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]

FDA Approval

BioPharma Drive: Drug Pricing

DECEMBER 4, 2024

Results from the head-to-head study showed treatment with Zepbound resulted in 47% greater relative weight loss than Wegovy, a finding that could advantage Lilly in the companies’ market competition.

Drug Trials

BioPharma Drive: Drug Pricing

FEBRUARY 15, 2024

Analysts pressed Alnylam executives on the reasons for the changes to HELIOS-B, a Phase 3 study in ATTR amyloidosis with cardiomyopathy that is important to the company’s future.

Drug Trials

BioPharma Drive: Drug Pricing

DECEMBER 4, 2023

The biotech said Phase 2 study results showed its experimental medicine for age-related macular degeneration was "non-inferior" to Regeneron's Eylea.

Drug Trials

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

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

Drugs.com

SEPTEMBER 22, 2023

22, 2023 – New research suggests some newer diabetes treatments may not be as beneficial for Black patients, after earlier drug trials included small numbers of non-white people. FRIDAY, Sept. Whether the medications -- called sodium-glucose.

Drug Trials

BioPharma Drive: Drug Pricing

JULY 17, 2023

The results come about two years after negative findings from an earlier phase of the study, and position the company to seek an approval in transthyretin amyloidosis cardiomyopathy.

Drug Trials

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. The double-blind, placebo-controlled trial, which has 32 participants across the U.S., San Diego, Calif.-based Source link.

Trials

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.

Drug Trials

Advarra

MARCH 21, 2023

While drugs require Phase I-III clinical trials—and are also subject to post-approval tracking—digital therapeutics, devices, and IVDs may be able to leverage bench testing, animal studies, pilot studies, and training sets. The greater the risk, the more likely a series of trials for market clearance or approval will be required.

Clinical Trials

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.

Drug Trials

Conversations in Drug Development Trends

NOVEMBER 3, 2022

In April 2022, the Food and Drug Administration (FDA) issued draft guidance on developing a “Race and Ethnicity Diversity Plan” for clinical trials. In turn, the FDA’s guidance recognized the difficulty in recruiting for rare diseases and encouraged sponsors to become more resourceful and intentional about trial enrollment.

Disease

Drug Target Review

SEPTEMBER 19, 2023

puts an end to the previous mandate that all drugs need to be tested on animals prior to human clinical trials. This major shift to the orthodox tradition of using animal experiments in drug testing dates back the Aristotle’s time and cemented 80 years ago with initial federal mandate of drug safety regulation of 1938.

Animal Testing

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

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

Bioinformatics

PPD

MARCH 28, 2024

One of the fundamental pillars of this efficiency is a well-thought-out trial design. By carefully planning the trial structure, including the selection of endpoints and the overall methodology, companies reduce the likelihood of costly delays or the need for additional trial arms.

Trials

New Drug Approvals

APRIL 30, 2025

“A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia” Trials. A clinical trial to test amlodipine as a new treatment for vascular dementia. 1] It is the L -form of acetylleucine. 22 (1): 84.

FDA

Agency IQ

AUGUST 23, 2024

At a high level, the pilot program was intended to offer sponsors of certain drug products that would need a CDx with the opportunity to submit information about their Clinical Trial Assay (CTA) – the assay they’re using in a pivotal trial – to the FDA [see here for background on that topic].

FDA

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.

Molecular Biology

Agency IQ

MAY 3, 2024

Two consultations on clinical trial considerations, one on advanced therapies and the other addressing non-inferiority and equivalence comparisons, are closing at the end of May. The pharma reform package remains in E.U. leaders’ eyesight as the Parliament winds down ahead of its June elections.

Regulations

Agency IQ

JULY 22, 2024

Ultimately, this module will interact with CTIS , the drug clinical trial information system. 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.

Regulations

Agency IQ

FEBRUARY 2, 2024

Companion diagnostics (CDx) and cancer trials: In 2023, the MDCG published guidance documents on classifying CDx. However, there are still outstanding concerns that the challenges in standing up the IVDR system could lead to delays in drug approval. No IVDs achieved certification in under one year and 10% took longer than two years.

FDA

Agency IQ

AUGUST 18, 2023

The subgroup continues to work through technical aspects of certificates, clinical trial and performance study applications, vigilance and trending reports and market surveillance. 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.

Science

Alta Sciences

JULY 31, 2024

How Improving Diversity Can Benefit Clinical Trials pmjackson Wed, 07/31/2024 - 19:19 In July 2024, the U.S. Food and Drug Administration (FDA) published a draft guidance to ensure greater diversity in clinical trials, which is expected to become a final guidance by June 2025.

Clinical Trials

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., The Houston, Tex., Source link.

Trials

The Pharma Data

NOVEMBER 10, 2020

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. This is encouraging as a pharmacodynamic biomarker in larger trials,” Dage said.

Disease

Advarra

JUNE 27, 2023

Clinical trials are studies involving human volunteers to research ways to prevent, detect, and treat various medical conditions. The goal is to identify safer, more effective methods of treating illnesses using new drugs or new combinations of existing drugs.

Clinical Research

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.

Medical Schools

Agency IQ

MARCH 29, 2024

regulatory framework Webinar/ Seminar ( OPEN ) EMA 4/17/2024 4/17/2024 CEP 2.0:

Regulations

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.

Disease

Codon

MARCH 14, 2023

The ALS Therapy Development Institute, located just ten minutes from my home in Cambridge, once tested more than 100 potential drugs to slow ALS progression in mice, all of which had been reported to be helpful in other studies. ” A recent article in The New Yorker suggests a solution to the problem. Free-range mice? .”

DNA

Codon

JANUARY 21, 2024

Human challenge trials were an indispensable part of the development of the malaria vaccine, R21/Matrix-M, endorsed by the World Health Organization last October. But for all of their benefits, human challenge trials have their drawbacks. Jake himself has participated in both Zika and Shigella challenge trials.

Vaccine

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 ). Indian makers of Sputnik V may seek waiver for local Phase-3 trials ( Economic Times ). . Trump tax law cut what US drugmakers owed.

Trials

New Drug Approvals

MAY 26, 2025

External links Clinical trial number NCT04186819 for “Imaging Study to Investigate the Safety and Diagnostic Performance of rhPSMA 7.3 (18F) 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] [2] SYNTHESIS Bejot, R., PMID 37439946.

Clinical Trials

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)

FDA

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.

FDA Approval