Drug Discovery World

NOVEMBER 24, 2022

The drug is approved for the treatment of adults with haemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening haemorrhage or have repeated, serious spontaneous bleeding episodes. . A unique approach. at 24 months post infusion.

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Gene Therapy FDA Approval Gene Clinical Trials 52

pharmaphorum

MAY 16, 2022

The findings were made via Lilly’s SURPASS phase 3 global clinical development programme, which comprised studies ranging from 40 to 52 weeks. These examined Mounjaro in 5mg, 10mg and 15 mg doses in five clinical trials, used either as a stand-alone therapy or as an add-on to other diabetes medicines.

FDA Approval 102

FDA Approval Insulin Drugs Hormones 102

Pharmaceutical Technology

NOVEMBER 30, 2022

This is the first approval for Biktarvy in paediatric indication in the EU and applies to all 27 EU member states, Liechtenstein, Norway and Iceland. The latest development is based on data obtained from an open-label clinical trial of Biktarvy.

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Marketing FDA Approval Clinical Trials Clinical Trials 246

Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). The post FDA approves gene therapy for rare neurological disorder appeared first on Drug Discovery World (DDW).

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Gene Therapy FDA Approval Gene Clinical Trials 52

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

Trials 52

Trials Clinical Trials Clinical Trials FDA Approval 52

pharmaphorum

JUNE 1, 2022

Medical imaging specialist Median Technologies has launched a new business unit that will provide decision-making tools – underpinned by artificial intelligence – for sponsors of clinical trials in oncology. The post Median Tech debuts AI-focused unit for cancer trial support appeared first on. sensitivity and 93.3%

Trials 83

Trials Clinical Trials Clinical Trials FDA Approval 83

XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases.

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Gene Therapy Clinical Trials Clinical Trials Gene 52

Pharmaceutical Technology

AUGUST 18, 2022

The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study. The open-label, single-arm, 24-month Phase III trials enrolled 41 subjects of the age four to 34 years with non-?0/?0 0 genotypes.

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Gene Therapy Gene Genotype Genetics 246

Advarra

JUNE 20, 2023

of new cancer drugs tested in Phase I were likely to receive Food and Drug Administration (FDA) approval. Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54

Trials 52

Trials Clinical Trials Clinical Trials FDA Approval 52

pharmaphorum

AUGUST 10, 2020

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA) drug Evrysdi (risdiplam). The FDA approved Evrysdi for the treatment of spinal muscular atrophy (SMA) in adults and children two months of age and older.

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FDA Approval Drugs Gene Therapy Protein 57

XTalks

APRIL 24, 2023

Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases. Reference: Chung DC, et al.

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Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

AUGUST 5, 2022

In June, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicine Agency (EMA) recommended approving the treatment in this setting. This EC approval was based on findings from the parallel-group, double-blind, placebo-controlled, global Phase III OlympiA clinical trial of Lynparza versus placebo.

FDA Approval 147

FDA Approval DNA Clinical Trials Clinical Trials 147

Pfizer

JULY 8, 2022

FDA Approval of their COVID-19 Vaccine COMIRNATY® For Adolescents 12 through 15 Years of Age. FDA Approval of their COVID-19 Vaccine COMIRNATY® For Adolescents 12 through 15 Years of Age. Favorable safety profile observed across more than 2,200 adolescents who participated in the clinical trial.

FDA Approval 102

FDA Approval Vaccination Vaccine Clinical Trials 102

pharmaphorum

FEBRUARY 21, 2022

One study found that only 5% of Black or Asian United Kingdom residents had ever participated in a clinical trial. A study of FDA-approved vaccine trials from 2011-2020 showed that 78% of participants were white, even though only 60% of the U.S. Clinical trial diversity improved during COVID-19 (but not enough) .

Clinical Research 107

Clinical Research Research Clinical Trials Clinical Trials 107

The Pharma Data

NOVEMBER 30, 2020

Two of the three planned late-stage efficacy trials for NVX-CoV2373 sponsored by Novavax are fully enrolled, and more than 20,000 participants have been dosed to-date. In alignment with Novavax’ commitment to transparency, Phase 3 clinical trial protocols are posted to the company’s website at Novavax.com/resources upon finalization.

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Clinical Development Vaccination Vaccine Development 52

Pharmaceutical Technology

SEPTEMBER 23, 2022

cyclosporine A) is currently at pre-registration stage with the FDA, following results from late-stage clinical trials that demonstrated CyclASol significantly reduced corneal and conjunctival staining, and improved ocular dryness compared with vehicle. Novaliq’s CyclASol (0.1%

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Development Drugs Clinical Trials Clinical Trials 147

The Pharma Data

MAY 26, 2023

Pfizer’s PAXLOVID™ receives FDA approval for adult patients at high risk of progression to severe COVID-19 Pfizer Inc. NYSE: PFE) announced today that the U.S. ” COVID-19 continues to cause significant burden in the U.S. Source link: [link]

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FDA Approval HR Vaccination Vaccine 40

pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one of three UK sites for the clinical trial and part of 55 globally, across 15 countries.

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Gene Therapy Gene Trials Protein 80

XTalks

JULY 3, 2023

We are delighted with the FDA’s approval of Suflave — which has efficacy equivalent to Suprep, but with a taste similar to a sports drink,” said Alan Cooke, president and CEO of Sebela Pharmaceuticals, in the company’s news release. How Can Suflave Boost Patient Compliance?

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FDA Approval Compliance Clinical Trials Clinical Trials 97

The Pharma Data

DECEMBER 17, 2020

The trial is a Phase 3 randomized, controlled, open-label, multicenter clinical trial, aiming to evaluate the efficacy and safety of ATG-010, bortezomib and dexamethasone (SVd) regimen against bortezomib and dexamethasone (Vd) regimen in Chinese adult patients with rrMM who have received one to three prior lines of therapy.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Drugs 52

The Pharma Data

JANUARY 31, 2021

Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immune responses in a phase 1 trial. Clover plans to initiate a global phase 2/3 trial in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021.

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Clinical Trials Clinical Trials Vaccination Vaccine 52

The Pharma Data

AUGUST 16, 2020

First and only FDA-approved subcutaneous treatment option for anti-aquaporin-4 antibody positive NMOSD that can be self-administered by a person with NMOSD or a caregiver every four weeks. First and only approved therapy for NMOSD designed to target and inhibit interleukin-6 receptor activity, using novel recycling antibody technology.

FDA Approval 52

FDA Approval Antibody Clinical Trials Clinical Trials 52

The Pharma Data

DECEMBER 16, 2020

The Company will initiate its trial during the first quarter of 2021 to investigate the efficacy of Berubicin in adults with GBM who have failed first-line therapy. “Since becoming a public company, our clear focus has been on advancing the clinical development of Berubicin.

FDA Approval 52

FDA Approval Drugs Clinical Trials Clinical Trials 52

Drug Discovery World

FEBRUARY 14, 2024

AlphaMedix is a targeted alpha therapy currently in Phase II clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles. In the Phase I study, treatment was well-tolerated, with a response rate of 62.5%

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In-Vivo FDA Approval Clinical Trials Clinical Trials 52

The Pharma Data

JANUARY 13, 2021

Fast Track designation facilitates the development and expedites the review of drugs that are intended to treat serious and life-threatening conditions and show the potential to fill an unmet medical need. Brilacidin, a versatile compound with broad therapeutic potential, is in a new chemical class called defensin-mimetics.

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Clinical Trials Clinical Trials Trials In-Vitro 52

The Pharma Data

DECEMBER 29, 2020

Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy. galactosidase-A product candidate under development for the treatment of Fabry disease. . CARMIEL, Israel and BOSTON , Dec. 30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc.

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Clinical Trials Clinical Trials Trials Protein 52

The Pharma Data

NOVEMBER 20, 2020

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies. Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that the U.S. PALO ALTO, Calif.,

FDA Approval 52

FDA Approval Genetics Clinical Trials Clinical Trials 52

Pharmaceutical Technology

MARCH 1, 2023

While the pandemic dramatically increased public awareness of mRNA therapeutics, lesser-known categories of RNA-based therapies have been established and approved for use by the FDA for almost a decade. RNA-based drug development warrants close monitoring as more RNA drug assets enter late-stage clinical trials.

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RNA Vaccination Vaccine Genetics 147

Pharmaceutical Technology

MARCH 6, 2023

Currently, most drugs being investigated for their healthy aging or anti-aging properties are being repurposed from readily accessible FDA-approved drugs. Barzilai hopes that the well-established profile of the drug may allow the FDA to accept the trial more easily and then other aging-related studies can follow.

Drugs 363

Drugs Regulation Clinical Trials Clinical Trials 363

The Pharma Data

NOVEMBER 24, 2020

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

FDA Approval 52

FDA Approval Production RNA Medicine 52

The Pharma Data

JANUARY 20, 2021

FDA Approves Verquvo (vericiguat) for Heart Failure with Reduced Ejection Fraction. mg, 5 mg, and 10 mg tablets is being jointly developed with Bayer AG. Armstrong, cardiologist and Distinguished University Professor of Medicine at the Canadian VIGOUR Centre, University of Alberta, and study chair of the VICTORIA trial.

FDA Approval 52

FDA Approval Production Trials Clinical Trials 52

pharmaphorum

SEPTEMBER 3, 2020

In a filing with the US financial regulator Compass laid out its plans to develop the drug known as COMP360, which is a crystalline formulation of psilocybin in phase 2b clinical development in patients who have not responded to standard therapy for depression.

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Drugs Life Science Clinical Development Regulation 109

XTalks

MAY 9, 2024

XTALKS CLINICAL EDGE: Issue 2 — Genmab’s Interview Xtalks Clinical Edge is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinical trials. Celebrating 25 years of innovation is a significant milestone.

Antibody 52

Antibody Clinical Trials Clinical Trials Medicine 52

pharmaphorum

MARCH 10, 2021

With so many novel drug candidates for Alzheimer’s disease failing in clinical development, researchers in the US have started using artificial intelligence (AI) to screen already-approved therapies for activity against the neurodegenerative disorder. .

Clinical Trials 84

Clinical Trials Clinical Trials Trials FDA Approval 84

Pharmaceutical Technology

FEBRUARY 24, 2023

Amivantamab is under clinical development by Johnson & Johnson and currently in Phase I for Colorectal Cancer. GlobalData’s report assesses how Amivantamab’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks.

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Recombinant DNA Technology Production Antibody FDA Approval 100

Drug Discovery World

AUGUST 10, 2023

Based on our vaping cessation trial, ORCA-V1, participants who were treated with cytisinicline were 2.6 JB: The successful results from our second, confirmatory Phase III trial set the stage for the future of Achieve. times more likely to quit vaping compared to those who received placebo. What comes next for Achieve?

Drugs 52

Drugs FDA Approval Clinical Trials Clinical Trials 52

pharmaphorum

SEPTEMBER 12, 2022

Reddy says one of LabCorp’s groundbreaking tests, OmniSeq INSIGHT, is an “all-in-one test that includes comprehensive genomics and immune profiling from a single tissue sample to identify all FDA-approved therapeutics, confirm immunotherapy eligibility for a patient, and identify appropriate nearby clinical trials.”.

Genome 101

Genome Genomics Clinical Trials Clinical Trials 101