FDA
Nov 19, 2024
FDA Approves PTC’s AADC Gene Therapy; DUPIXENT sBLA Acceptance for Urticaria; CHMP Recommends TAGRISSO for EGFR Lung Cancer; FDA Approves DANZITEN for CML; Syndax Wins FDA Approval for REVUFORJ in Acute Leukemia
FDA Approves AADC Deficiency Gene Therapy by PTC Therapeutics PTC Therapeutics, Inc. has secured FDA accelerated approval for KEBILIDI (eladocagene exuparvovec-tneq), marking a historic milestone as the first-ever gene therapy directly administered to the brain in the United States. This innovative therapy addre...
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Nov 12, 2024
AUCATZYL Approved for R/R B-ALL; FDA Accepts NDA for Unicycive’s Oxylanthanum Carbonate; AstraZeneca and Amgen Report Positive Results in Chronic Rhinosinusitis; Nipocalimab Granted Breakthrough Designation for Sjögren’s Disease; AbbVie’s Schizophrenia Drug Fails Phase Studies
FDA Approves Autolus's AUCATZYL for Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia Autolus Therapeutics has achieved a significant milestone with FDA approval for AUCATZYL (obecabtagene autoleucel), a next-generation CAR T-cell therapy for adults with relapsed or refractory B-cell acute lymphoblastic le...
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Nov 05, 2024
Prolong’s PP-007 Fast-Tracked for Stroke; FDA Expands JYLAMVO Pediatric Approval; Corcept’s Cushing’s Drug Shows Positive Phase III Results; ESSA Halts Phase II Study of Masofaniten for Prostate Cancer; SCEMBLIX Approved for Leukemia
Prolong Pharmaceuticals Secures FDA Fast Track for PP-007 in Stroke Therapy Prolong Pharmaceuticals, LLC, a clinical-stage biopharmaceutical company, announced that its investigational therapy, PP-007 (PEGylated carboxyhemoglobin, bovine), has been granted Fast Track designation by the FDA for the treatment of a...
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Nov 01, 2024
5 Promising Pompe Disease Drugs in Early-stage Development
Imagine a disease that stealthily incapacitates the heart, liver, and skeletal muscles—slowly breaking them down due to the absence of a crucial protein. That's the story of Pompe disease, also known as glycogen storage disease type II (GSDII). Caused by a gene mutation that blocks the body’s ability to break down ...
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Oct 29, 2024
FDA Grants Orphan Status to MDL-101 for LAMA2-CMD; Pfizer’s ABRYSVO Approved for High-Risk Adults (18-59); KIND’s AND017 Gains Orphan Designation for Sickle Cell Disease; HiberCell’s HC-7366 Fast-Tracked for AML; ORLYNVAH Approved for Uncomplicated UTIs
FDA Grants Orphan Drug Status to MDL-101 for LAMA2-CMD Treatment Modalis Therapeutics Corporation has received Orphan Drug Designation (ODD) from the FDA for MDL-101, its promising treatment for congenital muscular dystrophy type 1A (LAMA2-CMD). This designation is a significant step, aimed at fostering the deve...
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Oct 22, 2024
Gilead and Merck Announce Encouraging Phase II Results of Islatravir and Lenacapavir Combo; REGENXBIO Highlights Positive Data from Phase II ABBV-RGX-314 Wet AMD Study at AAO 2024; FDA Greenlights Astellas’ VYLOY for Advanced Gastric and GEJ Cancer Therapy; Merck’s CAPVAXIVE Vaccine Yields Strong Immune Response in Adults Vulnerable to Pneumococcal Disease; FDA Postpones LUMAKRAS Colorectal Cancer Verdict to Early 2025
Gilead and Merck Reports Phase II Data Showing that Switching to a Once-Weekly Oral Combination of Islatravir and Lenacapavir Maintained Viral Suppression in Adults Through 48 Weeks Gilead Sciences, Inc. and Merck have shared new findings from a Phase II clinical trial assessing the experimental combination of i...
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Oct 18, 2024
From NASH to MASH: Unraveling the Evolution and Future of Liver Disease Treatment
In the ever-evolving world of liver disease, the shift from Nonalcoholic Steatohepatitis (NASH) to Metabolic Associated Steatotic Hepatitis (MASH) marks a revolutionary leap in our understanding and approach to treatment. This transition isn't merely a rebranding; it signifies a profound recognition of the intricat...
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Oct 16, 2024
Transforming Pompe Disease Care: Latest Innovations and Strategic Advances in Treatment
Pompe disease is a rare genetic disorder that presents a spectrum of severity, with varying rates of progression and ages of onset. Symptoms can appear anywhere from infancy to late adulthood, with earlier onset generally associated with more rapid progression and increased severity. At all ages, the disease is mar...
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Oct 15, 2024
Astellas & AviadoBio’s Exclusive Deal for AVB-101; GSK’s Depemokimab Shows Positive Results for Nasal Polyps; Lilly’s Mirikizumab Outperforms Ustekinumab in Crohn’s Study; TREMFYA Delivers Strong Results in Crohn’s & Colitis; ENHERTU Approved in China for HER2-Mutant NSCLC.
Astellas & AviadoBio Sign Exclusive Deal for Gene Therapy AVB-101 in Frontotemporal Dementia AviadoBio Ltd. and Astellas Pharma Inc. have announced a strategic partnership under an exclusive option and license agreement for AVB-101, an investigational AAV-based gene therapy currently in Phase I/II developmen...
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Oct 01, 2024
IntraBio’s AQNEURSA Niemann-Pick Disease Approval; FDA Approves Novel Schizophrenia Drug After 35 Years; Selpercatinib Gets FDA Nod for RET-Mutated MTC; DUPIXENT Receives First-Ever COPD Approval; Pfizer Withdraws OXBRYTA for Sickle Cell Disease from Global Market
IntraBio's AQNEURSA Receives Historic FDA Approval for Niemann-Pick Disease Type C Treatment IntraBio Inc. has received approval from the FDA for AQNEURSA (levacetylleucine), marking a significant milestone in the treatment of neurological manifestations of Niemann-Pick disease type C in both adults and pe...
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