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Genetic Disorders

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Notizia - Recent Pharma, Healthcare and Biotech Happenings

X4 Pharmaceuticals’ XOLREMDI FDA Approval; ONO to Acquire Deciphera Pharmaceuticals; Johnson & Johnson’s SIRTURO CHMP Approval; BeiGene’s Tislelizumab EC Approval; Sanofi’s Rilzabrutinib LUNA 3 Phase III Trial

FDA Greenlights XOLREMDI Capsules from X4 Pharmaceuticals for WHIM Syndrome Treatment X4 Pharmaceuticals has declared that the FDA has granted approval for XOLREMDI™ (mavorixafor) capsules to be utilized in individuals aged 12 and above who have WHIM syndrome (characterized by warts, hypogammaglobulinemia, infections, and myelokathexis). This approval aims to enhance the count of mature neutrophils and lymphocytes circulating in the bloodstream. XOLREMDI, an inhibitor of the selective CXC chemokine receptor 4 (CXCR4), represents a breakthrough in treating WHIM syndrome, a rare disorder combining primary immunodeficiency and chronic neutropenia due to dysfunction...

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Pharma News for Cytokinetics, Chiesi, AstraZeneca, Zevra

Cytokinetics Announces Results From SEQUOIA-HCM Clinical Trial of Aficamten; FDA Approves Chiesi’s FILSUVEZ Topical Gel; Bristol Myers Squibb Adds Premier Radiopharmaceutical Platform; AstraZeneca to Acquire Gracell; Zevra Therapeutics Announces Resubmission of Arimoclomol New Drug Application; Innovent Dosed First Participant in Phase 3 Clinical Study (GLORY-2) of Mazdutide (IBI362)

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Pharma News for Merck, Ipsen, bluebird bio

Merck and Moderna Initiate Study to Evaluate V940; FDA Approves Vertex and CRISPR Therapeutics’ CASGEVY for SCD; Novartis Updated on its Investigational Iptacopan Phase III Study; FDA Grants Priority Review for New Drug Application for Elafibranor; FDA Approves bluebird bio’s LYFGENIA for Patients SCD; FDA Fast Track Designation for DMD Gene Therapy

Pharma News for AskBio, Almirall, Bayer, Pfizer

Key Updates on Phase 1 Trial of AB-1005 Gene Therapy for Multiple System Atrophy-Parkinsonian Type; European Commission Approves EBGLYSS; Bayer Stopped OCEANIC-AF Study; Pfizer and Astellas’ XTANDI Approved by FDA; FDA Orphan Drug Designation to Epic Bio’s EPI-321; FDA Fast Track Designation to Chemomab’s CM-101 for PSC

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AAV Gene Therapies for Hemophilia B Treatment: The Road to a Cure

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Can Novo’s Rivfloza Outperform Its Rival Alnylam’s Oxlumo in Primary Hyperoxaluria Treatment Space?

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Netherton Syndrome: Unraveling the Uncertainties of a Rare Genetic Skin Disorder

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A Turning Point: INGREZZA’s Impact on Huntington’s Disease Treatment and the Rise of Novel Therapies

Pharma News for Faron, Roche, Ergomed, Menarini

Daiichi Sankyo’s Trastuzumab Deruxtecan; ODD to Bexmarilimab for AML; Roche’ Alecensa; Ergomed Aims To Go Private; Tagraxofusp Receives ODD in Japan for BPCDN; FDA Fast Track Designation to Abliva’s KL1333

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