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.

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Protein FDA Approval Drugs Medicine 71

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).

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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

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FDA Approval Drugs Clinical Trials Clinical Trials 98

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

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

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.

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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

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.

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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.

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Drugs FDA Approval Clinical Trials Clinical Trials 147

Pharmaceutical Technology

JANUARY 22, 2023

In 2022, the FDA approved only 37 new medicines, an underwhelming number compared to 98 in 2018. However, while only around 34% of the approvals in 2018 were for orphan drugs, 54% new approvals in 2022 were for drugs to treat rare diseases. point reduction in the placebo arm.

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Drugs Gene Hormones Clinical Trials 317

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.

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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)?

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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

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

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.

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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

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

XTalks

NOVEMBER 1, 2024

Why it sold so well: Botox is FDA-approved for the treatment of chronic migraines and that is currently the only chronic condition for which it has received approval. Related: Top 30 Drugs to Watch in 2024: Insights from 2023 Sales Data 1. billion in the US and $4.1 billion worldwide last year.

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FDA Approval Drugs Botox Sales 85

The Pharma Data

MAY 31, 2022

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. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Food and Drug Administration in 2017.

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FDA Approval Protein In-Vitro Medicine 52

The Pharma Data

JANUARY 6, 2021

(“CytoDel” or “the Company”), a privately-held corporation, today announces the publication of preclinical data on the Company’s lead product, Cyto-111, in the peer-reviewed journal, Science Translational Medicine. Cyto-111 was conceived, expressed and purified in the laboratory of Konstantin Ichtchenko, Ph.D., Südhof, M.D.,

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Medicine Antibody Protein In-Vitro 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.

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Genetics Medicine Development Protein 40

pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. Translarna has had European approval for usage since 2014 and has also been available under terms similar to the MAA to DMD patients in Scotland since 2021.

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Gene Therapy Gene FDA Approval DNA 98

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.

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FDA Approval Protein Containment Genetic Disease 64

The Pharma Data

JUNE 7, 2022

New Indication for Amgen’s Fifth FDA-approved Biosimilar. Now Approved to Treat All Available Rituxan ® Indications. Overall, 311 patients were randomized and treated with RIABNI, rituximab RP approved in the EU (rituximab-EU) or rituximab RP approved in the US (rituximab-US).

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FDA Approval Antibody Medicine Manufacturing 52

pharmaphorum

MAY 21, 2021

US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. The treatment restore the activity of a protein that breaks down the toxic metabolites.

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Gene Therapy Gene FDA Approval Clinical Trials 96

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. The disorder primarily affects males.

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Gene Therapy Gene FDA Approval Antibody 98

XTalks

MAY 2, 2023

In December 2021, AstraZeneca and Amgen’s Tezspire (tezepelumab) was approved by the FDA for patients 12 years of age and older with severe asthma that is not controlled by their current asthma medicine. Regeneron and Sanofi’s Dupixent (dupilumab) received FDA approval in 2018 for the treatment of moderate-to-severe asthma.

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Protein FDA Approval Clinical Trials Clinical Trials 109

XTalks

JUNE 21, 2021

A study published in The New England Journal of Medicine shows that early and sustained peanut introduction reduced the risk of peanut allergies by 80 percent. They were randomly assigned to consume peanut protein in children as young as four months of age until five years of age.

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Allergies Production FDA Approval Protein 98

XTalks

FEBRUARY 24, 2023

The US Food and Drug Administration (FDA) granted accelerated approval to Genentech’s Lunsumio (mosunetuzumab) for adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) who have undergone two or more lines of systemic therapy. New treatment options are critically needed for FL.

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Drugs FDA Approval Antibody Medicine 52

pharmaphorum

SEPTEMBER 12, 2022

Personalised medicine looks at developing or utilising treatments that target those differences. Personalised medicine targeting an individual’s genetic makeup or cancer’s genetic makeup is beneficial and is now more widely available than ever before. In other words, every person’s cancer has its own genetic construct.

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Genome Genomics Clinical Trials Clinical Trials 105

XTalks

FEBRUARY 16, 2023

However, the company last aired the ad on May 2, 2022, the same day they announced the US Food and Drug Administration (FDA) approval of Quviviq. Quviviq is an FDA-approved prescription medicine that helps treat insomnia in adults, with a low risk of dependence and side effects. estimating Indorsia spent $6.2

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The Pharma Data

DECEMBER 17, 2020

NASDAQ: REGN ) today announced that the New England Journal of Medicine (NEJM) has published initial clinical data from an ongoing seamless Phase 1/2/3 trial of the antibody cocktail casirivimab and imdevimab in non-hospitalized patients with COVID-19. Casirivimab and imdevimab injection is not FDA approved for any use.

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Antibody Medicine Production Trials 52