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

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

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

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FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

MARCH 10, 2023

This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher. It is caused by a mutation in the FXN gene , which is responsible for the production of the protein frataxin. Skyclarys was approved based on the results of the MOXIe Part 2 clinical trial.

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FDA Approval Engineer Clinical Trials Clinical Trials 52

Drug Discovery World

JANUARY 23, 2023

“Put simply, radioresistant GBM cells prefer to stock up on fatty acids instead of utilising them as an energy source in order to reduce mitochondrial reactive oxygen species that may cause damage to their DNA, RNA, and proteins, and, in turn, cell death,” explains Prof Youn.

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pharmaphorum

MARCH 29, 2021

It is a CAR-T (chimeric antigen receptor T-cell) therapy, where a patient’s own T-cells are harvested, genetically modified and then reinjected to fight the cancer. However the payout worth up to $7 billion never happened after another CAR-T Breyanzi (lisocatbtagene maraleucel) was approved in February, missing a deadline set out in the CVR.

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FDA Approval Genetics Protein Antibody 62

XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. One strategy proposed the use of synthetic mRNA or modified mRNA analogs, which can enhance the stability and protein expression of mRNA.

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XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Gene Therapy Gene FDA Approval Dermatology 97

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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Trials Drugs FDA Approval Clinical Trials 98

The Pharma Data

NOVEMBER 20, 2020

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies. Food and Drug Administration (FDA) has approved Zokinvy (lonafarnib) for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies (PL). .

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FDA Approval Genetics Clinical Trials Clinical Trials 52

XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Regulatory Approvals for CTC-Based Assays in Oncology. The Utility of cfDNA for Cancer Management and Predicting Outcomes.

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Clinical Trials Clinical Trials Trials FDA Approval 98

The Pharma Data

MAY 31, 2022

More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. About Evrysdi® (risdiplam) Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.

FDA Approval 52

FDA Approval Protein In-Vitro Medicine 52

XTalks

MAY 1, 2023

Last week, the US Food and Drug Administration (FDA) granted accelerated approval to Biogen’s Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene. How Does Qalsody Work?

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FDA Approval Gene Genetics Protein 89

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

XTalks

FEBRUARY 27, 2023

Chiesi Global Rare Diseases recently announced in a press release that Lamzede (velmanase alfa-tycv) received US Food and Drug Administration (FDA) approval for the treatment of non-neurological manifestations of alpha-mannosidosis in both adult and pediatric patients.

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FDA Approval Life Science Containment Genetics 52

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. IGF-1 is an acceptable surrogate endpoint in trials of growth hormone receptor agonists like Somavert, and is preferred for pediatric patient populations.

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Trials Gene Hormones Clinical Trials 98

pharmaphorum

SEPTEMBER 16, 2022

A mere six months ago Verily launched the study with Sosei Heptares – a global leader in GPCR structure-based drug design – with an aim to “prioritise protein targets for therapeutic targeting in immune-mediated disease”. It also bodes well for improving clinical trial success rates. What, then, is the solution? AI relies on data.

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Development Research Drugs Clinical Trials 125

Drug Discovery World

OCTOBER 18, 2023

We’re beginning to see their potential come to fruition, with the FDA having approved three treatments as of January 2023 — for retinal dystrophy 1 spinal muscular atrophy, 2 and haemophilia B.

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Gene Therapy Gene Clinical Trials Clinical Trials 64

XTalks

JUNE 15, 2022

The Novavax COVID-19 vaccine was recommended for emergency use in adults by the World Health Organization (WHO) in December 2021 , and is approved in over 40 countries. The Novavax candidate NVX-CoV2373 is a protein-based vaccine developed using recombinant nanoparticle technology. Clinical Trial Results.

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Vaccination Vaccine Immune Response Protein 59

XTalks

JUNE 8, 2021

In addition, recent data from a Phase I trial has shown ‘remarkable’ long-term results of the gene therapy in children with the disease. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy?

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Gene Therapy Gene Protein Trials 110

XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Early clinical trials have shown promising results and this therapy could become a valuable addition towards CRC treatment in the future. Yang Liu and CFO Abid Ansari – Xtalks Life Science Podcast Ep.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

XTalks

AUGUST 17, 2020

As DMD is caused by a lack of functional dystrophin protein, which supports and strengthens muscle fibers, therapies designed to treat this rare genetic disorder have focused on exon skipping to avoid the area of the dystrophin gene that is mutated and produce a nearly full-length dystrophin protein.

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Drugs Clinical Trials Clinical Trials Gene 105

XTalks

AUGUST 6, 2020

The US Food and Drug Administration (FDA) has approved the use of an oral cannabidiol (CBD) solution called Epidiolex for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged one year and older. Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

Drug Discovery World

JUNE 1, 2023

A Nature publication confirmed that there are currently 12 FDA-approved ADCs on the market, and nine of these secured FDA approval in the past six years 2. DDW’s Megan Thomas explores three trends in the market.

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Antibody Marketing Drugs Licensing 98

Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP).

FDA Approval 52

FDA Approval Gene Clinical Trials Clinical Trials 52

Drug Discovery World

MAY 24, 2023

G protein-coupled receptors (GPCRs) are a large family of proteins that regulate many processes in the body and are the target of one third of clinically used drugs. Several FDA-approved drugs that target the NK1 receptor are used to prevent nausea and vomiting associated with chemotherapy or surgery.

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Research Drugs Protein FDA Approval 52

XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. The approval came after a couple of setbacks, including a rejection in 2020 and a delay earlier this year. SVB has forecasted around $2.2

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Gene Therapy Gene FDA Approval Antibody 98

The Pharma Data

NOVEMBER 30, 2020

CoV2373 is a stable, prefusion protein antigen derived from the genetic sequence of the SARS-CoV-2 coronavirus spike (S) protein and adjuvanted with Novavax’ proprietary Matrix?M. pivotal Phase 3 trial update. South Africa Phase 2b trial update. Mexico pivotal Phase 3 trial update. United Kingdom (U.K.)

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Clinical Development Vaccination Vaccine Development 52

XTalks

MARCH 6, 2024

This includes discussions on cutting-edge technologies and methodologies in brain research, such as neuroimaging, genetic engineering and artificial intelligence. Lilly is currently conducting a Phase III trial evaluating its beta-amyloid targeting monoclonal antibody, donanemab.

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FDA Approval In-Vitro Research Marketing 69

XTalks

DECEMBER 4, 2023

The FDA said it is investigating the identified risk of developing the T-cell malignancies as secondary cancers with serious outcomes, including hospitalization and death, and also looking at the need for regulatory action. However, the FDA’s investigation has now revealed actual reports of T-cell malignancies linked to the therapies.

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Gene Therapy Gene Clinical Trials Clinical Trials 69

The Pharma Data

JUNE 7, 2022

The new findings from the Phase 3 clinical trials (ADvocate 1 and 2) showed eight out of ten patients who achieved clinical response (EASI-75*) with lebrikizumab monotherapy at 16 weeks maintained skin clearance at one year of treatment with the once every two weeks or four weeks regimen. Source link: [link].

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Trials Clinical Trials Clinical Trials Medicine 40

XTalks

DECEMBER 12, 2023

The world of neuroscience trials is rapidly evolving, presenting new challenges and opportunities for researchers and clinicians. The worldwide market for neurology clinical trials stood at a valuation of $5.24 There is currently a heightened emphasis on imaging and liquid biomarkers in neuroscience clinical trials.

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Trials Clinical Trials Clinical Trials Genetics 111

The Pharma Data

SEPTEMBER 28, 2021

Also, the info from SRP-9001 have helped to optimise the planning of the upcoming phase III clinical trial trial for DMD,” said Levi Garraway, M.D., quite 4,000 patients are treated with Evrysdi in clinical trials, compassionate use, and real-world settings. Roche’s Chief medic and Head of worldwide development.

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Gene Therapy Protein Gene Clinical Trials 52

The Pharma Data

NOVEMBER 9, 2020

Food and Drug Administration (FDA) has granted Fast Track Designation for NVX-CoV2373, the Company’s COVID-19 vaccine candidate. Currently in late-phase clinical development, NVXCoV2373 is a stable, prefusion protein made using Novavax’ nanoparticle technology and includes its proprietary MatrixM adjuvant. and globally.”.

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Vaccination Vaccine Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 5, 2024

Revlimid (Lenalidomide) Revlimid 2022 sales: $9.978 billion Company/Developer: Bristol Myers Squibb Date of first FDA approval: December 27, 2005 Indications Revlimid is FDA-approved for: Myelodysplastic disease (MDS), multiple myeloma, mantle cell lymphoma (MCL), follicular lymphoma and marginal zone lymphoma.

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FDA Approval Drugs Sales Development 101