Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. References [1] 2023 Novel Small Molecule FDA Drug Approvals. 2] Iversen et al., Health Sci Rep.

Small Molecule 52

Small Molecule FDA Approval FDA Pharmacokinetics 52

New Drug Approvals

APRIL 2, 2025

1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 2] [6] Suzetrigine is the first medication to be approved by the US Food and Drug Administration (FDA) in this new class of pain management medicines. [2] Food and Drug Administration (FDA).

FDA 62

FDA Therapies Trials Treatment 62

Drug Target Review

APRIL 12, 2024

This holds particular significance, as it is a prerequisite for FDA approval in biotechnology that any cell clones must originate from a single-cell progenitor. He is co-inventor of SEED Biosciences’ core technology called DISPENCELL and co-authored several patents and scientific articles that made the cover of SLAS Technology in 2020.

Cell Biology 106

Cell Biology FDA Approval FDA Research 106

The Pharma Data

NOVEMBER 25, 2020

November 25, 2020 — Under FDA law, approval of a new drug requires substantial evidence of effectiveness and a demonstration of safety for the drug’s intended use(s). The approval of remdesivir ( Veklury ) for the treatment of patients hospitalized with COVID-19 met this legal and scientific standard. Source: FDA.

FDA 52

FDA Treatment Clinical Trials Hospitals 52

Drug Target Review

APRIL 12, 2024

This holds particular significance, as it is a prerequisite for FDA approval in biotechnology that any cell clones must originate from a single-cell progenitor. He is co-inventor of SEED Biosciences’ core technology called DISPENCELL and co-authored several patents and scientific articles that made the cover of SLAS Technology in 2020.

Cell Biology 98

Cell Biology FDA Approval FDA Research 98

DrugBank

FEBRUARY 1, 2024

The article highlights the myriad ways in which we have focused on making DrugBank not just a knowledgebase, but a vital partner in your healthcare research. This is in service of modernizing how you engage with medical research and treatment options so that you can shorten the road to medical breakthroughs. DrugBank 6.0

Research 52

Research Clinical Trials FDA Approval Trials 52

KIF1A

JULY 1, 2023

Read the Pre-print Rare Roundup A seemingly small semantic issue is a major roadblock to develop treatments for rare diseases We’ve spoken often about the goal of repurposing existing drugs to better treat KAND – this is a reality for many families who utilize off-label prescriptions. * What’s a pre-print?

Small Molecule 98

Small Molecule FDA Approval Disease Pharmaceutical Companies 98

New Drug Approvals

DECEMBER 19, 2022

FDA 12/1/2022, To treat adults with relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation, Rezlidhia Olutasidenib , sold under the brand name Rezlidhia , is an anticancer medication used to treat relapsed or refractory acute myeloid leukemia. [1] 1] It is taken by mouth. [1] Hz, 1 H), 4.62−4.75

FDA Approval 52

FDA Approval FDA Treatment Pharmaceuticals 52

Drug Target Review

MAY 15, 2024

Of course, with the billions of dollars being invested in AI-led drug discovery programmes, one would think that this is a pivotal moment in patient treatment – like Isomorphic Labs, Recursion, Exscientia, Flagship Pioneering, the big biopharmaceuticals, and even a “$1B+ bet” 2 on Xaira Therapeutics, who have invested heavily in the promise of AI.

FDA Approval 59

FDA Approval FDA Science Marketing 59

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]

FDA Approval 62

FDA Approval FDA Clinical Trials Trials 62

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. 20 August 2024. 20 August 2024.

FDA Approval 48

FDA Approval FDA International Treatment 48

National Institute on Drug Abuse: Nora's Blog

APRIL 10, 2023

mfleming Mon, 04/10/2023 - 14:43 Nora's Blog April 13, 2023 Image Getty Images/ AleksandarGeorgiev This article originally appeared in the Milken Institute’s Power of Ideas series. The need for new treatments is real and urgent. Is It Too Soon To Start Talking about a Cure for Addiction?

FDA Approval 52

FDA Approval FDA Treatment Clinical Trials 52

New Drug Approvals

SEPTEMBER 20, 2023

To use with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma Motixafortide , sold under the brand name Aphexda , is a medication used for the treatment of multiple myeloma. [1] 1] It is given by subcutaneous injection. [1]

FDA Approval 52

FDA Approval FDA International Clinical Trials 52

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

FDA 62

FDA International Trials FDA Approval 62

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.

Disease 52

Disease FDA FDA Approval Treatment 52

New Drug Approvals

DECEMBER 16, 2023

2 On December 6th, 2023, Iptacopan under the brand name Fabhalta was approved by the FDA for the treatment of adults with PNH. This approval was based on favorable results obtained from the phase III APPL-PNH and APPOINT-PNH studies, where 82.3% Food and Drug Administration (FDA). 1] It is taken by mouth. [1]

FDA 62

FDA Small Molecule FDA Approval Treatment 62

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] 3] All participants were required to have progression on aromatase inhibitor-based treatment. [3] PMID 37256976.

FDA Approval 62

FDA Approval FDA Production Therapies 62

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]

FDA 52

FDA Clinical Trials FDA Approval Treatment 52

The Pharma Data

OCTOBER 21, 2020

It’s reassuring to know that even in the midst of a global pandemic that has led to restricted access to some clinics and providers, there were few injection visit interruptions and those that occurred were effectively managed by providing participants with oral treatment. Kimberly Smith, M.D., Shionogi joined in October 2012. Source: GSK .

Clinical Development 52

Clinical Development Therapies Clinical Trials Treatment 52

New Drug Approvals

AUGUST 26, 2023

Zuranolone CAS 1632051-40-1 Zurzuvae FDA APPROVED 8/4/2023, To treat postpartum depression Press Release WeightAverage: 409.574 Monoisotopic: 409.272927379Chemical FormulaC 25 H 35 N 3 O 2 SAGE 217 SAGE-217 SAGE217 Zuranolone , sold under the brand name Zurzuvae , is a medication used for the treatment of postpartum depression. [1]

FDA 52

FDA FDA Approval Clinical Trials Treatment 52

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. According to the study’s authors, “The flexibility of the C1 ad molecular delivery platform offers several advantages for the rapid generation of new treatments for neurological disorders.

Science 52

Science Molecular Biology Treatment Production 52

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]. Current macrocycles in clinical use generally focus on treatment of infectious diseases, cancer and auto-immune disorders.

Drugs 52

Drugs Metabolic Stability FDA Approval Treatment 52

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]. Current macrocycles in clinical use generally focus on treatment of infectious diseases, cancer and auto-immune disorders.

Metabolic Stability 52

Metabolic Stability Drugs FDA Approval Treatment 52

Advarra

JULY 19, 2022

Q: If an emergency use authorization (EUA) is granted, once there is an approved treatment, does that mean that the EUA is no longer valid? If the sponsor wants their drug approved, they need to complete all clinical studies and submit an application. is this acceptable to the FDA? A: Yes, the EUA is just temporary.

Packaging 52

Packaging FDA Trials Regulations 52

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. Satam et al.

Clinical Trials 52

Clinical Trials Trials RNA Bioinformatics 52

The Pharma Data

JANUARY 18, 2021

OKYO has been developing the chemerin molecule as a promising anti-inflammatory treatment for dry-eye disease (“DED”) licensed from researchers at On Target Therapeutics LLC. When Lucentis ® (Ranibizumab) received FDA approval in late June 2006, the new macular degeneration drug was celebrated as a major medical breakthrough.

Science 40

Science Virus Treatment Disease 40

Vial

JUNE 12, 2024

As the names imply, disease-specific PROs measure features specific to a disease or syndrome, while condition-specific PROs describe aspects of a particular condition, intervention, or treatment not uniquely associated with one disease. Such complications may otherwise be missed if only disease-specific PROs are used.

Clinical Trials 52

Clinical Trials Trials FDA Clinical Research 52

Advarra

SEPTEMBER 30, 2022

In the funnel of transformative solutions, we see too many solutions stuck in pilot mode after a key case study or two, with few solutions trickling into transformative and driving toward faster treatments. . Our article, It’s Time for Sites to ‘Bring Your Own Technology’ (BYOT) outlines why site technology is so important for sites. .

Research 52

Research Clinical Research Trials FDA Approval 52

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. The FDA will follow these procedures for both agency-initiated operations (e.g.,

Science 40

Science FDA Clinical Trials Clinical Pharmacology 40

Policy Prescription

OCTOBER 22, 2024

They offer this advice in a new article in the Journal of the American Medical Association. Article Review The article, Strategies to Help Patients Navigate High Prescription Drug Costs , offers very digestible and evidence-driven guidance for clinicians to better assist their patients with the tangled web of drug pricing in America.

Online Pharmacies 113

Online Pharmacies Pharmacy Drugs FDA Approval 113

The Pharma Data

NOVEMBER 24, 2020

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. The approval of Oxlumo is a further testament to the impact RNAi therapeutics can have in transforming the treatment of severe, life-threatening diseases like PH1. CAMBRIDGE, Mass.–(BUSINESS –(BUSINESS WIRE)–Nov.

FDA Approval 52

FDA Approval Treatment FDA RNA 52

The Pharma Data

NOVEMBER 8, 2020

FDA Approves Sesquient (fosphenytoin sodium) for the Treatment of Status Epilepticus in Adult and Pediatric Patients. Food and Drug Administration (FDA) has approved Sesquient (fosphenytoin sodium for injection) for the treatment of status epilepticus in adult and pediatric patients. About Sesquient.

FDA Approval 52

FDA Approval FDA Long-Term Care Facilities Treatment 52

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). .

FDA Approval 52

FDA Approval FDA Clinical Trials Disease 52

KIF1A

JUNE 3, 2023

In this week’s article, researchers in Japan investigated the genetics of hereditary spastic paraplegia in a retrospective study : this means they went back and analyzed existing data, in this case the genetics and clinical profiles of 37 children diagnosed with HSP. In many cases, drug approval relies on a clinical trial’s primary endpoint.

FDA 98

FDA Trials FDA Approval Disease 98

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)-,

FDA Approval 52

FDA Approval FDA Treatment Clinical Trials 52

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.

FDA 40

FDA Treatment Production Trials 40

Agency IQ

MAY 17, 2024

The bill is effectively a successor to the RACE for Children Act of 2017, which became law as part of the FDA Reauthorization Act of 2017 (FDARA). The law required some drug sponsors to assess the efficacy of adult cancer treatments in children under specific conditions. DORIS MATSUI (D-Calif.) and GUS BILIRAKIS (R-Fla.),

FDA 40

FDA Disease FDA Approval Production 40