancements in the treatment of this muscular disorder.

4. New Fingerstick Blood Collection Device: The FDA has cleared a new fingerstick blood collection device, MiniDraw™, by Becton Dickinson. This less invasive method for obtaining blood samples aims to transform diagnostic testing by allowing lab-quality results for common blood tests without the need for traditional venous blood draws, offering a more patient-friendly alternative.

See the full write ups for today's episode at nascentmc.com/podcast

Intro and outro music

Garden Of Love by Pk jazz Collective