This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.
After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.
Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.
Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:
One expert views Amtagvi’s approval as a catalyst for further investment in TIL therapies, akin to how Kymriah’s 2017 clearance buoyed CAR-T treatment.
Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:
Food and Drug Administration has approved the SAPIEN 3 platform, a transcatheter aortic valve replacement (TAVR) therapy, for patients with severe aortic stenosis without symptoms. The approval is the first for TAVR. MONDAY, May 5, 2025 -- The U.S.
Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A. FRIDAY, June 30, 2023 -- The U.S. The treatment, Roctavian (valoctocogene roxaparvovec), will cost $2.9 million for a single.
A group of independent experts wasn't convinced by clinical trial data from company Lykos Therapeutics, which is seeking FDAapproval of MDMA-assisted treatment for post-traumatic stress disorder.
The agency granted a long-awaited clearance on Thursday, but unexpected aspects of the hemophilia treatment’s label had some investors worried about its commercial prospects.
The FDA-approvedtherapy, called Palforzia, was little used, leading Nestle to abandon a business it had secured in a $2.6 billion deal three years ago.
Elrexfio is the third bispecific antibody cleared to treat the blood cancer, and will compete with other therapies that target the “BCMA” protein on myeloma cells.
clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.
Elsewhere, Stealth Biotherapeutics and Taysha Gene Therapies each think they've found a path to FDAapproval. Keros will also stop work on a cardiovascular disease drug.
FDAApproves Tablet Formulation of BeOne’s BRUKINSA® for All Approved Indications, Offering Greater Convenience for Patients with B-cell Cancers BeOne Medicines Ltd. Food and Drug Administration (FDA). Notably, BRUKINSA has emerged as the market leader in new CLL patient starts across all lines of therapy in the U.S.
Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder. FRIDAY, June 30, 2023 -- The U.S. The treatment is not cheap: Roctavian will.
FDAApproves Merck’s ENFLONSIA™ to Protect Infants from Severe RSV Illness Merck operating as MSD outside the United States and Canada, has received a significant regulatory milestone with the U.S. Food and Drug Administration (FDA) granting approval for ENFLONSIA™ (clesrovimab-cfor).
Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If
The company is discussing various payment models for its sickle cell treatment with government and commercial insurers ahead of an FDAapproval decision later this year.
FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Results from the study that led to the FDAapproval appeared in The Lancet Neurology in April 2024 with commentary. Researchers have been working on developing gene therapy for DMD for decades.
Valentine — On November 22, 2022, FDAapproved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Recently, afatinib, an FDA-approved kinase inhibitor, has been shown to modulate brachyury levels in multiple chordoma cell lines. In doing so, we demonstrate the feasibility of direct brachyury modulation, which may further be developed into more potent tool compounds and therapies.
FDAApproves First Oral Hormone Therapy for Advanced Prostate Cancer. 21, 2020 — Orgovyx (relugolix) is now approved to treat advanced prostate cancer and is the first oral hormone therapyapproved for this indication, the U.S. Professional. MONDAY, Dec. Food and Drug Administration announced Friday.
It showed positive synergism with a lower than MIC dose of fluconazole (0.5mg/L) proposing its use in combination therapy against Candida infections. We identified besifloxacin (BS), an FDA-approved antibacterial, as a potent antifungal inhibitor.
The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done.
Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA. In the U.S., In the U.S.,
Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). All the data described above can be found in Sections 12 and 14 of the FDA-approved prescribing information (see here ).
Despite the standard of care therapy which includes surgical resection, temozolomide chemotherapy, radiation and the newly added tumor treating fields, median survival remains only ~20 months. Glioblastoma (GBM) is the most frequently diagnosed primary CNS tumor in adults.
Jump up to: a b c d e f g h i j k l “FDAapproves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene” (Press release). Food and Drug Administration (FDA). Jump up to: a b New Drug TherapyApprovals 2023 (PDF). Food and Drug Administration (FDA) (Report).
Researchers reprogram gene therapy viral vectors to bind specific protein targets By Allessandra DiCorato July 19, 2023 Breadcrumb Home Researchers reprogram gene therapy viral vectors to bind specific protein targets A new screening method zeroes in on adeno-associated viruses that enter the brain through a defined mechanism.
Food and Drug Administration (FDA) approval for the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with need […] PARAMUS, N.J.,
2] Fitusiran was approved for medical use in the United States in March 2025. [2] 2] The fixed dose of fitusiran is not approved because it led to excessive clotting in some participants. [2] 2] The US Food and Drug Administration (FDA) granted the application for fitusiran orphan drug and fast track designations. .
Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.
Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Kymriah ® (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.
Later, a new type of non-nucleoside reverse transcriptase inhibitors (NNRTIs) were approved as anti-HIV drugs. Zidovudine, didanosine, and stavudine are FDA-approved NRTIs, while nevirapine, efavirenz, and delavirdine are FDA-approved NNRTIs.
The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. In a paper published in Molecular Therapy: Methods & Clinical Development , the team discussed why they choose this type of gene therapy to treat HIV. Air Force photo by Kemberly Groue.
The CET RAIDR program minimizes development and procurement costs by supplementing the military medical providers' toolbox with post-Phase II therapies that demonstrate established safety and manufacturing processes, leading to a cost-sparing model for niche medicines (i.e., CBRN MCMs).
Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDAapproved CAR T-cell therapy used to treat blood cancer.
“It’s important that Impulse Dynamics acted quickly in a joint effort with the FDA to remove this potential barrier standing in the way of critical diagnostic procedures for a subset of patients with HF. CCM therapy is the first approach designed to optimize heart contraction, allowing more oxygen-rich blood to reach the body.
We note that the only Phase 3 study funded in the FY2024 announcement is the first-ever late-stage clinical trial for the indication of microcystic lymphatic malformations , a serious, rare genetic skin disease with no FDA-approvedtherapies. Relative to other areas of medicine (e.g.,
Xywav: A Low-Sodium Alternative with FDAApproval Xywav is a uniquely formulated, low-sodium oxybate therapy, and remains the only product of its kind approved by the U.S. It is also approved for adult patients with idiopathic hypersomnia (IH).
We organize all of the trending information in your field so you don't have to. Join 15,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
Let's personalize your content