Pharmaceutical Technology

MARCH 27, 2023

It is claimed to be the first and only therapy to receive approval in the US for the treatment of APDS, a rare and progressive primary immunodeficiency. After assessing the New Drug Application (NDA) under priority review, the FDA granted the approval based on the data obtained from a Phase II/III clinical trial.

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FDA Approval Clinical Trials Clinical Trials Medicine 130

AuroBlog - Aurous Healthcare Clinical Trials blog

NOVEMBER 12, 2023

The US Food and Drug Administration (FDA) on Wednesday announced it had approved Eli Lilly’s highly anticipated weight loss drug Zepbound, the latest entrant in a field of powerful – and lucrative – new obesity medicines.​

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FDA Approval Drugs Medicine Clinical Trials 192

Pharmaceutical Technology

SEPTEMBER 5, 2022

Earlier, the therapy had obtained FDA approval for usage in CF patients aged two years and above with two F508del mutation copies. The latest approval for use in children aged 12 to below 24 months is based on an open-label, multicentre Phase III clinical trial for 24 weeks.

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FDA Approval Genotype Clinical Trials Clinical Trials 130

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. AstraZeneca is also venturing into cell therapy and genetic medicine.

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Pharmaceutical Technology

JUNE 1, 2023

The US Food and Drug Administration (FDA) has granted a combination of AstraZeneca and MSD ’s Lynparza (olaparib), with standard therapies for treating BRCA-mutated (BRCAm) metastatic castration-resistant prostate cancer (mCRPC). Safety and tolerability were in line with that observed in prior trials and the known profiles of the medicines.

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FDA Approval Hormones Trials Clinical Trials 244

Pharmaceutical Technology

JUNE 21, 2023

With today’s FDA approval of Talzenna plus Xtandi, we are proud to be able to offer this potentially practice-changing treatment to patients and add to their options in managing this aggressive disease.” The regulatory approval was based on the findings obtained from the Phase III TALAPRO-2 trial.

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FDA Approval Gene DNA Medicine 130

Pharmaceutical Technology

JUNE 21, 2023

It can be used as a monotherapy or along with cholesterol-lowering medicines. It is claimed to be the first anti-inflammatory atheroprotective cardiovascular treatment to receive approval from the regulator.

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FDA Approval Cardiology Clinical Trials Clinical Trials 246

XTalks

NOVEMBER 30, 2023

Pfizer spinout SpringWorks Therapeutics’ Ogsiveo (nirogacestat) has received US Food and Drug Administration (FDA) approval for the treatment of desmoid tumors, an ultra-rare subtype of non-cancerous soft tissue sarcomas that can cause severe pain and disfigurement.

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FDA Approval Trials Clinical Trials Clinical Trials 101

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Safety and Efficacy of Filsuvez The FDA’s approval of Filsuvez is supported by compelling clinical evidence. Specifically, 41.3 percent observed in the control gel arm. percent of cases.

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FDA Approval Gene Therapy Gene Trials 111

JAMA Internal Medicine

FEBRUARY 12, 2023

This cross-sectional study determines the frequency of and rationale for US Food and Drug Administration (FDA) approval of drugs not meeting pivotal trial primary efficacy end points.

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FDA Approval Trials Drugs 76

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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FDA Approval Gene Therapy Genetic Disease Gene 130

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.

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FDA Approval Genetics Gene Therapy Gene 105

Pharmaceutical Technology

MAY 1, 2023

“This milestone is a testament to advancing our robust biopharmaceutical pipeline of innovative medicines that aim to support more people living with mental health disorders and neurological diseases in the coming years.” Uzedy reduced the risk of schizophrenia relapse by up to 80% compared to placebo in a Phase III clinical trial.

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FDA Approval Clinical Trials Clinical Trials Trials 189

XTalks

JANUARY 3, 2024

Wainua is the only FDA-approved drug for the treatment of ATTRv-PN that can be self-administered via an auto-injector. Approval of Wainua represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease,” said Michael J.

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FDA Approval Protein RNA Clinical Trials 105

XTalks

FEBRUARY 6, 2023

GlaxoSmithKline LLC (GSK) recently announced that the US Food and Drug Administration (FDA) approved Jesduvroq (daprodustat), a new once-a-day oral treatment for anemia due to chronic kidney disease (CKD). The approval is currently only for adult patients who have been undergoing dialysis for at least four months.

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FDA Approval Clinical Trials Clinical Trials Trials 97

Bio Pharma Dive

MARCH 30, 2021

The drugmaker's pill fell short on a critical safety measure in two large trials last fall. That result, and the surprisingly close FDA scrutiny of a rival medicine from FibroGen, makes the approval prospects of both treatments uncertain.

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Drug Discovery World

MARCH 26, 2024

“People who are immunocompromised continue to be disproportionally impacted by Covid-19 even after receiving multiple vaccine doses,” said Cameron Wolfe, Professor of Medicine, Transplant Infectious Disease at Duke University School of Medicine.

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FDA Approval Antibody Clinical Trials Clinical Trials 52

Pharmaceutical Technology

APRIL 12, 2023

The US Food and Drug Administration (FDA) has approved Takeda ’s supplemental Biologics Licence Application (sBLA) to expand the use of its HyQvia subcutaneous immune globulin (ScIG) for the treatment of primary immunodeficiency (PI) in children aged between two and 16 years. It was previously approved in the US to treat PI in adults.

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FDA Approval Clinical Trials Clinical Trials Containment 130

Drug Discovery World

JUNE 23, 2023

Phase III EMBARK trial Consistent with the accelerated approval pathway, the company has committed to the completion of a confirmatory trial. The approval of Elevidys is a watershed moment for the treatment of Duchenne,” said Doug Ingram, President and Chief Executive Officer of Sarepta. “If

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Gene Therapy FDA Approval Gene Protein 97

Drug Discovery World

MARCH 22, 2024

Michael Weber, Professor of Medicine, Division of Cardiovascular Medicine, State University of New York, and an investigator in the PRECISION study, commented: “Today, we are not able to reduce blood pressure below recommended levels in at least 10% of the hypertensive patients we treat.

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Drug Discovery World

JULY 25, 2023

The efficacy of quizartinib with chemotherapy was evaluated in the QuANTUM-First trial of 539 patients with newly diagnosed FLT3-ITD positive AML. FLT3-ITD status was determined prospectively with a clinical trial assay and verified retrospectively with the companion diagnostic LeukoStrat CDx FLT3 Mutation Assay.

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FDA Approval Trials Clinical Trials Clinical Trials 52

Drug Discovery World

AUGUST 21, 2023

The FDA approval is based on data from the Phase III MOVE trial, which demonstrated that palovarotene effectively reduced annualised heterotopic ossification (HO) volume compared with no treatment beyond standard of care (54% reduction with weighted linear mixed effect model). “As Sohonos is not for everyone.

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FDA Approval Medicine Drugs Regulation 52

XTalks

OCTOBER 4, 2022

XTALKS WEBINAR: Time for Change in NASH: How Non-Invasive Biomarkers are Driving a Paradigm Shift in Clinical Trials and Clinical Care. Outcomes of Clinical Trials of Terlipressin. The clinical trial results were published in the New England Journal of Medicine in March 2021. Possible Side Effects of Terlivaz.

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FDA Approval Clinical Trials Clinical Trials Trials 98

Pharmaceutical Technology

APRIL 17, 2023

The US Food and Drug Administration (FDA) has approved an update to the indications and usage section of Horizon Therapeutics ’ Tepezza (teprotumumab-trbw) label to specify its use to treat thyroid eye disease (TED) patients regardless of disease activity or duration. The FDA’s approval was granted in January 2020.

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FDA Approval Drugs Insulin Clinical Trials 130

Delveinsight

AUGUST 17, 2021

FDA’s Green Flag to Keytruda and Lenvima combination by Merck and Eisai for Advanced renal cell carcinoma (RCC). The FDA approved the combination of Keytruda and Lenvima produced by Merck and Eisai, as a first-line treatment of adult patients with advanced renal cell carcinoma (RCC). Jazz Pharmaceuticals announced the U.S.

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FDA Approval Medicine Vaccination Vaccine 98

Drug Discovery World

JUNE 6, 2023

The FDA’s decision is based on the data from the pivotal Phase III clinical trial RENOIR (RSV vaccine Efficacy study iNOlder adults Immunized against RSV disease). The post FDA approves first vaccine for respiratory syncytial virus appeared first on Drug Discovery World (DDW).

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FDA Approval Vaccination Vaccine Clinical Trials 52

Drug Discovery World

AUGUST 23, 2023

Pfizer’s Abrysvo (Respiratory Syncytial Virus Vaccine) was approved for use at 32-36 weeks gestational age of pregnancy to prevent lower respiratory tract disease (LRTD) and severe LRTD caused by RSV in infants from birth to six months of age. In infants and children, the risk of RSV-associated LRTD is highest during the first year of life.

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FDA Approval Vaccination Vaccine Clinical Trials 52

pharmaphorum

MAY 16, 2022

GLP-1 and GIP are hormones involved in blood sugar control and Mounjaro, a first-in-class medicine that activates both GLP-1 and GIP receptors, demonstrated improved blood sugar control. And, in trials comparing Mounjaro to other diabetes medications, individuals who received 15mg of Mounjaro had lowering of their HbA1c by 0.5%

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FDA Approval Insulin Drugs Hormones 108

pharmaphorum

JANUARY 12, 2023

Avillion – clinical co-development partner with AZ – conducted the global phase 3 trial of over 4,000 patients across four studies (including the MANDALA and DENALI trials). Additionally, under its 2018 agreement, Avillion had regulatory responsibility, including filing the New Drug Application (NDA) through to FDA approval.

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FDA Approval Allergies Trials Life Science 52

STAT News

SEPTEMBER 9, 2022

… Running clinical trials remotely or virtually can provide substantial financial savings for pharmaceutical companies, STAT writes, citing a new pilot study. million, on average, for Phase 2 clinical trials. million when these methods were used during both trial stages. Or simply plan the rest of your life. But be safe.

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Clinical Trials Clinical Trials FDA Approval Trials 98

Medical Xpress

NOVEMBER 22, 2022

An analysis of 144 pivotal trials for U.S. Food and Drug Administration (FDA)-approved medications in cancer, cardiovascular disease, and neurology has found that more than half of the studies recruit patients from low- and middle-income countries (LMIC). The analysis is published in Annals of Internal Medicine.

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Trials FDA Approval Medicine Drugs 96

STAT News

OCTOBER 1, 2022

Food and Drug Administration approved a new medicine for ALS from Amylyx Pharmaceuticals, providing a desperately needed new treatment option for a devastating disease , STAT writes. The But be safe. Enjoy, and see you soon. … The U.S.

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Medicine FDA Approval Drugs Clinical Trials 98

XTalks

NOVEMBER 10, 2023

Today, we are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.” It’s also been granted orphan designation by the European Medicines Agency (EMA) and Japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of TTP.

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FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

APRIL 11, 2024

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

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FDA Approval Manufacturing Trials Clinical Trials 52

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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FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

MAY 4, 2022

Bristol Myers Squibb (BMS) was granted US Food and Drug Administration (FDA) approval for its highly awaited cardiac drug Camzyos (mavacamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (HCM). The FDA approved 2.5 Live and On-Demand: Tuesday, May 24, 2022, at 1pm EDT (10am PDT).

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FDA Approval Drugs Clinical Trials Clinical Trials 98

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Gene Therapy FDA Approval Gene Gene Editing 52