Drug Trials and FDA Approval - Drug Discovery Digest

Drug Trials

FDA Approval

Tofersen

New Drug Approvals

MAY 17, 2025

Jump up to: a b c d e f g h i j k l “FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene” (Press release). Food and Drug Administration (FDA). “Biogen’s ALS Drug Gets Partial Backing From FDA Panel” Bloomberg News. 25 April 2023.

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Inavolisib

New Drug Approvals

APRIL 25, 2025

3] Inavolisib was approved for medical use in the United States in October 2024. [3] 19] Society and culture Legal status In October 2024, the US Food and Drug Administration (FDA) approved inavolisib for the treatment of PIK3CA -mutant breast cancer based on the results from the INAVO120 trial. [3] cd-21-0072.

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Vamorolone

New Drug Approvals

JULY 25, 2025

Syn EuropeanJournalofMedicinalChemistry265(2024)116124 Vamorolone (Agamree) On October 26, 2023, Vamorolone, developed jointly by Santhera Pharmaceuticals and ReveraGen BioPharma, has received FDA approval to treat DMD in patients aged 2 years and older [1]. “Drug Trials Snapshots: Agamree” U.S. PMC 6371196.

FDA

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Elacestrant

New Drug Approvals

MAY 9, 2025

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] 2] The FDA granted the application for elacestrant priority review and fast track designations. [2] Food and Drug Administration (FDA). 1] [4] It is taken by mouth. [1]

FDA

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Pirtobrutinib

New Drug Approvals

MAY 8, 2025

7] Pirtobrutinib was approved for medical use in the United States in January 2023, [4] [8] [9] [10] and in the European Union in November 2023. [2] Jump up to: a b c “Drug Trials Snapshots: Jaypirca” U.S. Food and Drug Administration (FDA). Food and Drug Administration (FDA). 12 (5): 80.

FDA Approval

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Leniolisib

New Drug Approvals

MAY 15, 2025

5] Leniolisib was approved for medical use in the United States in March 2023. [5] 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] hdl : 10665/330984. 24 March 2023.

FDA Approval

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An Evolving Regulatory Environment for Rare and Orphan Diseases

Advarra

AUGUST 16, 2022

Orphan drugs have historically faced a number of barriers, such as limited research and development (R&D) investment due to an expected lack of profitability as well as challenges in clinical trial design and recruitment. Before 1983, only 38 orphan drugs had received U.S. Food and Drug Administration (FDA) approval.

Disease

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Levacetylleucine

New Drug Approvals

APRIL 30, 2025

Jump up to: a b c d e f g h i j k l m n o “FDA Approves New Drug to Treat Niemann-Pick Disease, Type C” U.S. Food and Drug Administration (Press release). FDA Approval of Aqneursa for the Treatment of Niemann-Pick Disease Type C” IntraBio (Press release). 24 September 2024. N Engl J Med.

FDA

FDA FDA Approval Disease Trials

Advanced 3D cell-based technologies

Drug Target Review

SEPTEMBER 19, 2023

In spite of current success and possibility to be a successful cell technology model, the integration of organ-on-chips into drug development process needs more optimisation to be validated for FDA approval. Nature Cancer. 2022;3(4):418-36. Passini E, Britton OJ, Lu HR, Rohrbacher J, Hermans AN, Gallacher DJ, et al.

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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. PMC 8572892.

FDA Approval

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Perfluorhexyloctane

New Drug Approvals

MAY 20, 2025

Retrieved 15 February 2024. ^ “Bausch + Lomb and Novaliq Announce FDA Approval of Miebo (Perfluorohexyloctane Ophthalmic Solution) for the Treatment of the Signs and Symptoms of Dry Eye Disease” (Press release). Bausch + Lomb Corporation. 18 May 2023.

FDA Approval

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Lotilaner

New Drug Approvals

MAY 30, 2025

1] It was approved for medical use in the United States in July 2023. [1] 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. ^ “Xdemvy: FDA-Approved Drugs” U.S.

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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

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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] 13 August 2007. ^ “ANDA 091289 approval letter” (PDF).

FDA Approval

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Rezafungin

New Drug Approvals

MAY 14, 2025

5] Rezafungin was approved for medical use in the United States in March 2023, [1] [6] [5] and in the European Union in December 2023. [2] 2] It is an echinocandin antifungal [1] [4] that acts as a fungal -glucan synthase inhibitor. [5] 2] [3] CAS No. Antimicrob Agents Chemother. 2022 Jan 18;66(1):e0139021. doi: 10.1128/AAC.01390-21.

Pharmacokinetics

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Durlobactam

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.

FDA

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Flotufolastat F 18

New Drug Approvals

MAY 26, 2025

1] Flotufolastat ( 18 F) was approved for medical use in the United States in May 2023. [1] FDA Approves Blue Earth Diagnostics’ Posluma (Flotufolastat F 18) Injection, First Radiohybrid PSMA-targeted PET Imaging Agent for Prostate Cancer” (Press release). 18F-rhPSMA-7.3 1] [2] SYNTHESIS Bejot, R., 2 June 2023.

Clinical Trials

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Omaveloxolone

New Drug Approvals

MAY 11, 2025

5] Omaveloxolone was approved for medical use in the United States in February 2023, [2] [5] [6] [7] [8] and in the European Union in February 2024. [3] 3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] Food and Drug Administration (FDA). 2] [5] It is taken by mouth.

FDA

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Building better drugs: how 3D models are shaping pre-clinical development

Drug Target Review

APRIL 25, 2025

Addressing the gaps associated with liver-on-chips, a recent 3D hepatic spheroid study predicted DILI liabilities of 152 FDA-approved small molecules using a high-throughput 384 well plate-based system with a sensitivity of 72 percent and specificity of 89 percent. link] [19] Safarzadeh M, Richardson LS, Kammala AK, et al.

Clinical Development

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Sulbactam

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] 1] It was patented in 1977 and approved for medical use in 1986. [2] Food and Drug Administration (Press release). National Library of Medicine. 29 March 2023. 24 May 2023.

Pharmacokinetics

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Probenecid

New Drug Approvals

APRIL 23, 2025

Probenecid 57-66-9 4-(Dipropylsulfamoyl)benzoic acid Probenecid acid Benemid 4-(dipropylsulfamoyl)benzoic acid C 13 H 19 NO 4 S, 285.359 HC 5006 NSC-18786 FDA APPROVED, 10/25/2024, sulopenem etzadroxil, probenecid, Orlynvah , To treat uncomplicated urinary tract infections (uUTI) Drug Trial Snapshot Probenecid , also sold under the brand name Probalan (..)

Pharmacokinetics

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Acoramidis

New Drug Approvals

APRIL 21, 2025

Jump up to: a b c d e f g h i j k “FDA approves drug for heart disorder caused by transthyretin-mediated” U.S. Food and Drug Administration. Jump up to: a b “Novel Drug Approvals for 2024” U.S. Jump up to: a b “Novel Drug Approvals for 2024” U.S. PMC 6276790.

FDA Approval

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Tofersen

Inavolisib

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Vamorolone

Elacestrant

Pirtobrutinib

Leniolisib

An Evolving Regulatory Environment for Rare and Orphan Diseases

Levacetylleucine

Advanced 3D cell-based technologies

Sulopenem

Perfluorhexyloctane

Lotilaner

Olezarsen

Trospium chloride

Rezafungin

Durlobactam

Flotufolastat F 18

Omaveloxolone

Building better drugs: how 3D models are shaping pre-clinical development

Sulbactam

Probenecid

Acoramidis

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