Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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Alta Sciences

APRIL 17, 2024

In this blog article, we review some of these areas of investigation where Altasciences has robust expertise and solution offerings. According to a 2022 article published in Molecular Psychiatry, treatment resistance affects 20 to 60% of patients with psychiatric disorders.

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The Pharma Data

OCTOBER 21, 2020

In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days. Source: GSK .

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New Drug Approvals

SEPTEMBER 10, 2024

2] Vorasidenib was approved for medical use in the United States in August 2024. [2] 2] [3] It is the first approval by the US Food and Drug Administration (FDA) of a systemic therapy for people with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation. [2]

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New Drug Approvals

SEPTEMBER 6, 2024

2 , 3 Lazertinib was first approved in South Korea on January 18, 2021, for the treatment of EGFR T790M mutation-positive non-small cell lung cancer (NSCLC) with EGFR mutations. 1 It was approved by the FDA on August 19, 2024. 1 It was approved by the FDA on August 19, 2024. Food and Drug Administration (FDA).

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New Drug Approvals

SEPTEMBER 8, 2024

2222514-07-8 ] Palopegteriparatide , sold under the brand name Yorvipath , is a hormone replacement therapy used for the treatment of hypoparathyroidism. [1] 1] Palopegteriparatide was approved for medical use in the European Union in November 2023, [2] and in the United States in August 2024. [1] mg/dL, a magnesium concentration ≥1.3

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New Drug Approvals

DECEMBER 24, 2023

3] In November 2023, capivasertib was approved in the United States for people with hormone receptor-positive, human epidermal growth factor receptor 2 -negative breast cancer when used in combination with fulvestrant. [3] Jump up to: a b c d e f “FDA approves capivasertib with fulvestrant for breast cancer” U.S.

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Nvidia Developer: Drug Discovery

JULY 14, 2022

The five existing FDA-approved therapies for the disease are unable to halt or reverse this loss of function, which affects more than 700,000 people around the world. “The Read more in Insilico Medicine’s press release and Frontiers in Aging Neuroscience article. ALS is a debilitating disease. Do you have a startup?

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New Drug Approvals

APRIL 11, 2025

2] Fitusiran was approved for medical use in the United States in March 2025. [2] 2] The fixed dose of fitusiran is not approved because it led to excessive clotting in some participants. [2] 2] The US Food and Drug Administration (FDA) granted the application for fitusiran orphan drug and fast track designations. .

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New Drug Approvals

JANUARY 3, 2023

Adagrasib Formula C32H35ClFN7O2 cas 2326521-71-3 Mol weight 604.1174 Antineoplastic Disease Non-small cell lung cancer 2022/12/12 FDA APPROVED, KRAZATI (Mirati Therapeutics) MRTX-849 MRTX849 KRAS G12C inhibitor MRTX849 Adagrasib , sold under the brand name Krazati , is an anticancer medication used to treat non-small cell lung cancer. [1]

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The Premier Consulting Blog

OCTOBER 11, 2022

However, acceptance of that data depends on a sponsor’s understanding of the FDA criteria for historical control groups, study data standards, and clinical meaningfulness. In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases.

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Agency IQ

MARCH 25, 2024

FDA addressed the expanding practice of drug compounding in 1992 by issuing a compliance policy guide that clarified that pharmacies which compounded products at certain scales, for certain purposes, or without FDA approval were clearly operating “outside the bounds of traditional pharmacy practice.”

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New Drug Approvals

DECEMBER 16, 2023

Retrieved 10 December 2023. ^ “Novartis receives FDA approval for Fabhalta (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH” Novartis (Press release). Food and Drug Administration (FDA). twitter +919321316780 call whatsaapp EMAIL.

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Vial

DECEMBER 5, 2023

In 2017, the Food & Drug Administration (FDA) approved the first gene therapy for cancer and for inherited diseases, the first multiplex NGS panel for companion diagnostics (CDx), and the first drug targeting a genetic signature though not a disease. Fountzilas et al.

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Metabolite Tales Blog

JULY 5, 2023

Metabolism of de novo-designed macrocyclic drugs approved by the FDA By Julia Shanu-Wilson To date, only 4% (67) of FDA approved drugs are macrocycles [1]. A combination therapy of glecaprevir and pibrentasvir has recently been implicated in a case of liver injury. Drug Hunter article, June 2023.

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Metabolite Tales Blog

JULY 5, 2023

Metabolism of de novo-designed macrocyclic drugs approved by the FDA By Julia Shanu-Wilson To date, only 4% (67) of FDA approved drugs are macrocycles [1]. A combination therapy of glecaprevir and pibrentasvir has recently been implicated in a case of liver injury. Drug Hunter article, June 2023.

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The Pharma Data

JANUARY 6, 2021

The complete text of the article titled, “Neuronal Delivery of Antibodies has Therapeutic Effects in Animal Models of Botulism,” can be found h e re. This is a landmark study in converting the power of lethal botulinum neurotoxins into therapies.

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Advarra

SEPTEMBER 30, 2022

In the past decade, trials have evolved to include not just traditional drugs and medical devices, but also innovations like cell and gene therapies, wearables, and more. For example, the sponsor needs clinical data in a format consistent across sites to run statistical analysis and drive to expedient FDA approval. .

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The Pharma Data

JANUARY 18, 2021

This pioneering research led to the clinical development of a humanized anti-VEGF Fab (Ranibizumab, Lucentis ® ), which has also been approved as a therapy for neovascular age-related macular degeneration (AMD), retinal vein occlusion and diabetic macular edema. Enquiries: OKYO Pharma Limited. Gary S.

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Codon

MARCH 28, 2023

Although this article has a lot, too!) The rest of this article gives a brief history of DARPA and its neurotechnology programs. Researchers used an off-the-shelf, FDA-approved machine, developed by Blackrock Neurotech ; the algorithms were the main advancement.) Please reach out to suggest edits or additions.

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Agency IQ

SEPTEMBER 15, 2023

Until recently, the FDA relied on a monograph process through which firms could bring OTC drugs to market without FDA approval so long as it adhered to pre-set terms under the monograph. CHPA considered the original studies to be sound and instead found issues with the newer studies.

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KIF1A

MARCH 23, 2024

In this week’s article, researchers in Serbia expanded genetic testing for adult HSP patients. We know that gene therapies are on the horizon, so it’s important to take note when groups make tangible progress. The gene therapy is currently the world’s most expensive medicine, priced at $4.25

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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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The Pharma Data

NOVEMBER 20, 2020

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies. Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that the U.S. PALO ALTO, Calif.,

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Agency IQ

MAY 17, 2024

As the legislation explained, the FDA could require the sponsor of an approved “molecularly targeted” medicine to complete a pediatric assessment if the target is “germane” to a pediatric cancer, either in terms of the number of patients or the therapeutic benefit over existing therapies. DORIS MATSUI (D-Calif.)

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New Drug Approvals

SEPTEMBER 10, 2024

Deuruxolitinib C 17 H 18 N 6 , 314.422 Fda approved Leqselvi , 7/25/2024, To treat severe alopecia areata C-21543, CTP 543, CTP-543, CTP543 (3r)-3-(2,2,3,3,4,4,5,5-d8)cyclopentyl-3-(4-(7h-pyrrolo(2,3-d)pyrimidin-4-yl)-1h-pyrazol-1-yl)propanenitrile 1h-pyrazole-1-propanenitrile,beta.-(cyclopentyl-2,2,3,3,4,4,5,5-d8)-4-(7h-pyrrolo(2,3-d)pyrimidin-4-yl)-,

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Agency IQ

JANUARY 19, 2024

Only one product was an advanced therapy medicinal product (ATMP), three products held PRIME designation, and three products were evaluated under accelerated assessments. However, BeiGene announced that it is seeking approval in the U.S. Inaqovi (cedazuridine/decitabine; Otsuka Pharmaceuticals) is approved in the E.U.

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Agency IQ

OCTOBER 6, 2023

In a first, FDA tackles treatments for stimulant use disorder A new draft guidance from the FDA gives developers a roadmap to advance the development of novel therapies to address stimulant use disorders. While stimulant use disorder is increasing, there are currently no FDA-approved medications.

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The Pharma Data

OCTOBER 22, 2020

With today’s FDA approval, and regulatory approvals or temporary authorizations in approximately 50 additional countries around the world, Veklury® (remdesivir) is one of the tools available today. [ii] Related Articles: Veklury (remdesivir) FDA Approval History. Published online on October 8, 2020.

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Agency IQ

JULY 21, 2023

subsequent superior/inferior supportive care, lifestyle changes, and/or use of additional therapies). That’s why, according to FDA’s Oncology Center of Excellence, the determination of an appropriate endpoint in oncology is based on the specific disease and is highly dependent upon numerous factors. But if not ORR or PFS, then what?

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The Pharma Data

DECEMBER 18, 2020

AstraZeneca in CVRM Cardiovascular, Renal and Metabolism (CVRM) together forms one of AstraZeneca’s three therapy areas and is a key growth driver for the Company. Related Articles: Roxadustat FDA Approval History. The Lancet 2020; 395(10225):709–33. . Source: AstraZeneca . Posted: December 2020.

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The Pharma Data

FEBRUARY 1, 2021

Plegridy is a proven, effective therapy for relapsing MS, and this approval gives new and current MS patients a different delivery method that has the potential to significantly reduce injection site reactions.”. Related Articles: Plegridy (peginterferon beta-1a) FDA Approval History. Source: Biogen.

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The Anticancer Fund

OCTOBER 18, 2023

Being listed is no endorsement of the results and conclusions of the article. All articles need to be critically assessed and viewed in their broader research context. The results show that enoxacin, an FDA approved antibiotic, was able to slow tumour growth compared to untreated controls and compared to etoposide alone.

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Codon

JUNE 18, 2024

Sign up to receive articles from Asimov Press. In 2010, Shapiro picked up a review article and noticed two short paragraphs —included almost as an afterthought—about gas vesicles. At least six types of CAR-T cells have already passed through clinical trials and garnered FDA approval. Richly storied.

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Codon

APRIL 2, 2023

This is Codon Digest, a weekly roundup of research papers, news articles, and industry highlights about engineered biology. Read A one-time gene therapy injection for spinal muscular atrophy. Mice and human cells that were given the therapy had normal levels of the survival motor neuron protein, and no symptoms. Gene Therapy.

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The Pharma Data

JUNE 18, 2021

. “These data add to the overall body of evidence that patients treated with ibrutinib can achieve extended progression-free survival and overall survival when used as a first-line therapy,” said Paul M. Single-agent ibrutinib was the most common treatment across line of therapy at enrollment. Barr , M.D., IMBRUVICA ®.

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The Pharma Data

DECEMBER 21, 2020

Tagrisso was recently granted Breakthrough Therapy Designation for patients in the early-stage disease setting by the US FDA. Related Articles: Tagrisso (osimertinib) FDA Approval History. The safety and tolerability of Tagrisso in this trial was consistent with previous trials in the metastatic setting.

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