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A new publication from the FDA Oncology Center of Excellence (OCE) and the ClinicalTrials Transformation Initiative (CTTI), published in the Clinical Cancer Research journal, provides important insights into how decentralized clinicaltrial (DCT) elements were used in cancer trials leading to FDAapproval during the COVID-19 pandemic.
The agency’s approval comes months after a large clinicaltrial showed the drug, called Leqembi, could slow the disease’s progression. Yet experts have raised concerns about its safety.
The regulator cleared Saphnelo despite mixed clinicaltrial data, giving patients another option after the recent OK for a drug aimed at the autoimmune disease's severe side effects.
PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA also authorized the SmartFlow Neuro Cannula, a specialized infusion device by ClearPoint Neuro, Inc.,
In addition, Traumagel, an FDA-cleared plant-based hemostatic gel , is designed to rapidly control bleeding, demonstrating the variety of solutions emerging in trauma and wound care. Symvess does carry risks, including thrombosis, graft rupture and infection.
A group of independent experts wasn't convinced by clinicaltrial data from company Lykos Therapeutics, which is seeking FDAapproval of MDMA-assisted treatment for post-traumatic stress disorder.
The agency proposes that pharma move away from the common maximum tolerated dose (MTD) to trial designs that prioritise drug dosage optimisation at different stages. Since this approval, KRAS inhibitors have risen in popularity with the FDA also approving Mirati Therapeutics’ (NASDAQ:MRTX) inhibitor Krazati (adagrasib) as a therapy for NSCLC.
Based on objective response rate (ORR) and duration of response (DoR), the indication received approval under an accelerated pathway. The latest approval was based on the findings from the Phase II DESTINY-Lung02 clinicaltrial, which assessed the safety and efficacy of two doses of Enhertu in HER2 mutant metastatic NSCLC patients. .
SUMMARY: Over half of all clinicaltrial sites are outside the U.S. 87 % of all subjects in recent biologics trials were enrolled outside the U.S. DTC may or may not work for clinicaltrial enrollment it really depends on the condition and risk as judged by the patient. According to antidote.com.
Leqembi is indicated to treat mild cognitive impairment or mild dementia stage of the disease in patients whose treatment started in clinicaltrials. The regulatory approval is based on the data obtained from the Phase II trial.
Tzield was evaluated in the placebo-controlled, double-blind, randomised, event-driven TN-10 clinicaltrial, for delaying T1D (Stage 3, or clinical T1D) in Stage 2 T1D patients. Provention Bio stated that Tzield delayed the median onset of Stage 3 T1D by 25 months, or nearly two years, compared to a placebo in a clinicaltrial.
“With the approval of Kevzara for polymyalgia rheumatica, patients now have an FDA-approved treatment to help offer relief from the disabling symptoms of this disease and long-term dependence on steroids.”
Two near identical clinicaltrials of Aduhelm yielded conflicting results, dividing experts and even FDA staff. A third, confirmatory study is required, but Biogen has until 2029 to finish it.
The US Food and Drug Administration (FDA) on Friday approved a highly anticipated new drug designed to slow cognitive decline in patients in mild and early stages of Alzheimer’s disease.
Emrosi’s approval was based on data from a pair of Phase III clinicaltrials for the treatment of rosacea. The trials met all co-primary and secondary endpoints, with participants having successfully completed the 16-week treatment with no significant safety issues.
bluebird bio was granted a rare paediatric priority review voucher following approval. The latest development is based on findings from the single-arm, open-label Phase II/III ALD-102 (Starbeam) and Phase III ALD-104 clinicaltrials. 1) in Skysona-treated and untreated subjects. “For thalassemia.
The FDA indicates Blujepa specifically to treat uUTIs caused by E.coli and Staphylococcus saprophyticus. ClinicalTrial Results Blujepas approval was based on data from the Phase III EAGLE-2 and EAGLE-3 trials that enrolled over 3,000 patients.
The US Food and Drug Administration (FDA) has granted a combination of AstraZeneca and MSD ’s Lynparza (olaparib), with standard therapies for treating BRCA-mutated (BRCAm) metastatic castration-resistant prostate cancer (mCRPC). Safety and tolerability were in line with that observed in prior trials and the known profiles of the medicines.
With the necessary prioritization of various factors when designing your clinicaltrial, the patient experience can be overlooked or improperly addressed in study design. There are endpoints that can be easily measured, that are already standardized, and they are FDA-approved,” says Dr. Ojeda.
Syros Pharmaceuticals announced that its cancer drug tamibarotene failed to meet its primary endpoint in the SELECT-MDS-1 Phase III trial. The placebo-controlled SELECT-MDS-1 trial involved 190 patients with complete response rate (CRR) being the study’s primary endpoint. percent and 18.8
It was initially FDA-approved in December 2021 and is claimed to be the only biologic approved to treat severe asthma with no phenotype or biomarker limitation. In the PATH-HOME trial, 92% of healthcare providers, patients and caregivers successfully administered Tezspire in the clinic and at home throughout the study.
The US Food and Drug Administration (FDA) on Wednesday announced it had approved Eli Lilly’s highly anticipated weight loss drug Zepbound, the latest entrant in a field of powerful – and lucrative – new obesity medicines.
On February 7, at a town hall organised to discuss clinicaltrial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.
Related: FDAApproves New Use for Ozempic to Reduce the Risks of Kidney Disease Maze stands out in the biotech space with its Compass platform, which analyzes genetic data to identify disease-linked variants. The Phase I trial showed promising safety and pharmacokinetics. The offering, which includes 8.75
Pfizer has received the US Food and Drug Administration (FDA) approval for its supplemental New Drug Application (sNDA) for Cibinqo (abrocitinib) to treat a type of atopic dermatitis (AD). The label expansion for Cibinqo was supported by the data obtained from the placebo-controlled, randomised Phase III JADE TEEN clinicaltrial.
Oramed Pharmaceuticals announced this week that it has enrolled 100% of the patients in the worldâs first Phase 3 study of oral insulin under FDAapproved protocols.
The regulatory approval is based on the data obtained from the Risperidone Subcutaneous Extended-Release Study (RISE) and the SHINE Phase III trials. Uzedy reduced the risk of schizophrenia relapse by up to 80% compared to placebo in a Phase III clinicaltrial.
The approval was based on findings from the multicentre, open-label, single-arm Phase II FIGHT-203 clinicaltrial that assessed the safety and efficacy of Pemazyre in 28 adult patients with r/r MLNs with FGFR1 rearrangement. For all the trial subjects, a complete cytogenetic response rate of 79% was reported.
Anteris approach has shown promising results in clinicaltrials, demonstrating better hemodynamics and exercise capacity compared to traditional three-piece valves. With US Food and Drug Administration (FDA) approval for an early feasibility study (EFS), Anteris has treated 15 patients across multiple heart valve centers in the US.
The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.
The COVID-19 pandemic has catalysed significant changes in the way pharma develops drugs, particularly in the clinicaltrial space. Hybrid or decentralised clinicaltrials (DCTs) have gained traction as technology, infrastructure and knowledge have evolved to support their use. Source: Izmailova et al, 2017.
Clinicaltrials provide the scientific foundation for justifying the safety and efficacy of drugs, biologics, and devices—but are laborious, expensive, and risky. Only 10% of drugs entering clinicaltrials receive U.S. clinicaltrial workforce reported burnout since the 2020 COVID-19 pandemic.{4}
Despite its potential to slow cognitive decline, the delays highlight the challenges of timely drug approval for life-threatening conditions. The draft builds on prior FDA policies but introduces new provisions from the Consolidated Appropriations Act, 2023.
SUMMARY: Medicare spent nearly $600 million over a three-year period to pay for four cancer drugs with no clinical benefit an analysis published Monday by JAMA Internal Medicine found. Between 2011 and 2018, cumulative spending on 44 FDA-approved oral targeted therapy drugs was $3.5
The FDAs accelerated approval of Vanrafia was based on an interim analysis of the global Phase III ALIGN trial, in which atrasentan added to optimized reninangiotensin system inhibitor therapy achieved a 36.1% reduction in urine proteintocreatinine ratio versus placebo (p<0.0001).
The FDA IND clearance allows the company to expand its ongoing first-in-human Phase I/II clinicaltrial of EP0042. This will enable the addition of US trial sites, as well as the recruitment of eligible patients in the country to the trial.
… Running clinicaltrials remotely or virtually can provide substantial financial savings for pharmaceutical companies, STAT writes, citing a new pilot study. million, on average, for Phase 2 clinicaltrials. million when these methods were used during both trial stages. Or simply plan the rest of your life.
Neilan has contributed to the rare disease community through his comprehensive work in the rare disease space that has included research, academia, medical practice and clinicaltrial design and execution. Can you tell us a bit about the work you are doing at NORD and how it impacts clinicaltrials for rare diseases?
Sling Therapeutics has positive topline results from its Phase IIb/III LIDS clinicaltrial evaluating its oral small-molecule therapy linsitinib for the treatment of thyroid eye disease (TED). Ninety patients in the trial were randomized to receive one of two doses of linsitinib or placebo.
Earlier, the therapy had obtained FDAapproval for usage in CF patients aged two years and above with two F508del mutation copies. The latest approval for use in children aged 12 to below 24 months is based on an open-label, multicentre Phase III clinicaltrial for 24 weeks.
The approval makes Datroway AstraZenecas eighth new medicine since 2020, the first US approval for Daiichi Sankyo and AstraZenecas TROP2-directed ADC and the second based on their proprietary DXd technology. The FDAapproval sent Daiichi Sankyos shares soaring by 8.7% to 4,437 yen, reflecting investor confidence.
Continued approval for the indication is dependent on verification and description of the clinical benefit of Epkinly in confirmatory trials. The FDAapproval of Epkinly represents a new treatment mechanism of action for third-line DLBCL patients. “The
It is claimed to be the first and only therapy to receive approval in the US for the treatment of APDS, a rare and progressive primary immunodeficiency. After assessing the New Drug Application (NDA) under priority review, the FDA granted the approval based on the data obtained from a Phase II/III clinicaltrial.
The US Food and Drug Administration has approved a fast-acting nasal spray from Pfizer designed to treat migraines, the US pharmaceutical giant said Friday.
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