Drug Discovery World

APRIL 12, 2024

The US Food and Drug Administration (FDA) has accepted an Investigational New Drug (IND) application for Tr1X’s TRX103 for the prevention of Graft versus Host Disease (GvHD) in patients undergoing HLA-mismatched haematopoietic stem cell transplantation (HSCT). “As a result, innovative treatments are urgently needed.”

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Drug Discovery World

JUNE 12, 2023

The US Food and Drug Administration (FDA) has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).

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Drug Discovery World

NOVEMBER 29, 2023

At sites in the US, Japan, and the EU, the clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children, one to six years of age, who have a confirmed APDS diagnosis. The post Trials investigate targeted APDS treatment in a paediatric setting appeared first on Drug Discovery World (DDW).

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Drug Discovery World

AUGUST 21, 2023

Ipsen’s Sohonos (palovarotene) has become the first drug approved for patients with fibrodysplasia ossificans progressiva (FOP) in the US, following the granting of a marketing authorisation by the Food and Drug Administration (FDA). It affects an estimated 400 people in the US and 900 people globally.

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Drug Discovery World

JUNE 21, 2023

Citius Pharmaceuticals has revealed positive results from its Phase IIb Study of Halo-Lido (CITI-002) for the treatment of haemorrhoids. Following the 21 st Century Cures Act, higher emphasis is placed on using Patient Reported Outcome (PRO) instruments in clinical trials.

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Drug Discovery World

AUGUST 8, 2024

Two veteran advocacy organisations, Reason for Hope and the Veteran Mental Health Leadership Coalition, have issued a joint statement urging the US Food and Drug Administration (FDA) to approve MDMA-assisted therapy for post-traumatic stress disorder (PTSD).

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Drug Discovery World

AUGUST 20, 2024

The US Food and Drug Administration (FDA) has approved axatilimab-csfr (Niktimvo), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD).

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Drug Discovery World

AUGUST 5, 2024

Theriva Biologics’ VCN-01 for retinoblastoma has been granted rare paediatric drug designation (RPDD) by the US Food and Drug Administration (FDA). The FDA grants RPDD for rare diseases (fewer than 200,000 affected persons in the United States) that are serious and life-threatening and primarily affect children ages 18 years or younger.

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Drug Discovery World

AUGUST 28, 2024

It is approved for the first-line treatment of adult patients with unresectable or metastatic cancer, who are eligible for platinum-containing chemotherapy. I look forward to seeing the treatment combination implemented as a first-line regimen in the clinical setting.” months compared to 16.1 The median PFS of 12.5

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Drug Discovery World

OCTOBER 11, 2023

Collaborators Boehringer Ingelheim and CDR-Life have commenced a Phase I evaluation of BI 771716, their antibody fragment-based treatment developed to preserve the vision of people with geographic atrophy (GA).

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Drug Discovery World

AUGUST 22, 2024

The US Food and Drug Administration (FDA) has approved lazertinib (Lazcluze) in combination with amivantamab-vmjw (Rybrevant) for the first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC).

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Drug Discovery World

OCTOBER 4, 2022

The US Food and Drug Administration (FDA) has granted approval to AMX0035, a new treatment for people living with amyotrophic lateral sclerosis (ALS). million from the ALS Association, raised by the 2014 social media craze the ‘Ice Bucket Challenge’ Another 40 potential new treatments are being investigated. .

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Drug Discovery World

AUGUST 14, 2024

The US Food and Drug Administration (FDA) has approved a Phase II clinical trial for Biosyngen’s BRG01, an EBV-specific CAR-T cell therapy. This marks the first cell therapy to enter Phase lI trials in both the US and China for the treatment of relapsed/metastatic EBV-positive nasopharyngeal carcinoma.

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Drug Discovery World

AUGUST 8, 2024

The first cohort of participants has been dosed in a Phase I trial MTX-474, a human IgG1 antibody designed to neutralise EphrinB2 signalling to treat fibrosis. I look forward to completion of the Phase I studies and future planned Phase II trials in systemic sclerosis and IPF, respectively.”

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Drug Discovery World

JULY 14, 2022

Biotech company Actigen has moved closer to clinical trials for rare disease treatment for Hunter syndrome, following a pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA). ? . Hunter syndrome . Official comments .

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Drug Discovery World

SEPTEMBER 13, 2022

The US Food and Drug Administration (FDA) has approved Bristol Myers Squibb’s allosteric tyrosine kinase 2 inhibitor Sotyktu (deucravacitinib) for the treatment of adults with moderate-to-severe plaque psoriasis. The post FDA approves new first-line treatment for plaque psoriasis appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 8, 2024

The US Food and Drug Administration (FDA) has revealed plans to convene a meeting of the Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) to discuss the Phase III TRAILBLAZER-ALZ 2 trial. The trial evaluated the efficacy and safety of Eli Lilly’s donanemab in early symptomatic Alzheimer’s disease.

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Drug Discovery World

APRIL 7, 2023

Sequana Medical has submitted an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for its second-generation DSR product (DSR 2.0) for the treatment of congestive heart failure. The post FDA reviews IND for congestive heart failure treatment appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JUNE 21, 2024

Allogene Therapeutics and Foresight Diagnostics have announced the initiation of a Phase II trial evaluating the use of cemacabtagene ansegedleucel (cema-cel) as part of the first line treatment regimen for newly diagnosed large B-cell lymphoma (LBCL) patients who are likely to relapse.

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Drug Discovery World

MAY 6, 2024

Food and Drug Administration (FDA). The company’s lead therapeutic peptide candidate is currently in a Phase III clinical trial for the treatment of hypoparathyroidism. Amolyt Pharma has announced that its lead therapeutic peptide candidate, eneboparatide, has been granted fast track designation by the U.S.

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Drug Discovery World

NOVEMBER 30, 2023

The Food and Drug Administration (FDA) said it will investigate the safety of BCMA- or CD19-directed autologous CAR-T cell immunotherapies, following reports of T cell malignancies, including chimeric antigen receptor CAR-positive lymphoma, in these patients.

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Drug Discovery World

JUNE 23, 2023

The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD. The post FDA approves first gene therapy for Duchenne muscular dystrophy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 1, 2024

The US Food and Drug Administration (FDA) has announced its final decision to withdraw approval of Pepaxto (melphalan flufenamide), despite an appeal by manufacturer Oncopeptides. Oncopeptides was required to conduct the OCEAN trial as a post-approval requirement under the accelerated approval programme.

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Drug Discovery World

JUNE 25, 2024

The US Food and Drug Administration (FDA) has expanded its approval for Elevidys (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least four years of age. The FDA granted accelerated approval for non-ambulatory patients.

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Drug Discovery World

FEBRUARY 29, 2024

The stock market value of Minerva Biosciences has dropped nearly 60%, following the US Food and Drug administration (FDA) decision not to approve roluperidone for symptoms of schizophrenia. The company received a ‘Complete Response Letter’ from the FDA highlighting the clinical deficiencies in its new drug application (NDA).

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Drug Discovery World

OCTOBER 12, 2023

The US Food and Drug Administration (FDA) has granted clearance to Transcenta to proceed with its TranStar 301 global Phase III pivotal trial of Osemitamab (TST001) for gastric cancer. The trial will investigate the drug in combination with Nivolumab and chemotherapy as a first-line treatment in patients with HER2-negative, CLDN18.2

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Drug Discovery World

AUGUST 5, 2024

Peter Marks, Director of the Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER), said the approval of the “state-of-the-art immunotherapy technology” provides a “critical new option for a patient population in need”. The median duration of response was six months.

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Drug Discovery World

MARCH 26, 2024

The US Food and Drug Administration (FDA) has granted emergency use authorisation (EUA) to half-life extended monoclonal antibody (mAb) Pemgarda (pemivibart, or VYD222) for the pre-exposure prophylaxis (prevention) of Covid-19.

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Drug Discovery World

DECEMBER 5, 2023

It has been granted approval by the Food and Drug Administration (FDA) to treat patients who have received at least two lines of therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor and a BCL-2 inhibitor. Jaypirca is the first FDA-approved non-covalent (reversible) BTK inhibitor.

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Drug Discovery World

AUGUST 11, 2023

The US Food and Drug Administration (FDA) has approved Izervay (avacincaptad pegol intravitreal solution) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Slowing of disease progression was observed as early as six months with up to a 35% reduction in the first year of treatment.

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Drug Discovery World

JANUARY 24, 2024

The Phase IIa ACCENT trial has launched to explore the activity of focal adhesion kinase (FAK) inhibitor narmafotinib in combination with standard-of-care chemotherapy in advanced pancreatic cancer patients. The trial will initially enrol 26 patients and an interim analysis of efficacy will then be conducted in mid-2024.

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Drug Discovery World

JANUARY 4, 2024

AstraZeneca and Ionis’ Wainua (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults (hATTR-PN or ATTRv-PN). The post Wainua approval marks important milestone in amyloidosis treatment appeared first on Drug Discovery World (DDW).

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Drug Discovery World

FEBRUARY 19, 2024

The US Food and Drug Administration has approved Amtagvi (lifileucel), the first cellular therapy indicated for the treatment of metastatic, unresectable melanoma. The approval of Amtagvi represents the culmination of scientific and clinical research efforts leading to a novel T cell immunotherapy for patients with limited treatment options.”

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Drug Discovery World

AUGUST 2, 2022

Biotechnology company Calidi Biotherapeutics has announced that a Phase I US clinical trial will use the company’s licensed oncolytic virotherapy platform NeuroNova to deliver an oncolytic adenovirus selectively to tumor sites in patients with recurrent high-grade glioma. .

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Drug Discovery World

JANUARY 25, 2024

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Pleco Therapeutics’s lead compound PTX-252 for the treatment of acute myeloid leukaemia (AML). The FDA’s designation acknowledges the potential of PTX-252 in addressing AML, a rare blood cancer with a poor prognosis.

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Drug Discovery World

JULY 25, 2023

The US Food and Drug Administration (FDA) has approved Daiichi Sankyo’s quizartinib (Vanflyta) for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3 internal tandem duplication (ITD)-positive. The FDA also approved LeukoStrat CDx FLT3 Mutation Assay as a companion diagnostic for Vanflyta.

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Drug Discovery World

DECEMBER 20, 2023

The US Food and Drug Administration (FDA) has approved antibody-drug conjugate (ADC) enfortumab vedotin-ejfv (Padcev) in combination with pembrolizumab (Keytruda) for patients with locally advanced or metastatic urothelial cancer (la/mUC). In the Phase III EV-302 trial, median overall survival (OS) was 31.5 months compared to 6.3

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