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.

FDA Approval 99

FDA Approval Drugs Gene Trials 99

XTalks

APRIL 3, 2024

The RBCs in circulation are marked by proteins in the complement system for removal by the spleen and liver. Voydeya’s FDA approval was based on positive results from the pivotal ALPHA Phase III trial. AstraZeneca is also venturing into cell therapy and genetic medicine.

FDA Approval 102

FDA Approval Sales Trials Development 102

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. Duvyzat received Priority Review, Orphan Drug and Rare Pediatric Disease designations from the FDA.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

Scienmag

MARCH 26, 2021

Discovery opens door to testing more effective drugs for treating COVID-19 patients University of Maryland School of Medicine (UMSOM) researchers have identified the most toxic proteins made by SARS-COV-2–the virus that causes COVID-19 – and then used an FDA-approved cancer drug to blunt the viral protein’s detrimental effects.

Protein 71

Protein FDA Approval Drugs Medicine 71

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

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

FDA Approval 98

FDA Approval Drugs Clinical Trials Clinical Trials 98

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

FEBRUARY 26, 2024

After a 16- to 20-week treatment period, among those who received Xolair, 68 percent were able to eat at least a single 600-mg dose of peanut protein without moderate to severe allergic symptoms (such as whole-body hives, persistent coughing or vomiting) compared to six percent who received placebo.

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Allergies FDA Approval Drugs Protein 100

XTalks

JANUARY 26, 2023

This is the fourth US FDA approval that BeiGene has received for Brukinsa since 2019. Tyrosine Kinase Inhibitors for Cancer Treatment Tyrosine kinases, or TKs, are a group of signaling molecules that affect multiple downstream proteins. Results from the ALPINE study have been published in the New England Journal of Medicine.

FDA Approval 97

FDA Approval Life Science Clinical Trials Clinical Trials 97

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 Containment Licensing 98

Pharmaceutical Technology

NOVEMBER 1, 2022

The European Commission (EC) has granted orphan medicinal product designation for Karyopharm Therapeutics and the Menarini Group’s Nexpovio (selinexor) to treat myelofibrosis (MF). An oral inhibitor of exportin 1 (XPO1), Nexpovio works by selectively attaching to hindering the nuclear export protein XPO1.

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Protein FDA Approval Production Licensing 130

Pharmaceutical Technology

FEBRUARY 10, 2023

On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5 Access to oncology medicines varies across countries, and their cost against safety/efficacy is generally perceived as being low, such as in the US.

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Gene Therapy Marketing Gene Drugs 263

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 59

FDA Approval Drugs Gene Therapy Protein 59

Pharmaceutical Technology

NOVEMBER 13, 2022

A human monoclonal antibody (mAb), Imfinzi attaches to the PD-L1 protein and hinders its interaction with the PD-1 and CD80 proteins, preventing the immune-evading strategy of the tumour. Imjudo (tremelimumab) is also a mAb that acts on the cytotoxic T-lymphocyte-associated protein 4’s (CTLA-4) activity.

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Protein FDA Approval Antibody Trials 130

XTalks

MARCH 28, 2024

The US Food and Drug Administration (FDA) has given the nod to Winrevair (sotatercept-csrk injection, 45mg, 60mg) for the treatment of adults with pulmonary arterial hypertension (PAH) to increase exercise capacity, improve WHO (World Health Organization) functional class (FC) and reduce the risk of clinical worsening events.

FDA Approval 81

FDA Approval Clinical Trials Clinical Trials Trials 81

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 69

FDA Approval Protein Trials Packaging 69

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. FCS also severely impacts quality of life, causing chronic fatigue and recurrent stomach pain.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

Pharmaceutical Technology

JUNE 28, 2022

The latest research is part of the consortium of a new antiviral drug development centre named Center for Antiviral Medicines and Pandemic Preparedness (CAMPP), to be led by Scripps Research. The antiviral will be a low-dose drug which can be taken in the at-home setting. Meanwhile, the NIH also granted a $2.7m

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Development Bioinformatics Drugs Allergies 147

Pharmaceutical Technology

DECEMBER 23, 2022

It acts by hindering the protein shell (capsid) of the HIV-1 virus to hamper the viral lifecycle’s steps. Sunlenca is also said to be the first-ever capsid inhibitor-based HIV therapy to receive such approval. These subjects also had a mean rise in CD4 count of 82 cells/µL.

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

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Vaccination Vaccine FDA Approval Protein 98

Pharmaceutical Technology

AUGUST 23, 2022

On obtaining the FDA EUA, the Omicron BA.4/BA.5-adapted 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. 5-adapted bivalent vaccine will be made readily available for shipping.

Vaccination 130

Vaccination Vaccine Gene Therapy Protein 130

Pharmaceutical Technology

NOVEMBER 9, 2022

Haemophilia A is caused by insufficient levels of the clotting protein factor VIII, while patients with haemophilia B lack adequate factor IX levels. After its approval, bluebird had set Zynteglo’s price at roughly EUR1.6 Since its FDA approval in August 2022, the beta-thalassemia therapy was launched in the US with a $2.8

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Gene Therapy Gene FDA Approval Medicine 278

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

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. Analysts at William Blair estimate that Augtyro’s sales could climb to $258 million by 2027, contingent on its anticipated approval in Europe next year.

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Protein FDA Approval Medicine Trials 115

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.

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Vaccination Vaccine Protein Immune Response 111

The Pharma Data

FEBRUARY 25, 2022

Several FDA-approved drugs – including for type 2 diabetes, hepatitis C and HIV – significantly reduce the ability of the Delta variant of SARS-CoV-2 to replicate in human cells, according to new research led by scientists at Penn State. Pfizer’s COVID-19 therapy Paxlovid, for example, targets Mpro.

FDA Approval 52

FDA Approval Drugs Protein In-Vitro 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.

FDA Approval 59

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. In the EU, Skyclarys has been granted the Orphan Drug designation and is under review by the European Medicines Agency (EMA). What is Friedreich’s Ataxia?

FDA Approval 52

FDA Approval Engineer Clinical Trials Clinical Trials 52

pharmaphorum

JANUARY 11, 2023

Anima uses its mRNA Lightning platform to discover small molecules and determine their mechanisms of action for diseases previously deemed undruggable, with scientists searching for compounds that target proteins with roles in regulating mRNA, so as to affect disease states through post-transcriptional regulation (i.e.

Protein 105

Protein Regulation FDA Approval Licensing 105

XTalks

DECEMBER 29, 2023

It targets delta-like ligand 3 (DLL3), a protein usually found inside normal cells but abnormally present on the surface of SCLC cells. Clinical Evidence of Tarlatamab The FDA’s decision to grant tarlatamab priority review stems from the Phase II DeLLphi-301 clinical trial results, published in The New England Journal of Medicine.

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Clinical Trials Clinical Trials FDA Approval Trials 119

pharmaphorum

MAY 13, 2021

There are already several drugs on the market to treat Duchenne, notably from Sarepta Therapeutics which has three FDA-approved drugs that use “exon skipping” technology. These allow the body to produce a correct version of the misfolded dystrophin protein that is the root cause of DMD.

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Gene Therapy Gene Trials Protein 102

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

The Pharma Data

NOVEMBER 29, 2021

“The FDA’s approval of Cytalux can help enhance the ability of surgeons to identify deadly ovarian tumors that may otherwise go undetected,” said Alex Gorovets, M.D., deputy director of the Office of Specialty Medicine in the FDA’s Center for Drug Evaluation and Research. “By The FDA, an agency within the U.S.

FDA Approval 52

FDA Approval Drugs Protein Cosmetics 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.

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Gene Therapy Gene FDA Approval Dermatology 97

Pharmaceutical Technology

FEBRUARY 1, 2023

RSV researchers at major pharmaceutical companies are currently working to develop new RSV drugs to beat future waves of RSV infection and gain the first RSV vaccine FDA approval. According to the Centers for Disease Control and Prevention (CDC) , 45.8 This is a significant increase compared to the 2021–20 season when the rate was 28.3

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Vaccination Vaccine Antibody Development 162

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