Pharmaceutical Technology

APRIL 27, 2023

Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) to expand the use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to treat cystic fibrosis (CF) in children aged between two and five years with certain mutations.

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XTalks

JANUARY 6, 2025

The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.

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Pharmaceutical Technology

MAY 11, 2023

Otsuka Pharmaceutical and H Lundbeck have received approval for the supplemental new drug application (sNDA) from the US Food and Drug Administration for Rexulti (brexpiprazole) to treat agitation associated with dementia due to Alzheimer’s disease. The regulator accepted and gr anted priority review for the sNDA in January 2023.

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Pharmaceutical Technology

SEPTEMBER 5, 2022

The treatment is indicated for CF patients who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Earlier, the therapy had obtained FDA approval for usage in CF patients aged two years and above with two F508del mutation copies.

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Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

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Pharmaceutical Technology

MAY 22, 2023

Epkinly has been approved as the first and only T-cell-engaging bispecific antibody to treat adults with DLBCL not otherwise specified (NOS), including DLBCL arising from indolent lymphoma and high-grade B–cell lymphoma (HGBL), following two or more lines of systemic therapies.

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FDA Law Blog

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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XTalks

JANUARY 21, 2025

TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). The tricuspid valve, one of the heart’s four valves, regulates blood flow from the right atrium to the right ventricle, preventing backflow between these chambers.

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pharmaphorum

JANUARY 26, 2022

Immunocore has secured a piece of biotech industry history, becoming the first company to get an FDA approval for a cancer therapeutic based on T cell receptor (TCR) technology. The post Immunocore claims first-ever FDA approval for TCR cancer therapy appeared first on.

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Pharmaceutical Technology

MAY 22, 2023

It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious genetic disease affecting the skin and mucosal tissues. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

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pharmaphorum

AUGUST 4, 2021

Bristol-Myers Squibb’s HDAC inhibitor Istodax has been on the US market for a decade as a treatment for peripheral T-cell lymphoma (PTCL), but will now be withdrawn from sale after a failed phase 3 trial. The post Failed trial nixes another FDA approval, this time for BMS’ Istodax appeared first on.

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XTalks

DECEMBER 12, 2023

After receiving US Food and Drug Administration (FDA) approval for Fabhalta (iptacopan) last week for the treatment of the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH), Novartis presented trial data yesterday showing the drug’s promise in another indication.

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XTalks

NOVEMBER 27, 2024

Syndax Pharmaceuticals’ Revuforj (revumenib) has won US Food and Drug Administration (FDA) approval for the treatment of relapsed or refractory (R/R) acute leukemia in patients aged one year and older who have a lysine methyltransferase 2A ( KMT2A, formerly MLL ) gene translocation. times its initial investment.

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Pharmaceutical Technology

MAY 10, 2023

The US Food and Drug Administration (FDA) has granted approval for AstraZeneca ’s Farxiga (dapagliflozin) to reduce the risks of cardiovascular (CV) death, hospitalisation for heart failure (hHF) and urgent heart failure (HF) emergency care visits in HF adult patients.

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pharmaphorum

SEPTEMBER 16, 2021

Takeda has had a run of bad luck with its Wave1 pipeline of new drug candidates of late, but can now celebrate a vestry after getting FDA approval for first-in-class lung cancer therapy Exkivity. The post Takeda gets a win for its Wave1 pipeline, as Exkivity nabs FDA approval appeared first on.

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pharmaphorum

AUGUST 16, 2021

Pfizer and BioNTech have formally asked for FDA approval of a third dose of their COVID-19 vaccine BNT162b2 in people aged over 16, as the US prepares to get its booster campaign underway. The post Pfizer, BioNTech file for FDA approval of COVID booster shot appeared first on.

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XTalks

APRIL 3, 2024

Related: Novartis’ Fabhalta Gets FDA Approval for Rare Complement Blood Disorder The FDA’s green light for Voydeya comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator in the world to back the therapy.

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XTalks

DECEMBER 9, 2024

Despite its potential to slow cognitive decline, the delays highlight the challenges of timely drug approval for life-threatening conditions. The draft builds on prior FDA policies but introduces new provisions from the Consolidated Appropriations Act, 2023.

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XTalks

DECEMBER 18, 2024

Currently, the standard treatment for CAH involves glucocorticoids, which replace cortisol and help regulate hormone levels. In the first trial, 122 adults received Crenessity twice daily and 60 received placebo twice daily for 24 weeks. Crenessity marks the third approved drug for Neurocrine.

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XTalks

SEPTEMBER 12, 2022

Sanofi picked up its first Xenpozyme approval for the treatment of Niemann-Pick from Japanese regulators in March followed by an approval from European officials in May. The FDA approval was based on data from the randomized, double-blind, placebo-controlled ASCEND study involving 31 adult patients.

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BioPharma Reporter

MAY 5, 2022

Oramed Pharmaceuticals announced this week that it has enrolled 100% of the patients in the worldâs first Phase 3 study of oral insulin under FDA approved protocols.

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XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. The study is one of the largest DMD Phase III trials to date. million last year, of which $131.3

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XTalks

JUNE 2, 2021

The sotorasib approval is therefore a major breakthrough in the world of cancer and targeted therapies. The oral treatment was approved for adults with locally advanced or metastatic NSCLC, as determined by an FDA-approved test, who have received at least one prior systemic therapy.

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BioSpace

DECEMBER 14, 2023

Despite the lack of a randomized clinical trial to support eflornithine’s efficacy, the regulator approved US WorldMeds’ oral maintenance treatment for high-risk neuroblastoma in adults and children.

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XTalks

JANUARY 26, 2023

The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinical trials. Brukinsa is now approved to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

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Pharmaceutical Technology

JANUARY 8, 2023

There are at least six different biologics being studied in Phase III trials in COPD clinical programmes around the world. Results from a Phase III trial with Sanofi and Regeneron Pharmaceuticals’ IL-4/IL-13 monoclonal antibody (mAb) Dupixent (dupilumab) are expected by mid-2023, followed by others. Biologics: From asthma to COPD?

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pharmaphorum

JULY 8, 2022

The COVID-19 pandemic has catalysed significant changes in the way pharma develops drugs, particularly in the clinical trial space. Hybrid or decentralised clinical trials (DCTs) have gained traction as technology, infrastructure and knowledge have evolved to support their use. New sensor technology revolutionising data collection.

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XTalks

DECEMBER 28, 2023

BillionToOne, a company specializing in molecular diagnostics, has partnered with Janssen Research & Development, LLC, a Johnson & Johnson division, to conduct the AZALEA Phase III clinical trial globally. The trial is set to occur across the US, the European Union (EU) and selected global locations.

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XTalks

JANUARY 28, 2022

Kimmtrak has also become the first bispecific T cell engager to be FDA-approved for the treatment of a solid tumor. Immunocore received Breakthrough Device Designation for Kimmtrak from the FDA in February 2021 followed by acceptance of its Biologics License Application (BLA) several months later by both US and European regulators.

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XTalks

OCTOBER 6, 2020

The US Food and Drug Administration (FDA) has given approval to Janssen Pharmaceutical for Simponi Aria (golimumab) in the treatment of patients two years of age and older with active polyarticular juvenile idiopathic arthritis (pJIA). FDA approval for the treatment was based on results from the Phase III GO-VIVA clinical trial.

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ACRP blog

SEPTEMBER 4, 2024

Clinical trials provide the scientific foundation for justifying the safety and efficacy of drugs, biologics, and devices—but are laborious, expensive, and risky. Only 10% of drugs entering clinical trials receive U.S. clinical trial workforce reported burnout since the 2020 COVID-19 pandemic.{4}

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XTalks

APRIL 29, 2022

Eli Lilly announced that its obesity drug tirzepatide has scored favorably in a late-stage clinical trial, with results showing that people who took the drug lost an average of 50 pounds, or 21 percent, of their body weight compared to placebo. percent and 22.5 percent, respectively, compared to placebo.

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Pharmaceutical Technology

AUGUST 16, 2022

According to the deal, Horizon will provide the funding to develop the therapy until the conclusion of its two Phase II clinical trials. Under the agreement, Horizon will hold an option for the acquisition of the ADX-914 programme, which is exercisable through a period after concluding the Phase II trials.

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XTalks

JANUARY 3, 2025

For the first time in the US, adults living with familial chylomicronemia syndrome (FCS) have an FDA-approved treatment option. Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism. In clinical trials, it showed up to an 86 percent reduction in triglyceride levels.

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XTalks

SEPTEMBER 17, 2021

The ALS Association is pushing for rapid FDA approval of Amylyx Pharmaceuticals Inc.’s After asking for a placebo-controlled Phase III study for the drug in April that upset the ALS Association, the FDA dialed back on the request this week, agreeing to accept only Phase II trial data for evaluation of Amylyx’s drug application.

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pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

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BioSpace

SEPTEMBER 18, 2023

The latest data show Izervay is safe and effective in a two-year trial. Just six weeks after securing FDA approval, Astellas plans to submit it to the regulator to expand its label for a longer treatment timeframe.

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