Pharmaceutical Technology

JUNE 9, 2023

Rocket Pharmaceuticals stated that the fast-track designation enables increased communication with the regulator across the development cycle of the gene therapy. Fast-track status also offers the potential for expedited approval of RP-A601 and permits a rolling review of the biologic licence application.

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Advarra

JANUARY 11, 2024

While mRNA usage has played several roles in clinical research , oncology researchers in particular are eager to explore the possibilities of mRNA-based cancer vaccines. The mRNA constructs used in COVID-19 vaccines, for example, direct cells to produce a version of the “spike” protein studding the surface of SARS-CoV-2.

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XTalks

JANUARY 8, 2024

Initially receiving accelerated approval in December 2021 based on proteinuria as a surrogate marker, Tarpeyo has now achieved the status of being the first treatment for IgAN to receive full FDA approval predicated on evaluations of kidney function.

FDA Approval 64

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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. However, NHP models cannot fully predict AAV transduction in the human liver.

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Gene Therapy Gene Clinical Trials Clinical Trials 64

XTalks

JUNE 28, 2023

DMD is caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells. The FDA approval came after several delays and a narrow advisory committee vote of eight to six in favor of the gene therapy’s risk-benefit profile. It is injected into a patient’s muscle tissue.

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Gene Therapy Gene Protein FDA Approval 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). A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.

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Drug Discovery World

MAY 21, 2024

Modern experimental procedures, such as immunisation, B-cell screening, and synthetic library generation, have been pivotal in developing approximately 80 FDA-approved antibody therapeutics. Existing antibody discovery methods have mostly failed for certain target groups, such as G protein-coupled receptors (GPCRs) and ion channels.

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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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XTalks

FEBRUARY 8, 2024

Ozempic (Semaglutide) Ozempic sales in 2022: $8.713 billion Company/Developer: Novo Nordisk Date of first FDA approval: December 5, 2017 Indications Ozempic is FDA-approved for: Type 2 diabetes Price of Ozempic: $1,029 for 1.5 Read on to learn more about the top 15 diabetes drugs in 2023, based on 2022 sales statistics.

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XTalks

NOVEMBER 26, 2020

In addition to innovative new antibody drugs and mRNA vaccines, researchers have found that the sleep-regulating hormone melatonin may also offer protection against COVID-19. Melatonin, which regulates the body’s circadian rhythm and sleep-wake cycle, is also available in pill form for use as a sleep aid.

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The Pharma Data

JANUARY 15, 2021

Food and Drug Administration (FDA) approval of Darzalex Faspro ® (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.[1]

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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.

Research 52

Research Drugs Protein FDA Approval 52

XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

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The Pharma Data

JANUARY 29, 2021

Additional safety and efficacy data for the vaccine will continue to accumulate from ongoing clinical trials and is expected to be published in the coming weeks. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body. Source: AstraZeneca .

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The Pharma Data

JANUARY 29, 2021

Additional safety and efficacy data for the vaccine will continue to accumulate from ongoing clinical trials and is expected to be published in the coming weeks. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body. Source: AstraZeneca.

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Vaccination Vaccine Clinical Trials Clinical Trials 52

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

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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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STAT News

MARCH 6, 2023

Unlike most people who receive medications yet to be cleared by the Food and Drug Administration, none of these patients is getting the vaccines as part of a clinical trial. Instead, the 26 patients paid the foundation to create the vaccines and provide them through the FDA’s expanded access program.

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The Pharma Data

JANUARY 6, 2021

The vaccine was shown in clinical trials to be safe and effective at preventing symptomatic COVID-19, with no severe cases and no hospitalisations more than 14 days after the second dose. The approval in India is an important milestone as it will enable to supply India but also a large number of countries around the world.

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XTalks

APRIL 26, 2021

Related: After 18 Years, FDA Approves Malaria Prevention Drug. The vaccine is protein-based and contains an adjuvant (MM), a component that helps generate a stronger immune response, that was developed by Novavax. Malaria Vaccine Phase II Trial. Changing the Face of Malaria.

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The Pharma Data

MAY 17, 2023

The safety profile of fenebrutinib was consistent with previous and ongoing fenebrutinib clinical trials across more than 2,400 people to date. The Phase III fenebrutinib clinical trial programme in RMS and primary progressive MS (PPMS) is ongoing. There were no new safety concerns identified in the FENopta study.

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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 52

Delveinsight

OCTOBER 1, 2021

This disease is associated with antibodies directed against the acetylcholine receptor, muscle-specific kinase (MUSK), lipoprotein-related protein 4 (LRP4), or agrin in the postsynaptic membrane at the neuromuscular junction. Myasthenia Gravis Approved Therapies. Some of the approved drugs are: Prograf (Tacrolimus).

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The Pharma Data

DECEMBER 18, 2020

The vaccine contains a small piece of the SARS-CoV-2 virus’s mRNA that instructs cells in the body to make the virus’s distinctive “spike” protein. Director of the FDA’s Center for Biologics Evaluation and Research. Director of the FDA’s Center for Biologics Evaluation and Research.

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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. Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012.

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Drug Discovery World

APRIL 14, 2023

Kathryn E Wellen, PhD – Outstanding Achievement in Basic Cancer Research Wellen is an investigator at the Penn Epigenetics Institute and Vice Chair and Professor in the Department of Cancer Biology in the Perelman School of Medicine at the University of Pennsylvania. Jaffee’s award lecture will be held on Monday April 17 at 4:45pm ET.

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Genetics Research Medicine Clinical Research 52

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

NOVEMBER 16, 2020

We are pleased to submit these extended stability conditions for mRNA-1273 to regulators for approval. Food and Drug Administration (FDA) to submit data from its ongoing stability testing for approval. MRNA-1273 (SARS-CoV-2 vaccine) FDA Approval History. About mRNA-1273. Source: Moderna, Inc. .

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The Pharma Data

JANUARY 13, 2021

Food and Drug Administration (FDA) has designated as a Fast Track development program the investigation of Brilacidin as a potential treatment for COVID-19. Brilacidin is a first-in-class Host Defense Protein (HDP) mimetic with antiviral, anti-inflammatory and antibacterial properties. Source link.

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Clinical Trials Clinical Trials Trials In-Vitro 52

pharmaphorum

NOVEMBER 26, 2020

Y-mAbs Therapeutics has claimed its first product approval, getting a green light from the FDA for Danyelza for the rare cancer neuroblastoma. It’s very exciting to see this treatment go from being an experimental therapy used at my daughter’s bedside to now being FDA approved,” said Gad, who is chairman and president of Y-mAbs. “I

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Drugs Antibody Clinical Trials Clinical Trials 105

Drug Discovery World

AUGUST 21, 2023

Combination therapy with pembrolizumab In July 2021, the Food and Drug Administration (FDA) approved Merck’s pembrolizumab (Keytruda) for high-risk, early-stage TNBC in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery. and 79.6%, respectively.

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Protein Trials Clinical Trials Clinical Trials 52

XTalks

AUGUST 2, 2023

Paxlovid was first authorized under the FDA Emergency Use Authorization in December 2021 ; however, it has received FDA approval on May 25, 2023. pneumoniae serotypes) replaced the company’s first pneumococcal conjugate vaccine Prevnar (PCV7, approved by the FDA in February 2000) in a February 2010 FDA approval.

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The Pharma Data

FEBRUARY 8, 2021

Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019. 6] ERLEADA ® is being studied in five Phase 3 registrational clinical trials. ERLEADA ® received U.S. 2 To date, more than 10,000 patients worldwide have been treated with ERLEADA ®.

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Drug Discovery World

AUGUST 10, 2022

Finally, in 2017, after years of improving T cell engineering techniques, the first chimeric antigen receptor (CAR) T cell therapy received FDA approval. Since 2017, five CAR T cell therapies have been approved and have shown incredible success in the clinic. Identifying the right CAR T cell target.

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XTalks

JUNE 12, 2023

The growth is attributed to regulatory approvals, strategic partnerships and the acquisitions of innovative medical technology companies. Additionally, Abbott launched over 125 new products in 2022, in conjunction with a series of FDA approvals. The company reported an investment of $2.7 billion ($22.83 billion USD), a 20.65

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The Pharma Data

JUNE 18, 2021

Clinical remission per DAS28-CRP is defined as Disease Activity Score with 28 joint counts C-reactive protein (DAS28-CRP) less than 2.6. Clinical remission per CDAI is defined as CDAI less than or equal to 2.8. More information on this trial can be found at www.clinicaltrials.gov (NCT02629159). years of treatment.

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The Pharma Data

APRIL 8, 2021

Data from the Phase III SAkuraStar and SAkuraSky clinical trials reinforce the favourable safety and efficacy of this therapy for those living with NMOSD, including patients with concomitant autoimmune diseases (CAIDs). P6: Neuromuscular Disorders and Clinical Trials. P6: Neuromuscular Disorders and Clinical Trials.

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