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 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 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

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 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

JUNE 21, 2024

A new machine learning (ML) software platform has been launched to facilitate wider adoption of advanced and adaptive clinical trials. Novel Response Adaptive Randomization (RAR) capabilities that support multi-interims in a trial while maintaining interactivity and accuracy at the highest standards.

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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

DECEMBER 19, 2023

The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.

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ProRelix Research

AUGUST 3, 2023

Although the United States Food and Drug Administration (FDA) has always supported and advocated the idea of decentralized trials, the real adoption and application of decentralized aspects in trials has […] The post FDA Guidance on Advancement of Decentralized Clinical Trials appeared first on ProRelix Research.

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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

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

JULY 8, 2024

Biopharmaceutical company Syncromune has had its lead-candidate for the treatment of metastatic castrate-resistant prostate cancer (mCRPC) granted Fast Track designation by the FDA. We look forward to initiating trials at multiple US sites later this year to expand our efforts to develop the SYNC-T SV-102 Therapy.”

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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. Phase III EMBARK trial Consistent with the accelerated approval pathway, the company has committed to the completion of a confirmatory trial.

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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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BioPharma Drive: Drug Pricing

JUNE 20, 2024

The agency has lifted a partial trial suspension based on one-year data showing PTC’s pill suppressed a key protein associated with the disorder.

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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. . Official comments .

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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

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

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

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

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

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). The FDA approval of Izervay is great news for the retina community and our patients suffering from GA.” GA impacts an estimated 1.5

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Drug Discovery World

FEBRUARY 19, 2024

Amtagvi is the first FDA-approved tumour-derived T cell immunotherapy. Amtagvi was approved through the Accelerated Approval pathway, which gives patients the opportunity for earlier access to a promising therapy while the company conducts further trials to verify the predicted clinical benefit.

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Drug Discovery World

AUGUST 18, 2023

The US Food and Drug Administration (FDA) has granted accelerated approval to Pfizer’s Elrexfio (elranatamab-bcmm) for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM).

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Drug Discovery World

OCTOBER 3, 2023

The US Food and Drug Administration (FDA) has announced a pilot programme with the aim of accelerating the development of novel drug and biological products for rare diseases. The FDA will be accepting applications to the START programme between 2 January 2024 and 1 March 2024.

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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

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

JUNE 27, 2023

The US Food and Drug Administration (FDA) has published new draft guidance to highlight fundamental considerations to researchers investigating the use of psychedelic drugs for the potential treatment of medical conditions, including psychiatric or substance use disorders.

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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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Drug Discovery World

FEBRUARY 27, 2024

Arvinas has commenced dosing in the Phase I clinical trial of ARV-102, its first oral PROTAC (PROteolysis-TArgeting Chimera) protein degrader in development to treat neurodegenerative diseases. The post First PROTAC degrader for neurodegenerative disease enters trials appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 1, 2023

SpringWorks Therapeutics’ Ogsiveo (nirogacestat) has become the first drug to be approved for desmoid tumours in the US, following the go-ahead from the US Food and Drug Administration (FDA). The post FDA approves first therapy for rare non-cancerous tumours appeared first on Drug Discovery World (DDW).

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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

NOVEMBER 23, 2023

Minoryx has announced enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in the US Phase III clinical trial, CALYX. This will push the Phase III CALYX trial forward and bring leriglitazone to patients as quickly as possible,” said Sílvia Pascual, VP Clinical Development, Minoryx. “In

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Drug Discovery World

JUNE 8, 2023

Grown in vitro, these cells can provide patient specific human brain models from a large cohort of AD patients – to create a ‘clinical trial in a dish’. The recent US Federal Food and Drug Administration Agency (FDA) Modernization Act 2.0

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ProRelix Research

NOVEMBER 14, 2023

The growing impetus for the conduct of clinical trials outside the United States for products and medical devices intended to be used in the US include multiple reasons such as […] The post US FDA Foreign Clinical Trial Data Requirements appeared first on ProRelix Research.

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Drug Discovery World

AUGUST 31, 2023

The US Food and Drug Administration (FDA) has expanded the use of Bristol Myers Squibb’s Reblozyl (luspatercept-aamt) to include first-line treatment of anaemia in adults with lower-risk myelodysplastic syndromes (MDS). In the COMMANDS trial, results showed 58.5% of patients treated with Reblozyl vs. 31.2%

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