DelveInsight
Jun 03, 2025
Beam’s BEAM-302 Earns FDA Orphan Drug Tag for AATD; Incannex’s IHL-42X Moves to Phase III After FDA Protocol OK; Kura and Kyowa Kirin’s Ziftomenib Gets FDA Priority Review in NPM1-Mutant AML; GSK’s Linerixibat NDA Accepted by FDA for PBC-Linked Pruritus; Ascendis’ TransCon CNP Granted FDA Priority Review for Achondroplasia
Beam Therapeutics Secures FDA Orphan Drug Designation for BEAM-302 in AATD Beam Therapeutics Inc. announced that the FDA has granted Orphan Drug Designation to its investigational therapy, BEAM-302, for the treatment of alpha-1 antitrypsin deficiency (AATD). BEAM-302 is a liver-targeting lipid nanoparticle (LNP)...
Read More...
May 28, 2025
Everything You Need to Know About Niemann-Pick Disease – From Types to Therapies
Niemann-Pick Disease (NPD) is a cluster of rare, genetically inherited lysosomal storage disorders characterized by the pathological accumulation of lipids within various organs. This buildup leads to progressive multisystem dysfunction, with severity and progression varying significantly across subtypes. While ind...
Read More...
May 27, 2025
Gilead’s TRODELVY Shows PFS Benefit in 1L Metastatic TNBC; Otsuka’s Sibeprenlimab Gets FDA Priority Review for IgA Nephropathy; Liquidia Wins FDA Nod for YUTREPIA in PAH and PH-ILD; GSK’s BLENREP Combos Get CHMP Backing in Myeloma; Relief Therapeutics Receives FDA Rare Pediatric Disease Tag for RLF-TD011
Gilead’s TRODELVY Demonstrates Significant PFS Benefit in First-Line Metastatic TNBC in ASCENT-03 Trial Gilead Sciences has announced positive topline results from the Phase III ASCENT-03 trial of TRODELVY (sacituzumab govitecan-hziy) in first-line metastatic triple-negative breast cancer (mTNBC) patients who ar...
Read More...
May 21, 2025
Unveiling Lysosomal Storage Disorders: Exploring Rare Diseases Impacting Millions Worldwide
Lysosomal Storage Disorders, or LSDs, are rare genetic conditions that may not make headlines, but they affect thousands worldwide, often with life-changing consequences. LSDs happen when the body’s lysosomes, tiny cellular recycling centers, fail to break down waste materials due to missing or faulty enzymes. This...
Read More...
May 20, 2025
Incyte’s ZYNYZ Approved as First-Line Treatment for Advanced Anal Cancer; Amneal Gets FDA Nod for BREKIYA for Migraines; FDA Accepts Travere’s sNDA for FILSPARI in FSGS; AbbVie’s EMRELIS Approved for Advanced NSCLC With High c-Met; Merck’s WELIREG Approved for Advanced PPGL in Patients 12+
Incyte’s ZYNYZ Approved by FDA as First-Line Treatment for Advanced Anal Cancer The FDA approved ZYNYZ (retifanlimab-dlwr), a PD-1 inhibitor developed by Incyte, for the treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). The approval incl...
Read More...
May 13, 2025
Eli Lilly’s ZEPBOUND Surpasses WEGOVY in SURMOUNT-5 Trial; Verastem Oncology Secures FDA Approval for AVMAPKI + FAKZYNJA in KRAS-Mutant Ovarian Cancer; Thermosome Gains Orphan Drug Designation for THE001; Beam Therapeutics Receives RMAT Status for BEAM-302 in AATD; Capsida Biotherapeutics Gets IND Clearance for STXBP1 Gene Therapy
Eli Lilly’s ZEPBOUND Outperforms WEGOVY in SURMOUNT-5 Trial Eli Lilly and Company released detailed results from the Phase IIIb SURMOUNT-5 clinical trial, showing that ZEPBOUND (tirzepatide) significantly outperformed WEGOVY (semaglutide) in weight loss among adults with obesity or overweight and at least one we...
Read More...
May 06, 2025
Abeona Secures FDA Nod for ZEVASKYN, the First Gene Therapy for RDEB; AbbVie Gains FDA Approval for RINVOQ in Giant Cell Arteritis; Lantern Pharma Advances LP-184 with IND Clearance for TNBC Trial; Ichnos Glenmark Earns FDA Fast Track for ISB 2001 in Multiple Myeloma; Rezolute Wins Breakthrough Therapy Designation for Ersodetug in Tumor-Induced Hypoglycemia
FDA Approves Abeona’s ZEVASKYN as First Cell-Based Gene Therapy for RDEB The FDA approved Abeona Therapeutics’ ZEVASKYN (prademagene zamikeracel, or pz-cel), the first and only autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). ZEVASKYN is d...
Read More...
Apr 30, 2025
Navigating U.S. Tariffs in 2025: Impacts and Strategies for Pharma and Healthcare
In 2025, the United States launched a bold new wave of tariffs, targeting imports critical to the pharmaceutical and healthcare industries. These policies, driven by a push to strengthen domestic manufacturing, impose a 10% tariff on all imported goods, alongside steeper levies of up to 245% on Chinese active pharm...
Read More...
Apr 29, 2025
Boston Scientific Pushes Forward Despite $200M Tariff Challenge; Northstrive Biosciences Gets FDA Green Light to Advance EL-22 Obesity Trial; Roche Acts Swiftly to Offset U.S. Tariff Impact with Global Engagements; Verastem Oncology Secures IND Clearance for KRAS G12D Inhibitor Trial; BlackfinBio Gets FDA OK for Phase I/II Trial of BFB-101 in SPG47
Boston Scientific Pushes Forward Despite $200M Tariff Challenge Boston Scientific has announced that tariffs are expected to add approximately $200 million to its costs this year, but the company remains confident in its ability to absorb the financial hit. Despite the tariff burden, the company reported robust ...
Read More...
Apr 23, 2025
Exploring the Impact of AI in Mental Health: How it is Going to Revolutionize Diagnosis and Treatment for a Better Tomorrow?
One in every eight people worldwide lives with a psychiatric disorder—a staggering statistic that underscores the deep and growing impact of mental illness on global health. The mental health crisis has evolved into a pressing public health emergency, fueled by increasing social pressures, lifestyle changes, enviro...
Read More...
-Agonist.png "Toll-like Receptors Agonist")
