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.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

MAY 26, 2022

The US FDA approved Dupixent (dupilumab) developed by Regeneron as the first treatment for eosinophilic esophagitis, a chronic immune disorder that damages the esophagus. These proteins cause the type 2 inflammation characteristic of EoE and related allergic diseases like asthma and atopic dermatitis (both treated with Dupixent).

FDA Approval 105

FDA Approval Antibody Clinical Trials Clinical Trials 105

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).

FDA Approval 98

FDA Approval Drugs Clinical Trials Clinical Trials 98

pharmaphorum

NOVEMBER 16, 2022

RxDx Assay – Roche’s immunohistochemistry (IHC) companion diagnostic for identifying ovarian patients eligible for ELAHERE – has been contemporaneously approved by the FDA. also known as Folate receptor 1 protein) and who had been treated with one to three prior systemic treatment regimens. Additionally, VENTANA FOLR1 (FOLR1-2.1)

FDA Approval 67

FDA Approval Protein Trials Packaging 67

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.

FDA Approval 97

FDA Approval Clinical Trials Clinical Trials Trials 97

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).

FDA Approval 97

FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

MARCH 10, 2023

This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher. In the EU, Skyclarys has been granted the Orphan Drug designation and is under review by the European Medicines Agency (EMA). Skyclarys was approved based on the results of the MOXIe Part 2 clinical trial.

FDA Approval 52

FDA Approval Engineer Clinical Trials Clinical Trials 52

XTalks

JUNE 8, 2022

These autoantibodies include rheumatoid factor and antibodies against post-translational modified proteins such as carbamylation (anti-CarP antibodies) and citrullination (anti-citrullinated protein antibodies [ACPA]). This marks a new indication for Riabni, the fifth FDA-approved biosimilar by the biopharma Amgen.

FDA Approval 96

FDA Approval Antibody Clinical Trials Clinical Trials 96

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.

FDA Approval 52

FDA Approval Manufacturing Trials Clinical Trials 52

Roots Analysis

JANUARY 19, 2024

Introduction In a strategic move to accelerate the development and launch of genetic medicines, BridgeBio Pharma has successfully secured up to USD 1.25 In a deal worth USD 500 million in cash, BridgeBio Pharma has committed to providing the investors with a 5% royalty on worldwide net sales of its drug, acoramidis, pending FDA approval.

Genetics 40

Genetics Medicine Development Sales 40

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.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 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.

FDA Approval 57

FDA Approval Drugs Gene Therapy Protein 57

XTalks

DECEMBER 29, 2023

. “The FDA’s Priority Review designation for this application underscores the urgency to provide new treatment options for patients with advanced SCLC who have progressed following treatment with platinum-based chemotherapy,” said David M. months (the median duration of response was not evaluable).

Clinical Trials 119

Clinical Trials Clinical Trials FDA Approval Trials 119

Pharmaceutical Technology

NOVEMBER 13, 2022

The US Food and Drug Administration (FDA) has granted approval for AstraZeneca ’s Imfinzi (durvalumab) plus Imjudo (tremelimumab) and platinum-based chemotherapy to treat Stage IV (metastatic) non-small cell lung cancer (NSCLC) in adults.

Protein 130

Protein FDA Approval Antibody Trials 130

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.

FDA Approval 98

FDA Approval Development Licensing Licensing 98

Drug Discovery World

JANUARY 17, 2024

Brett Swansiger, Chief Commercial Officer at ANGLE discusses the importance of liquid biopsies in precision medicine trials for cancer. Furthermore, oncology clinical trials are frequently challenged by low enrolment rates, failure to achieve primary endpoints, study design complexity and limited funding 5.

Medicine 59

Medicine DNA Genome Genomics 59

XTalks

AUGUST 10, 2022

HER2-low is characterized by the presence of some HER2 proteins on the cell surface, but not enough to be classified as HER2-positive. In the US, it is estimated that 287,850 new cases of female breast cancer will be diagnosed in 2022, according to data from the National Cancer Institute (NCI) outlined by the FDA. Enhertu Trial Data.

FDA Approval 98

FDA Approval HR Hormones Antibody 98

Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Initial approval in 1994: FDA, U.S.

Gene 162

Gene Clinical Trials Clinical Trials Gene Therapy 162

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.

Trials 111

Trials Clinical Trials Clinical Trials Genetics 111

Pharmaceutical Technology

DECEMBER 23, 2022

This approval is backed by findings from the multicentre, double-blinded, placebo-controlled, international Phase II/III CAPELLA clinical trial of lenacapavir plus an optimised background regimen. Subcutaneous lenacapavir’s antiviral activity administered every six months was analysed in the trial.

Drugs 147

Drugs FDA Approval Clinical Trials Clinical Trials 147

XTalks

JUNE 5, 2023

Abrysvo is an unadjuvanted vaccine and is composed of two preF proteins selected to optimize protection against RSV A and B strains. “A In February 2023, Pfizer revealed that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for Abrysvo. percent and severe RSV-associated LRTD by 94.1

Vaccination 98

Vaccination Vaccine FDA Approval Protein 98

XTalks

DECEMBER 5, 2023

As its active ingredient, repotrectinib inhibits the proto-oncogene tyrosine-protein kinase ROS1 and the tropomyosin receptor tyrosine kinases (TRKs) TRKA, TRKB and TRKC. Today’s approval brings a new treatment option for the ROS1 -positive patient community, which gives us hope for more time with loved ones.” percent, 79.2

Protein 115

Protein FDA Approval Medicine Trials 115

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.

Gene Therapy 80

Gene Therapy Gene Trials Protein 80

XTalks

JULY 10, 2023

Euronext Brussels (UCB), a biopharma company headquartered in Brussels, Belgium, recently announced that the US Food and Drug Administration (FDA) has granted approval for its drug, Rystiggo (rozanolixizumab), for the treatment of generalized myasthenia gravis (gMG). What Is Generalized Myasthenia Gravis (gMG)?

FDA Approval 98

FDA Approval Antibody Protein Engineer 98

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.

FDA Approval 101

FDA Approval Drugs Sales Development 101

Bioengineer

JULY 20, 2021

FDA approval based on positive results of international, multi-center ProDERM study. The prospective, double-blind, placebo-controlled Phase III clinical trial enrolled 95 patients at 36 sites globally, including 17 sites in the U.S., Patients were allowed to switch treatment if they deteriorated during the trial.

FDA Approval 40

FDA Approval Clinical Trials Clinical Trials Trials 40

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.

Vaccination 52

Vaccination Vaccine Protein Antibody 52

Pharmaceutical Technology

AUGUST 23, 2022

It comprises messenger ribonucleic acid (mRNA) that encodes for the initial spike protein of SARS-CoV-2 along with mRNA that encodes for Omicron BA.4/BA.5 5 variant spike protein. The companies have also commenced rolling submission for conditional marketing authorization from the European Medicines Agency (EMA) for this Omicron BA.4/BA.5-adapted

Vaccination 130

Vaccination Vaccine Gene Therapy Protein 130

The Pharma Data

NOVEMBER 12, 2021

Treatment is First FDA-Approved Option Patients Can Take Regardless of Previous Therapies. Food and Drug Administration approved Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red blood cells. The FDA, an agency within theU.S.

FDA Approval 40

FDA Approval Gene Production Cosmetics 40

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.

Trials 98

Trials Drugs FDA Approval Clinical Trials 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 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). Krystal expects Vyjuvek to be available by the third quarter of this year.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

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.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

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

Pfizer

JULY 26, 2022

Pfizer and BioNTech Advance COVID-19 Vaccine Strategy With Study Start of Next-Generation Vaccine Candidate Based on Enhanced Spike Protein Design. Pfizer and BioNTech Advance COVID-19 Vaccine Strategy With Study Start of Next-Generation Vaccine Candidate Based on Enhanced Spike Protein Design. deliesschef. Tue, 07/26/2022 - 17:41.

Vaccination 111

Vaccination Vaccine Protein Immune Response 111

The Pharma Data

DECEMBER 17, 2020

Lupus causes the production of proteins called autoantibodies in the immune system, which attack tissues and organs, including the kidneys. There will be a brand-new treatment for actinic keratosis on the market next year, with Tuesday’s FDA approval of Athenix’s Klisyri® (tirbanibulin). Athenex, Inc.

FDA Approval 52

FDA Approval Genome Genomics Production 52

The Pharma Data

NOVEMBER 20, 2020

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies. The increase in survival observed with Zokinvy was derived from two open-label clinical trials (N=62) conducted at Boston Children’s Hospital. PALO ALTO, Calif., ABOUT ZOKINVY (LONAFARNIB).

FDA Approval 52

FDA Approval Genetics Clinical Trials Clinical Trials 52