New Drug Approvals

MAY 17, 2025

Liu A (1 May 2019). “Biogen’s antisense ALS drug shows promise in early clinical trial” FierceBiotech. Tofersen , sold under the brand name Qalsody , is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [3] Food and Drug Administration (FDA) (Report). January 2024. Retrieved 25 April 2023.

FDA Approval

DrugBank

JUNE 18, 2025

The failure rate in clinical trials exceeds 90%, often due to insufficient safety data, efficacy concerns, or regulatory non-compliance. Even drugs that complete clinical trials may face delays or rejections if submission documents are incomplete or do not align with regulatory expectations.

Drug Development

New Drug Approvals

APRIL 25, 2025

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] Han C, Kelly SM, Cravillion T, Savage SJ, Nguyen T, Gosselin F (2019). 20 May 2019. 3 November 2006. 2019.04.057.

FDA

New Drug Approvals

JUNE 11, 2025

5] It was submitted for the preregistration (NDA) phase again in May 2007 after adding additional information from clinical trials as the FDA required in 2009. Archived from the original (PDF) on 18 February 2019. July 2019). The FDA rejected approval for gepirone in 2002 and 2004. [5] 5] SYN Synthesis Ormaza, V. PMID 24262766.

FDA

Codon

JULY 28, 2025

That year, Terrence Flotte’s team at Johns Hopkins University used a natural strain of AAV, called AAV2, to introduce a cystic fibrosis transmembrane conductance regulator (CFTR) gene — defective in those with cystic fibrosis — into patients during a phase 1 clinical trial.

Immune Response

Drug Target Review

MARCH 3, 2025

This approach has the potential to revolutionise clinical trial design and lead to more effective, personalised treatments. In 2019, Jens co-founded OrganoTherapeutics SARL, where he currently serves as CEO. “Then, the organoid model would be tested with, say, five, six, or seven drugs available on the market.

Disease

The Pharma Data

JUNE 15, 2025

Effective immediately, Roche is halting the administration of Elevidys to non-ambulatory DMD patients in both commercial and clinical trial settings. Furthermore, outside of Europe, dosing will be paused immediately in the ENVISION trial, adding additional safeguards to protect trial participants.

Regulations

Codon

JUNE 5, 2025

” Four years earlier, in 2019, Gray had become the first patient with sickle cell anemia — a genetic disorder that causes red blood cells to become sticky and rigid — to receive an experimental treatment using CRISPR genome editing. Dozens more clinical trials, based upon similar gene-editing technologies, are now underway.

DNA

New Drug Approvals

JUNE 28, 2025

2] [5] [6] Clinical trials In September 2023, the drug’s developer, Sorrento Therapeutics, announced top-line data that olgotrelvir had met its primary endpoints in a phase III clinical trial that enrolled 1,212 patients with mild or moderate COVID-19. days on average compared to patients in the placebo group.

Clinical Trials

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. 24 September 2024. .

FDA

Broad Institute

FEBRUARY 26, 2025

So the researchers used a method developed at the Broad Institute in 2019 called consensus non-negative matrix factorization (cNMF) , which can define cells by their identity and activity independently. With this approach, the team identified four programs shaping the immune system.

Immune Response

New Drug Approvals

APRIL 18, 2025

2] History Crinecerfont’s approval is based on two randomized, double-blind, placebo-controlled trials in 182 adults and 103 children with classic congenital adrenal hyperplasia. [2] 2] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks. [2] 1 December 2024.

FDA

New Drug Approvals

APRIL 19, 2025

elimination half-life (min) cardio-selectivity (1/2) metabilization Landiolol 4 250 pseudocholinesterases Esmolol 9 30 ery-esterases Metoprolol 420 3 cytochrom P2D6 (Leber) History The beneficial effects of landiolol have been demonstrated in over sixty clinical trials (pubmed search -August 2018). IV -Blocker max. 42 (5): 373498. July 2017).

FDA

BioPharma Drive: Drug Pricing

JUNE 26, 2025

Incyte expects multiple pivotal trial readouts this year, along with proof-of-concept data for several pipeline candidates. Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy.

Therapies

New Drug Approvals

AUGUST 4, 2025

2019, 23, 341−350. Donafenib versus sorafenib in first-line treatment of unresectable or metastatic hepatocellular carcinoma: A randomized, open-label, parallel-controlled phase II-III trial. Donafenib , sold under the brand name Zepsun , is a pharmaceutical drug for the treatment of cancer. 227) Qin, S.; 2021, 39, 3002−3011.

Metabolic Stability

Conversations in Drug Development Trends

JANUARY 6, 2025

In contrast, an adaptive trial design allows for modifications to an ongoing trial and its analyses under a pre-specified framework, which is outlined in the FDAs Adaptive Designs for Clinical Trials for Drugs and Biologics Guidance for Industry, published in 2019.

Trials

Science Daily: Pharmacology News

MAY 17, 2024

An HIV vaccine candidate triggered low levels of an elusive type of broadly neutralizing HIV antibodies among a small group of people enrolled in a 2019 clinical trial.

Vaccine

BioPharma Drive: Drug Pricing

SEPTEMBER 26, 2024

The withdrawal comes as trial results indicate safety concerns with the drug, Oxbryta, which was first approved in the U.S.

Trials

New Drug Approvals

JULY 18, 2025

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)). 1] A phase II study (NCT04109183) was finished in March 2019 by Genuine Biotech. 16] A phase III trial was performed in 2022 in China.

RNA

ProRelix Research

JUNE 6, 2023

Being the second most populous country in the world with skilled labor and good infrastructure, India is a favorable destination for clinical trials for a wide variety of diseases.

Clinical Trials

BioPharma Drive: Drug Pricing

AUGUST 28, 2023

The treatment, developed by a biotech Bayer acquired in 2019, appeared safe in a small trial and showed signs of an effect on disease progression.

Therapies

Creative Biolabs

JANUARY 15, 2020

Drug Name Trade Name Company Approval Date 1 PrabotulinmtoxinA Jeuveau Evolus 2019/2/1 2 Caplacizumab-yhdp Cablivi Ablynx 2019/2/6 3 Triclabendazole Egaten Novartis 2019/2/13 4 Brexanolone Zulress Sage Therapeutics 2019/3/19 5 Solriamfetol Sunosi Jazz Pharmaceuticals 2019/3/20 6 Siponimod Mayzent Novartis 2019/3/26 7 Romosozumab-aqqg Evenity Amgen (..)

Clinical Trials

Conversations in Drug Development Trends

AUGUST 23, 2023

Patients are the backbone of clinical trials, playing an essential role in the drug development process. This engagement is often less understood and is underutilized by sponsors, meaning a significant element of the trial and drug experience is missed during sponsor engagement with the FDA.

FDA

Antidote

JULY 29, 2022

In 2019 alone, research and development spending totaled $186 billion for the global pharmaceutical industry. Between 2009 and 2018, U.S. biopharmaceutical companies spent about $1 billion per drug according to an analysis published in JAMA , and other studies have found that it can cost up to $2.8 billion to bring a new therapy to market.

Clinical Trials

SCIENMAG: Medicine & Health

JULY 14, 2023

billion in project funding for phased clinical trials involving drugs approved by the FDA from 2010-2019, according to a new study from Bentley University’s Center for Integration of Science and Industry. BENTLEY UNIVERSITY Credit: Bentley University BENTLEY UNIVERSITY The U.S. National Institutes of Health (NIH) contributed $8.1

Clinical Trials

The Pharma Data

MAY 27, 2022

billion in Medicare funds in 2019 on drugs whose clinical benefits have yet to be confirmed by the Food and Drug Administration, a new study led by researchers from the Johns Hopkins Bloomberg School of Public Health suggests. billion on 36 of these drugs across 55 indications in 2019. federal government spent an estimated $1.8

Drugs

The Pharma Data

AUGUST 14, 2020

Clinical trial programme resumes after safety concerns. Novo Nordisk is set to resume the phase 3 clinical trials of concizumab in haemophilia A and B, with or without inhibitors, following a previous safety scare. The trials are expect to enrol approximately 293 patients from 32 countries.

Trials

The Pharma Data

NOVEMBER 9, 2020

Almost two-thirds of participants in clinical trials to support U.S. drug approvals from 2015 to 2019 were outside the U.S., the FDA’s Center for Drug Evaluation and Research (CDER) said in a new report unveiled yesterday. Source link.

Clinical Trials

The Pharma Data

NOVEMBER 3, 2020

The agreement will fund Clover’s coronavirus spike protein-targeting vaccine candidate, SCB-2019, through licensing in China and distribution by COVAX, the World Health Organization-backed initiative aimed at supplying 2 billion doses of COVID-19 vaccines globally by the end of 2021 that CEPI is running. Earlier this year, CEPI provided $3.5

Vaccine

Drug Target Review

NOVEMBER 1, 2023

2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. Andrew McCarthy Andrew is a Team Leader at EMBL Grenoble since 2007.

Clinical Trials

FDA Law Blog: Biosimilars

JANUARY 15, 2024

FDA published a June 2019 guidance , which stated that manufacturers would not need to perform long-term studies or submit long-term data in PMTAs. FDA said randomized clinical trials could be used, but so could observational studies with respect to cessation data. FDA also directed manufacturers to produce detailed marketing plans.

FDA

The Pharma Data

DECEMBER 5, 2020

.–(BUSINESS WIRE) December 5, 2020 — Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, today announced new clinical data from its Phase 2 trial evaluating SY-1425, its first-in-class selective retinoic acid receptor alpha (RAR?)

Clinical Trials

The Pharma Data

AUGUST 28, 2020

The Phase 1 trial will be conducted by MindMed later this year and will be the first to investigate what happens when you mix the two drugs to work as a therapy. Coaster420. Researchers are set to investigate a combined dose of LSD and MDMA to treat patients undergoing psychotherapy.

Clinical Trials

The Pharma Data

APRIL 3, 2021

The CoVIg-19 Plasma Alliance today announced that the Phase 3 Inpatient Treatment with Anti-Coronavirus Immunoglobulin (ITAC) clinical trial sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), did not meet its endpoints. About the ITAC Trial.

Clinical Trials

The Pharma Data

SEPTEMBER 8, 2020

Phase III clinical trial programme initiated for investigational medicine fenebrutinib, designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS).

Clinical Trials

Drug Target Review

NOVEMBER 17, 2023

Up until 2023 around 600 NK cell clinical trials have been reported and some 200 are clinically active and still recruiting patients. The vast majority (65/74; 88%) of those trials involved NK cells without CARs; while only 12 percent were CAR-NK studies. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients.

Therapies

The Pharma Data

AUGUST 7, 2020

The governing body claims this proposal could have positive effects on mitigating burdensome requirements for gene therapy clinical trials in the future. In this light, Pharma IQ’s weekly round-up focuses on advancing therapies and clinical trials to combat Covid-19. Dr. Francis S.

Clinical Trials