The Pharma Data

JANUARY 20, 2021

Drug Industry Daily (DID) the premier online resource for savvy pharmaceutical professionals whose jobs depend on accurate knowledge about the daily activities of Congress, the FDA, other key regulators … and what their competitors are up to.

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The Pharma Data

OCTOBER 26, 2020

based aTyr Pharma has finalized enrollment for a phase 2 trial to evaluate an investigational anti-inflammatory drug in COVID-19 patients with severe respiratory complications. San Diego, Calif.-based ATYR1923 is meant to reduce inflammation and restore the lung’s immune balance.

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New Drug Approvals

MAY 17, 2025

Tofersen , sold under the brand name Qalsody , is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [3] 3] Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1 , an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis.

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New Drug Approvals

MAY 9, 2025

1] [4] It is taken by mouth. [1] 1] [4] Elacestrant is an antiestrogen that acts as an antagonist of estrogen receptors , which are the biological targets of endogenous estrogens like estradiol. [1] 2] Elacestrant was approved for medical use in the United States in January 2023, [1] [2] [5] [6] and in the European Union in September 2023. [3]

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New Drug Approvals

MAY 8, 2025

Jump up to: a b c “Drug Trials Snapshots: Jaypirca” U.S. Food and Drug Administration (FDA). 1] [2] [4] It inhibits B cell lymphocyte proliferation and survival by binding and inhibiting Bruton’s tyrosine kinase (BTK). [5] 5] It is taken by mouth. [1] 2] PATENTS Guisot, N. 12 (5): 80. PMC 9123190.

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The Pharma Data

NOVEMBER 30, 2020

Sponsors of drug trials should determine what effect the use of acid-reducing agents (ARA), such as antacids, proton pump inhibitors and histamine blockers, could have on the solubility of an orally administered drug, according to a new FDA draft guidance released Monday.

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New Drug Approvals

JUNE 15, 2025

6] [9] [10] It is a complement inhibitor that is injected subcutaneously (under the skin). [6] 6] Zilucoplan is a cyclic peptide that binds to the protein complement component 5 (C5) and inhibits its cleavage into C5a and C5b. [11] 1] Zilucoplan is a 15 amino-acid, synthetic macrocyclic peptide with formula C172H278N24O55.

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New Drug Approvals

MAY 15, 2025

5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] 2] [5] It is a kinase inhibitor [2] [6] that is taken by mouth. [2] 2] The most common side effects include headache , sinusitis , and atopic dermatitis. [5] 5] Leniolisib was approved for medical use in the United States in March 2023. [5]

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Drug Target Review

SEPTEMBER 19, 2023

Human In Silico Drug Trials Demonstrate Higher Accuracy than Animal Models in Predicting Clinical Pro-Arrhythmic Cardiotoxicity. Functional patient-derived organoid screenings identify MCLA-158 as a therapeutic EGFR × LGR5 bispecific antibody with efficacy in epithelial tumors. Nature Cancer. 2022;3(4):418-36. Frontiers in Physiology.

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Advarra

AUGUST 16, 2022

That is part of the responsibility of the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER). Department for Health and Human Services (DHHS), prepared guidance on expedited programs for sponsors of orphan drug trials. These centers, in conjunction with the FDA and U.S.

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Conversations in Drug Development Trends

NOVEMBER 3, 2022

Looking to diversify the reach of your orphan drug trial? Reach out to learn how Worldwide Clinical Trials can help. The post What the FDA’s guidance on diversity means for rare disease sponsors appeared first on Worldwide Clinical Trials. Originally published in BioPharma Dive on October 31st, 2022.

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Advarra

MARCH 21, 2023

These products will require clinical trial data to get to market, but with significantly fewer participants required as compared to a drug trial. Class 3: Applied to products with the highest risk, or to Class 2 products that are the first in their category. Think of the intent or purpose for the intended use (e.g.,

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Clinical Trials Trials Production Regulations 52

Drug Target Review

OCTOBER 3, 2024

FDA no longer needs to require animal tests before human drug trials [Internet]. 1975 Aug;256(5517):495–7. Crescioli S, Kaplon H, Chenoweth A, et al. Antibodies to watch in 2024. 2024 Jan 5;16(1). www.science.org. Available from: [link] ‌Baker M. Reproducibility crisis: Blame it on the antibodies. 2015 May;521(7552):274–6.

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Molecular Biology Pharmacokinetics Laboratories Animal Testing 52

The Open Targets Blog

OCTOBER 31, 2023

[ Deanne Taylor , lead Principal Investigator of MTP] In the US, formal clinical trials are required to establish the safety and utility of those treatments in pediatric cancers. Despite the evidence that pediatric cancers could benefit from the same drugs used to treat adult cancers, drug trials are much rarer in children than adults. To

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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]

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PPD

MARCH 28, 2024

Furthermore, partnering with a CRO can bend the cost and time curve of drug trials, a critical factor for investors seeking returns on their capital. This assurance reduces executional risk and provides investors with a clear path to understanding how their investment will be used to test hypotheses efficiently.

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Agency IQ

JULY 22, 2024

Because this also includes device studies that involve drug products , for instance when diagnostics are part of a drug trial, this has complicated development of what was already a complex and ambitious EUDAMED module.

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New Drug Approvals

APRIL 30, 2025

1] [2] Levacetylleucine is a modified version of the amino acid leucine. [1] 1] It is the L -form of acetylleucine.

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Agency IQ

AUGUST 23, 2024

The reclassification initiative: In February 2024, CDRH announced a new initiative to review the risk-based classifications of “most” IVDs that are regulated as “high risk” (Class III) devices to see if they could be appropriately regulated in a lower risk-based category (Class II, moderate risk).

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Agency IQ

MAY 3, 2024

.: Hands-on training course Webinar/ Seminar ( OPEN ) EMA 5/14/2024 5/14/2024 From paper to digital leaflets: making patient information more inclusive Webinar/ Seminar ( OPEN ) Association of the European Self-Care Industry (AESGP) 5/14/2024 5/14/2024 Changes in the area of DiGA due to the Digital Act Webinar/ Seminar ( OPEN Germany’s Federal Institute (..)

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Agency IQ

FEBRUARY 2, 2024

This remains a major area of concern for both industry and regulators, with a March 2023 survey from EFPIA finding that the challenges with the IVDR has led to issues with IVDs used in drug trials , and particularly cancer trials. Featuring previous research and expert analysis by Corey Jaseph.

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Agency IQ

AUGUST 18, 2023

We’ve seen what’s been going on with the MDR, but the main concern we’ve heard of IVDR is the performance study application process is affecting drug trials. What is going to happen with the IVDR? A big question is how many of the IVDs that used to be self-certified (most of them) plan to remain on the market?

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The Pharma Data

DECEMBER 8, 2020

The FDA has issued a clinical hold on Bellicum Pharmaceuticals for its ongoing phase 1/2 trial evaluating CAR-T therapy BPX-601 in patients who received prior treatment for metastatic pancreatic or prostate cancer, following the death of a trial participant. The Houston, Tex.,

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The Pharma Data

NOVEMBER 10, 2020

This is encouraging as a pharmacodynamic biomarker in larger trials,” Dage said. The assay is being explored for applications in clinical research, including new drug trials, based on its capability to identify patients with Alzheimer’s disease and also to predict their risk of progression.

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The Pharma Data

AUGUST 16, 2020

Exploring the fact that drug trials predominantly involve white, male participants, and looking at how this may lead to inaccurate and harmful prescribing for other patients. Teaching on the history of medicine, including the exploitation of people of colour in scientific research.

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Alta Sciences

JULY 31, 2024

Public Health Service Syphilis Study at Tuskegee, conducted between 1932 and 1972), as well as restrictions on equitable access to clinical services that continue to the present day. A 2022 study found that sharing results from a hypertension drug trial with 15% Black participants increased Black patients' confidence in the drug's effectiveness (..)

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Clinical Trials Trials Clinical Pharmacology Drug Development 52

Advarra

JUNE 27, 2023

Some Phase I trials are also first-in-human (FIH) studies, meaning the treatment has not previously been tested in people.

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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).

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Drug Target Review

NOVEMBER 30, 2023

We are the only company in the world including this population in any Alzheimer’s drug trial, and we are very proud of this! Too few people know that Alzheimer’s-like characteristics develop in more than 98 percent of people with Down syndrome over age 40. COVID-19 has shown the revolutionary impact of vaccines.

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Codon

MARCH 14, 2023

The FDA used to require animal tests before a human drug trial could proceed; they reversed that decision in January. .” When they repeated the experiment on identical mice, of the same strain and from the same vendor, those mice gained bone density. A third experiment found no effect. Each animal had a different microbiome.

DNA 52

DNA Laboratories Animal Testing Engineering 52

New Drug Approvals

MAY 23, 2025

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)

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New Drug Approvals

MAY 20, 2025

WeightAverage: 432.269 Monoisotopic: 432.112266666Chemical FormulaC 14 H 17 F 13 Perfluorhexyloctane 133331-77-8 MIEBO Tetradecane, 1,1,1,2,2,3,3,4,4,5,5,6,6-tridecafluoro- 7VYX4ELWQM NOV03 , NOV 03 1-(perfluorohexyl)octane F6H8 NOV03 Perfluorohexyloctane 1,1,1,2,2,3,3,4,4,5,5,6,6-tridecafluorotetradecane FDA APPROVED 8/16/2023, Sohonos , To reduce (..)

FDA Approval 62

FDA Approval FDA Disease Treatment 62

New Drug Approvals

MAY 26, 2025

Flotufolastat F 18 Flotufolastat F-18 Gallium POSLUMA CAS 2305081-64-3 18FrhPSMA-7.3 18F-rhPSMA-7.3 1] The active ingredient is flotufolastat ( 18 F). [1] 1] Flotufolastat ( 18 F) was approved for medical use in the United States in May 2023. [1] 1] [2] SYNTHESIS Bejot, R.,

Clinical Trials 62

Clinical Trials FDA FDA Approval Trials 62

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.

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FDA Approval FDA Treatment Clinical Trials 62

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

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New Drug Approvals

MAY 14, 2025

9,13 ]heptacosan-21-yl]oxy]ethyl-trimethylazanium Rezafungin ion Rezafungin cation CD-101 SP-3025 G013B5478J Rezafungin , sold under the brand name Rezzayo (by Melinta Therapeutics), is a medication used for the treatment of invasive candidiasis. [2] 2] It is an echinocandin antifungal [1] [4] that acts as a fungal -glucan synthase inhibitor. [5]

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Pharmacokinetics FDA FDA Approval Treatment 62

Codon

JANUARY 21, 2024

In some cases, I’d argue that a malaria challenge trial is safer than a typical oncology phase 1 drug trial, where you're taking people who don't have cancer and testing the safety of a drug that has maybe been tested in mice but has never been used in humans. What is your pitch for them?

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