Disease and Drug Trials - Drug Discovery Digest

Tofersen

New Drug Approvals

MAY 17, 2025

“FDA grants accelerated approval for Biogen ALS drug that treats rare form of the disease” CNBC. “FDA advisors vote against effectiveness of Biogen’s ALS drug for rare and aggressive form of the disease” CNBC. Approves Drug for Rare Form of A.L.S.” Retrieved 25 April 2023.

FDA Approval

FDA Approval FDA Treatment Clinical Trials

Elacestrant

New Drug Approvals

MAY 9, 2025

1] History The efficacy of elacestrant was evaluated in the EMERALD trial, which was a randomized, open-label, active-controlled, multicenter study involving 478 postmenopausal women and men with ER-positive, HER2-negative advanced or metastatic breast cancer. 1] [4] It is taken by mouth. [1] 3] [7] PATENTS Cruskie MP, et al.

FDA

FDA Pharmacokinetics Therapies Treatment

Inavolisib

New Drug Approvals

APRIL 25, 2025

2] [3] It is an inhibitor and degrader of mutant phosphatidylinositol 3-kinase (PI3K) alpha. [4] 4] The PI3K-mediated signalling pathway has shown to play an important role in the development of tumours as dysregulation is commonly associated with tumour growth and resistance to antineoplastic agents and radiotherapy. [5]

FDA

FDA FDA Approval Therapies Trials

Pirtobrutinib

New Drug Approvals

MAY 8, 2025

5] History Pirtobrutinib is manufactured by Eli Lilly and Company and was approved by the US Food and Drug Administration in January 2023, for the treatment of mantle cell lymphoma that has become refractory to other BTK inhibitors. [13] 6] The same trial was used to assess safety and efficacy. [6] Food and Drug Administration (FDA).

FDA Approval

FDA Approval FDA Therapies Trials

Zilucoplan

New Drug Approvals

JUNE 15, 2025

Its sodium salt is used for the treatment of generalised myasthenia gravis (a disease that leads to muscle weakness and tiredness) in adults whose immune system produces antibodies against acetylcholine receptors. 6] [9] [10] It is a complement inhibitor that is injected subcutaneously (under the skin). [6]

FDA

FDA Treatment Clinical Trials Production

Leniolisib

New Drug Approvals

MAY 15, 2025

5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] After prophylactic or therapeutic dosing, CDZ173 potently inhibited antigen-specific antibody production and reduced disease symptoms in a rat collagen-induced arthritis model. mmol) in CH 2 CI 2 (100 mL), was added TFA (41.8

FDA Approval

FDA Approval FDA Treatment International

ASH23: CAR-T for autoimmune disease, real-world questions and sickle cell’s social media mentions

BioPharma Drive: Drug Pricing

DECEMBER 9, 2023

Study results disclosed at the American Society of Hematology meeting Saturday suggested potential for cell therapy in lupus and pointed out drug trial limitations.

Drug Trials

Drug Trials Disease Therapies Trials

AZVUDINE

New Drug Approvals

JULY 18, 2025

-D-ARABINOFURANOSYL)- 4′-C-Azido-2′-deoxy-2′-fluoro-b-D-arabinocytidine Azvudine is an antiviral drug which acts as a reverse transcriptase inhibitor. [3] 9] Azvudine is under investigation in clinical trial NCT04668235 (Study on Safety and Clinical Efficacy of AZVUDINE in COVID-19 Patients (Sars-cov-2 Infected)).

RNA

RNA Clinical Trials Virus Trials

What the FDA’s guidance on diversity means for rare disease sponsors

Conversations in Drug Development Trends

NOVEMBER 3, 2022

This plan, designed to help make clinical trial enrollment more representative, stands to support diversity, equity and inclusion (DEI) initiatives in R&D — a sector that certainly needs more attention across all clinical trials. But in rare disease trials, that can be challenging given the barriers inherent in U.S.

Disease

Disease Clinical Trials Trials International

An Evolving Regulatory Environment for Rare and Orphan Diseases

Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Before 1983, only 38 orphan drugs had received U.S. Food and Drug Administration (FDA) approval.

Disease

Disease Clinical Trials FDA Approval FDA

Advanced 3D cell-based technologies

Drug Target Review

SEPTEMBER 19, 2023

The chips can be modelled with primary cells, IPS derived stem cells or patient derived cells and potentiates to unlock molecular mechanisms that drive the disease pathophysiology, holding a greater promise. Human organs-on-chips for disease modelling, drug development and personalized medicine. Lab on a Chip. 2012;12(12):2156-.

Animal Testing

Animal Testing Clinical Trials Pharmaceutical Companies FDA

Levacetylleucine

New Drug Approvals

APRIL 30, 2025

1] [2] [3] Levacetylleucine is the second medication approved by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease type C. [2] 1] [2] Levacetylleucine is a modified version of the amino acid leucine. [1] 1] It is the L -form of acetylleucine. 1] It is the L -form of acetylleucine. 24 September 2024.

FDA

FDA FDA Approval Disease Trials

Case study: NCI’s Molecular Targets Platform

The Open Targets Blog

OCTOBER 31, 2023

The goal of MTP is to integrate the FDA’s Relevant Molecular Target List and enrich the platform with pre-clinical paediatric cancer datasets containing somatic variation, gene expression, and gene fusion data to aid in identifying new paediatric cancer-specific drug targets. earlier this year.

Bioinformatics

Bioinformatics Engineering Clinical Trials Therapies

Top Three Reasons Why Your Medical Device Needs a Clinical Trial

Advarra

MARCH 21, 2023

Medical devices, IVDs, and digital therapeutics have the potential to bring significant health benefits to patients of every age group, including healthy populations and those with varying severity of health ailments and disease burdens. Think of the intent or purpose for the intended use (e.g.,

Clinical Trials

Clinical Trials Trials Production Regulations

Article EMA Thank You What we expect European regulators to do in May 2024

Agency IQ

MAY 3, 2024

Start Date End Date Event Event Type Organization 4/30/2024 4/30/2024 Seminar No. Start Date End Date Event Event Type Organization 4/30/2024 4/30/2024 Seminar No. legislation The list below includes legislation proposed by the European Commission that is yet-to-be-published.

Regulations

Regulations Clinical Trials Production Biosimilars

Article FDA Thank You What’s on FDA’s new Diagnostics Chief’s mind? Transitions, the LDT rule and cybersecurity

Agency IQ

AUGUST 23, 2024

This project, which is focused first on CDx (which are largely Class III products) and IVDs for infectious diseases, was also seen as a key aspect of implementing the LDT rule. Per the LDT final rule, the agency intends to have the reclassification initiative completed by Stages 4-5, which are the pre-market submissions stages of the rule.

FDA

FDA Laboratories Regulations Clinical Trials

Article FDA Thank You Diagnostics landscape analysis: Key issues and a look ahead at 2024

Agency IQ

FEBRUARY 2, 2024

BY LAURA DIANGELO, MPH | JAN 30, 2024 10:25 AM CST Diagnostic regulation: A quick overview Diagnostics products are those intended to identify, signal or detect a specific health condition, infection or disease, or monitor a person’s health or health status. Featuring previous research and expert analysis by Corey Jaseph.

FDA

FDA Laboratories Regulations Production

Analysis Life Sciences Thank You A deep dive into MDR and IVDR implementation: Where we are and where we’re going

Agency IQ

AUGUST 18, 2023

The remaining seven categories should be covered by the eight labs – hepatitis and retrovirus, herpesviruses, arboviruses, respiratory viruses that cause life-threatening diseases, parasites, and blood grouping tests. It’s not clear what happened to the original 30 laboratories that the Member States identified as candidates.

Science

Science Regulations Laboratories Production

Eli Lilly Designs P-Tau Biomarker Assay for Early Alzheimer’s Disease Diagnosis

The Pharma Data

NOVEMBER 10, 2020

Biomarkers will be critical to developing precision medicine for Alzheimer’s disease. Although a few are out there, phosphorylated tau (P-tau) may enable even earlier and more accurate detection, according to Eli Lilly-associated researchers speaking at the Clinical Trials on Alzheimer’s Disease (CTAD) virtual conference in early November.

Disease

Disease Clinical Trials Trials Research

Phases of Clinical Research: A Detailed Overview

Advarra

JUNE 27, 2023

Currently incurable diseases would remain so, without any improvements made to existing drugs and therapies. It is the best way for scientists to fully observe and analyze how drugs interact with the human body , and their efficacy as treatments for disease. Clinical trial data is also important for disease prevention.

Clinical Research

Clinical Research Research Clinical Trials Trials

How Improving Diversity Can Benefit Clinical Trials

Alta Sciences

JULY 31, 2024

Why Diversity in Clinical Trials Matters A wide variety of factors, from age or biological sex to environmental conditions and lifestyle choices (e.g., substance abuse, sexual activity), can influence the risk and likelihood of developing a disease or other long-term health issues—as well as the way an individual responds to treatment.

Clinical Trials

Clinical Trials Trials Clinical Pharmacology Drug Development

The medical school trying to become anti-racist

The Pharma Data

AUGUST 16, 2020

A number of diseases manifest differently depending on skin tone, but too little attention is given to this in training, according to Dr Joseph Hartland, who is helping to lead changes at the University of Bristol Medical School. . Life-or-death signs.

Medical Schools

Medical Schools Doctors Drug Trials Government

Article EMA Thank You What we expect European regulators to do in April 2024

Agency IQ

MARCH 29, 2024

Andrews, Oxford, the British Medical Journal Medicine products pending European Commission decision following CHMP opinion Below are the drug or biologic products for which decisions are expected to occur within the next two months, according to AgencyIQ’s review of opinions from the EMA’s Committee for Medicinal Products for Human Use (CHMP).

Regulations

Regulations Production Clinical Trials Biosimilars

Women in Stem with Andrea Pfeifer

Drug Target Review

NOVEMBER 30, 2023

From an early age, I’ve been passionate about finding cures for difficult-to-treat diseases. I accepted and I’ve been CEO ever since; our goal, from the beginning, was to pioneer new ways to diagnose, treat and prevent neurodegenerative diseases. That’s missing the point – we have learned how to slow the disease.

Disease

Disease Therapies Laboratories Treatment

Everything Wrong with Mouse Studies (Kinda)

Codon

MARCH 14, 2023

They had “seen no evidence of [graft-versus-host disease] in any patient,” wrote surgeon Steven A. The FDA used to require animal tests before a human drug trial could proceed; they reversed that decision in January. When the paper was published, surgeons in the U.S. A third experiment found no effect.

DNA

DNA Laboratories Animal Testing Engineering

Deliberate Dysentery

Codon

JANUARY 21, 2024

Today, refined versions of these human challenge studies have become standard practice in testing vaccines for vector-borne diseases (e.g., yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops. Why are challenge trials becoming more popular?

Vaccine

Vaccine Trials Disease Treatment

Recon: Sanofi, GKS report positive interim results for COVID shot; Regeneron resumes lymphoma trials

The Pharma Data

MAY 17, 2021

Regeneron resumes enrollment in lymphoma drug trials ( Reuters ). Incyte’s vitiligo treatment meets main goal in late-stage trial ( Reuters ). Bristol Myers backs up its case for heart drug mavacamten as FDA weighs app in cardiomyopathy ( Endpoints ). Now they fear an increase.

Trials

Trials Vaccine FDA Biosimilars

Perfluorhexyloctane

New Drug Approvals

MAY 20, 2025

It is an ophthalmic solution used to treat the signs and symptoms of dry eye disease. It has a role as an ophthalmology drug and a nonionic surfactant. Perfluorohexyloctane (branded as Evotears , Miebo , [a] and Novatears , among others) is a medication used for the treatment of dry eye disease. [4] Bausch + Lomb Corporation.

FDA Approval

FDA Approval FDA Disease Treatment

Durlobactam

New Drug Approvals

MAY 23, 2025

“Durlobactam, a Broad-Spectrum Serine -lactamase Inhibitor, Restores Sulbactam Activity Against Acinetobacter Species” Clinical Infectious Diseases. Durlobactam CAS 1467829-71-5 WeightAverage: 277.25 OCT-3-EN-6-YL SULFATE SULFURIC ACID, MONO((2S,5R)-2-(AMINOCARBONYL)-3-METHYL-7-OXO-1,6-DIAZABICYCLO(3.2.1)OCT-3-EN-6-YL)

FDA

FDA FDA Approval Hospitals Packaging

Omaveloxolone

New Drug Approvals

MAY 11, 2025

3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] 5] The condition can also cause heart disease. [5] 5] This disease tends to develop in children and teenagers and gradually worsens over time. [5] 3] [4] References ^ “Register of Innovative Drugs” Health Canada.

FDA

FDA FDA Approval Disease Production

Sulopenem

New Drug Approvals

APRIL 22, 2025

R*)))- FDA APPROVED sulopenem etzadroxil, probenecid, 10/25/2024, To treat uncomplicated urinary tract infections (uUTI) Drug Trial Snapshot Sulopenem ( CP-70,429 ) is a thiopenem antibiotic derivative from the penem family, which unlike most related drugs is orally active. 10 (4): 18151835. doi : 10.1007/s40121-021-00509-4.

FDA Approval

FDA Approval FDA Treatment Clinical Trials

Lotilaner

New Drug Approvals

MAY 30, 2025

1] [7] [8] [9] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [10] Jump up to: a b c “A Pill That Kills Ticks Is a Promising New Weapon Against Lyme Disease” Wired. Lotilaner CAS 1369852-71-0 Credelio XDEMVY lotilanerum 596.8 1] [7] It is used as an eye drop. [1] 26 July 2023.

FDA Approval

FDA Approval FDA Production International

Olezarsen

New Drug Approvals

APRIL 13, 2025

Olezarsen FDA APPROVED 12/19/2024, Tryngolza , To treat familial chylomicronemia syndrome Drug Trials Snapshot Synonyms AKCEA-APOCIII-LRX ALL-P-AMBO-5′-O-(((6-(5-((TRIS(3-(6-(2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYLOXY)HEXYLAMINO)-3-OXOPROPOXYMETHYL))METHYL)AMINO-5-OXOPENTANAMIDO)HEXYL))PHOSPHO)-2′-O-(2-METHOXYETHYL)-P-THIOADENYLYL-(3′-O->5′-O)-2′-O-(2-METHOXYETHYL)-P-THIOGUANYLYL-(3

FDA

FDA FDA Approval DNA Pharmaceuticals

Rezafungin

New Drug Approvals

MAY 14, 2025

“Salvage therapy of complicated Candida albicans spondylodiscitis with Rezafungin” European Journal of Clinical Microbiology & Infectious Diseases. 2] It is an echinocandin antifungal [1] [4] that acts as a fungal -glucan synthase inhibitor. [5] 2] [3] CAS No. doi : 10.1007/s10096-025-05117-5. PMID 40163284. April 2024).

Pharmacokinetics

Pharmacokinetics FDA FDA Approval Treatment

Probenecid

New Drug Approvals

APRIL 23, 2025

This use exploited probenecid’s interference with drug elimination (via urinary excretion) in the kidneys and allowed lower doses of penicillin to be used. [9] “Studies on the effect of probenecid (benemid) in gout” Annals of the Rheumatic Diseases. It is primarily used in treating gout and hyperuricemia. PMC 1030399.

Pharmacokinetics

Pharmacokinetics FDA Production Regulations

Trospium chloride

New Drug Approvals

APRIL 27, 2025

octane-8,1′-azolidin-1-ium]-3-yl 2-hydroxy-2,2-diphenylacetate;chloride FDA 2024, Cobenfy 9/26/2024, To treat schizophrenia Press Release Drug Trials Snapshot Trospium chloride is a muscarinic antagonist used to treat overactive bladder. [3] decane chloride 3-[(2-hydroxy-2,2-diphenylacetyl)oxy]-8lambda5-azaspiro[bicyclo[3.2.1]octane-8,1′-pyrrolidin]-8-yliumchloride

FDA Approval

FDA Approval FDA Pharmacokinetics Licensing

Acoramidis

New Drug Approvals

APRIL 21, 2025

1] [6] [10] ATTR-CM is a rare and serious disease that affects the heart muscle. [6] Jump up to: a b “Novel Drug Approvals for 2024” U.S. Food and Drug Administration (FDA). Retrieved 20 December 2024. ^ “FDA approves BridgeBio Pharma’s Attruby to treat rare heart disease ATTR-CM” PMLiVE.

FDA Approval

FDA Approval FDA Production Treatment

Drug Discovery Digest

Tofersen

Elacestrant

Trending Sources

Inavolisib

Pirtobrutinib

Zilucoplan

Leniolisib

ASH23: CAR-T for autoimmune disease, real-world questions and sickle cell’s social media mentions

AZVUDINE

What the FDA’s guidance on diversity means for rare disease sponsors

An Evolving Regulatory Environment for Rare and Orphan Diseases

Advanced 3D cell-based technologies

Levacetylleucine

Case study: NCI’s Molecular Targets Platform

Top Three Reasons Why Your Medical Device Needs a Clinical Trial

Article EMA Thank You What we expect European regulators to do in May 2024

Article FDA Thank You What’s on FDA’s new Diagnostics Chief’s mind? Transitions, the LDT rule and cybersecurity

Article FDA Thank You Diagnostics landscape analysis: Key issues and a look ahead at 2024

Analysis Life Sciences Thank You A deep dive into MDR and IVDR implementation: Where we are and where we’re going

Eli Lilly Designs P-Tau Biomarker Assay for Early Alzheimer’s Disease Diagnosis

Phases of Clinical Research: A Detailed Overview

How Improving Diversity Can Benefit Clinical Trials

The medical school trying to become anti-racist

Article EMA Thank You What we expect European regulators to do in April 2024

Women in Stem with Andrea Pfeifer

Everything Wrong with Mouse Studies (Kinda)

Deliberate Dysentery

Recon: Sanofi, GKS report positive interim results for COVID shot; Regeneron resumes lymphoma trials

Perfluorhexyloctane

Durlobactam

Omaveloxolone

Sulopenem

Lotilaner

Olezarsen

Rezafungin

Probenecid

Trospium chloride

Acoramidis

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