XTalks

OCTOBER 16, 2024

Hympavzi is the first anti-tissue factor pathway inhibitor (anti-TFPI) approved in the US for hemophilia A or B, and it’s also the first hemophilia therapy to be administered via a pre-filled auto-injector pen. Hympavzi reduces the activity of TFPI, a naturally occurring anticoagulation protein.

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FDA Approval Trials Clinical Trials Clinical Trials 105

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.

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FDA Approval Sales Trials Development 102

Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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Research Gene Gene Therapy Genomics 264

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.

FDA Approval 85

FDA Approval Genetics Protein Antibody 85

XTalks

JANUARY 26, 2023

This is the fourth US FDA approval that BeiGene has received for Brukinsa since 2019. Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations.

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FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

JUNE 23, 2022

In this episode, Ayesha and the team talked about the FDA approval of Eli Lilly’s JAK inhibitor Olumiant for the treatment of alopecia areata, also commonly just known as alopecia. The drug has become the first approved systemic treatment for the autoimmune disorder that causes patchy hair loss.

Life Science 52

Life Science FDA Approval Protein Drugs 52

pharmaphorum

NOVEMBER 12, 2021

Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients.

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Doctors Doctor Drugs Gene Expression 135

STAT News

NOVEMBER 23, 2022

The approval of Hemgenix provides physicians with a new, potentially curative treatment option for patients with severe hemophilia B, a rare bleeding disorder caused by a genetic mutation that prevents the body from producing sufficient quantities of a clotting protein called Factor IX.

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Gene Therapy FDA Approval Gene Genetics 97

XTalks

DECEMBER 13, 2023

The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. It also affects Hispanic Americans, but at a lower prevalence. The mutation causes red blood cells to develop a crescent or “sickle” shape.

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Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

JANUARY 3, 2025

For the first time in the US, adults living with familial chylomicronemia syndrome (FCS) have an FDA-approved treatment option. FCS is a rare genetic disorder that prevents the body from properly breaking down triglycerides (a type of fat in the blood), leading to dangerously high levels.

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FDA Approval Drugs Clinical Trials Clinical Trials 59

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 The genetic material of RSV (respiratory syncytial virus) encodes 11 proteins. The F-protein facilitates the fusion of the virus with the membrane of the host cell. How Does Abrysvo Work?

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Vaccine Vaccination FDA Approval Protein 98

The Pharma Data

APRIL 26, 2022

A drug used to treat asthma and allergies can bind to and block a crucial protein produced by the virus SARS-CoV-2, and reduce viral replication in human immune cells, according to a new study by researchers at the Indian Institute of Science (IISc). Targeting Nsp1 could therefore reduce the damage inflicted by the virus.

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Protein Drugs Allergies FDA Approval 52

The Pharma Data

AUGUST 13, 2020

NS Pharma has claimed a tentative FDA approval for its Viltepso (viltolarsen) injection in the treatment of Duchenne muscular dystrophy (DMD) in patients who are suitable to receive exon 53 skipping therapy. Mutations in the DMD gene can lead to an absence of the muscle cell protein dystrophin. of normal at baseline to 5.9%

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FDA Approval Genetics Protein Gene 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.

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FDA Approval Engineer Clinical Trials Clinical Trials 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). 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

MAY 15, 2023

Chiesi Global Rare Diseases in collaboration with Protalix BioTherapeutics announced the US Food and Drug Administration (FDA) approval of their new medication Elfabrio (pegunigalsidase alfa-iwxj) for the treatment of Fabry disease. Genetic confirmatory testing is then conducted to identify the specific mutations in the GLA gene.

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FDA Approval Gene Genetics Clinical Trials 98

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. It is injected into a patient’s muscle tissue.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

Worldwide Clinical Trials

MAY 30, 2024

Tofersen (Intrathecal injection) This is an antisense oligonucleotide (ASO) that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient.

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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”. What, then, is the solution? It also bodes well for improving clinical trial success rates. AI relies on data.

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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

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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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 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

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 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

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.

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XTalks

JANUARY 4, 2024

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions.

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Gene Therapy Gene Gene Editing Sales 111

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

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 Gene Clinical Trials 111

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

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

JUNE 8, 2021

SMA is a rare genetic condition that leads to a loss of motor neurons that results in progressive muscle weakness and wasting, paralysis and, when left untreated in its most severe form, breathing difficulties leading to permanent ventilation or death for most patients by the age of two. With a price tag of over $2.5

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Gene Therapy Gene Protein Trials 110

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.

FDA Approval 52

FDA Approval Protein In-Vitro Medicine 52

pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Furthermore, each cancer has various gene changes – also referred to as the genetic makeup of cancer. Tailored oncology.

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Genome Genomics Clinical Trials Clinical Trials 105

pharmaphorum

MAY 21, 2021

The decision means the EU looks set to get access to Skysona ahead of the US, as bluebird doesn’t intend to file for FDA approval of the gene therapy until the middle of this year – around the same time as the EMA is likely to make its final decision.

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Gene Therapy Gene FDA Approval Clinical Trials 96

XTalks

SEPTEMBER 17, 2021

The ALS Association is pushing for rapid FDA approval of Amylyx Pharmaceuticals Inc.’s The complex is designed to target the toxic unfolded proteins in the endoplasmic reticulum, as well as mitochondrial dysfunction, which is being increasingly recognized as a pathogenic mechanism underlying the disease.

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Trials Drugs FDA Approval Clinical Trials 98