New Drug Approvals

MAY 15, 2025

5] [7] [8] It is the first approved medication for the treatment of activated PI3K delta syndrome. [5] 5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] 5] [7] [8] It is the first approved medication for the treatment of activated PI3K delta syndrome. [5] hdl : 10665/330984.

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New Drug Approvals

APRIL 25, 2025

12] However, absorption, metabolism, and excretion data of taselisib , a molecule with a related chemical scaffold, suggest moderately slow absorption into the systemic circulation, metabolism to play a minor role in drug clearance, and biliary excretion to be the main route of excretion. [13] Hz, 1H), 6.41 (dd, J=8.8, Hz, 1H), 5.02-4.89

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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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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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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] 2] The duration was twelve weeks for each treatment period. [2] 2] Participants could receive miglustat , an enzyme inhibitor , as background treatment in the study. [2]

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Conversations in Drug Development Trends

NOVEMBER 3, 2022

So while Ansel said he’s seen many sponsors of orphan drugs successfully diversify U.S. Expanding abroad could help sponsors recruit more ethnicities within certain populations while also increasing treatment access to more people. Looking to diversify the reach of your orphan drug trial? enrollment by diversifying U.S.

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PPD

MARCH 28, 2024

Additionally, biomarkers identify patients who are most likely to benefit from a particular treatment, enabling companies to tailor their trials to those most likely to respond positively. By leveraging digital tools and platforms, sponsors often reach a broader range of patients, including those in underserved communities.

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Agency IQ

MAY 3, 2024

and 2 mg [EMA] Product-specific bioequivalence guidance 6/30/2024 Pharmeuropa 36.2 and 2 mg [EMA] Product-specific bioequivalence guidance 6/30/2024 Pharmeuropa 36.2

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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. As Takeshi Iwatsubo, M.D.

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Alta Sciences

JULY 31, 2024

Similarly, Health Canada's guidance highlights the need to represent various demographic groups in clinical trials, ensuring that study results are broadly applicable for enhanced safety and efficacy of drug development. For example, elderly individuals are more prone to comorbidities, dementia, and functional decline.

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Advarra

JUNE 27, 2023

Most modern medicines were developed from clinical trials , and clinical trials remain essential for drug development. Without clinical studies, our global understanding of medicine would likely stagnate, and novel treatments would cease to emerge.

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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. ” Experiments without consent.

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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! The recent positive clinical data from two new Alzheimer’s disease treatments – lecanemab from Eisai and Biogen and donanemab from Eli Lilly – bring real hope to patients and families.

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New Drug Approvals

MAY 20, 2025

Perfluorohexyloctane (branded as Evotears , Miebo , [a] and Novatears , among others) is a medication used for the treatment of dry eye disease. [4] 4] [6] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [7] New Drug Therapy Approvals 2023 (PDF). . 4] It is a semifluorinated alkane. [4]

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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. 53 (5): 21632170. doi : 10.1128/AAC.01557-08.

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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] [3] CAS No. 2] [3] CAS No.

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New Drug Approvals

MAY 23, 2025

REF [link] References ^ Jump up to: a b “FDA Approves New Treatment for Pneumonia Caused by Certain Difficult-to-Treat Bacteria” U.S. Food and Drug Administration (Press release). Durlobactam CAS 1467829-71-5 WeightAverage: 277.25 24 May 2023. Retrieved 24 May 2023. 12 : 709974. doi : 10.3389/fmicb.2021.709974. 2021.709974.

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Codon

JANUARY 21, 2024

yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops. Scientists studied black men infected with syphilis for several decades while withholding known treatments. Now, we have upwards of 98 or 99 percent cure rates in some cases.

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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 30, 2025

1] [7] [8] [9] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [10] Food and Drug Administration (FDA). Retrieved 5 August 2023. ^ “FDA Approves Xdemvy (lotilaner ophthalmic solution) 0.25% for the treatment of Demodex blepharitis” (Press release). 26 July 2023. 25 July 2023.

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New Drug Approvals

MAY 11, 2025

3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] 3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] Medical uses Omaveloxolone is indicated for the treatment of Friedreich’s ataxia. [2] Food and Drug Administration (FDA).

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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] “Trospium chloride treatment of overactive bladder” Ann Pharmacother.

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New Drug Approvals

MAY 22, 2025

This drug is given in combination with -lactam antibiotics to inhibit -lactamase , an enzyme produced by bacteria that destroys the antibiotics. [1] Retrieved 25 May 2023. ^ “Sulperazon” drugs.com. ^ “FDA Approves New Treatment for Pneumonia Caused by Certain Difficult-to-Treat Bacteria” U.S. 29 March 2023.

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New Drug Approvals

APRIL 23, 2025

It is primarily used in treating gout and hyperuricemia. Experimental Properties Property Value Source melting point (C) 195 C PhysProp water solubility 27.1 mg/L Not Available logP 3.21 HANSCH,C ET AL. 1995) pKa 3.4

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New Drug Approvals

APRIL 21, 2025

Medical uses Acoramidis is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. [1] Acoramidis AG-10 1446711-81-4 AG10 Acorami 292.30 g, 83.2%) as an off-white solid. 26 November 2024.

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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 ). Desperate for treatment, Covid ‘long haulers’ push for 9/11-style health registry ( Politico ). BioPharmaDive ).

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