FDA Approves Ctexli: First Treatment for Cerebrotendinous Xanthomatosis
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New Drug Approved for Rare Disease Neurofibromatosis Type 1-Associated Plexiform Neurofibromas
Time to read: 01:01. On February 11, 2025, the FDA approved mirdametinib (Gomekli), a kinase inhibitor, for patients aged 2 years and older with symptomatic neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PN) that are inoperable.
Source: FDA Press Release
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U.S. FDA approves AVTOZMA® (tocilizumab-anoh), a biosimilar to ACTEMRA®
Time to listen 01:04. The U.S. Food and Drug Administration (FDA) has approved Avtozma (tocilizumab-anoh), a tocilizumab biosimilar developed by Celltrion, for the treatment of multiple rheumatic conditions. It is the third biosimilar referencing Actemra (tocilizumab) to gain U.S. approval.
Source: FDA, Medscape, Biosimilars.com
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FDA approves brain injection for L-amino acid decarboxylase (AADC) deficiency
Time to read: 01:10 .The U.S. Food and Drug Administration (FDA) has approved Kebilidi (eladocagene exuparvovec-tneq), a groundbreaking gene therapy for treating aromatic L-amino acid decarboxylase (AADC) deficiency
Source: FDA
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KIND reports positive phase II results for AND017 in for treating anemia associated with CKD and receives FDA Orphan Drug designation for Sickle Cell Diseasee
Time to read: 02:44. Kind Pharmaceutical, a clinical-stage biopharma focused on haematological and cancer treatments, presented positive trial results for AND017 in treating anemia in both Nnon-dialysis dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD)at ASN Kidney Week in San Diego.
Source: PRNewwire
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First IL-23 Inhibitor shows robust results for management of Crohns Disease
Time to read: 01:33. In the Phase 3 GRAVITI study, a greater proportion of patients treated with subcutaneous TREMFYA® (guselkumab) achieved clinical and endoscopic remission at 48 weeks compared to those receiving a placebo
Source: PRNewsWire
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FDA Approves New Drug to Treat Niemann-Pick Disease, Type C
Time to read : 01:24 The U.S. FDA has approved Aqneursa (levacetylleucine) for treating neurological symptoms of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kilograms.
Source: FDA Press Release
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