The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. For such trials, the US Food and Drug Administration (FDA) may allow the use of credible real-world data (RWD) and real-world evidence (RWE) in lieu of data collected in a Phase 3 trial.

Disease 52

Disease FDA FDA Approval Regulations 52

The Pharma Data

MAY 7, 2021

regulators to seek approval of our COVID-19 vaccine based on our pivotal Phase 3 trial and follow-up data.”. This includes the most recent analyses from the pivotal Phase 3 clinical trial, where the vaccine’s efficacy and favorable safety profile were observed up to six months after the second dose.

Licensing 52

Licensing Licensing Vaccine FDA Approval 52

The Pharma Data

OCTOBER 14, 2020

WAKIX is the first and only treatment approved by the FDA for people with excessive daytime sleepiness or cataplexy associated with narcolepsy that is not scheduled as a controlled substance by the U.S. WAKIX received FDA approval for the treatment of excessive daytime sleepiness in adult patients with narcolepsy in August 2019.

FDA Approval 52

FDA Approval FDA Treatment Pharmaceutical Companies 52

FDA Law Blog: Biosimilars

JULY 24, 2023

Approximately six weeks after FDA approved Lumryz and issued its clinical superiority decision , Jazz filed a Complaint against FDA in the District Court of D.C. Jazz also argues that, notwithstanding the language that instructs FDA to promulgate regulations implementing the clinical superiority provisions at 21 U.S.C.

FDA 52

FDA Regulations Clinical Trials Production 52

The Pharma Data

OCTOBER 25, 2020

The Company plans to initiate a Phase 2 trial within the next several months. “We An exploratory endpoint of this trial will be to determine the overall clinical improvement after drug administration using the Clinical Global Impression – Improvement Scale (“CGI-I”).

Treatment 52

Treatment Trials FDA Hospitals 52

Drug Discovery World

MARCH 19, 2024

It is approved in adult patients who have received at least two prior lines of therapy, including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor. Among 89 patients in the study treated with Breyanzi, occurrences of cytokine release syndrome (CRS) and neurologic events (NEs) were mostly low grade.

FDA Approval 130

FDA Approval Therapies FDA Treatment 130

The Pharma Data

JANUARY 29, 2021

Following review of the application, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on data from a rolling review of trial data from the primary analysis of the Phase III programme led by the University of Oxford.

Vaccine 52

Vaccine Clinical Trials Virus Trials 52

The Pharma Data

JANUARY 29, 2021

Following review of the application, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on data from a rolling review of trial data from the primary analysis of the Phase III programme led by the University of Oxford.

Vaccine 52

Vaccine Clinical Trials Virus Trials 52

The Pharma Data

MARCH 11, 2021

Retevmo was approved under the FDA’s Accelerated Approval regulations based on the LIBRETTO-001 Phase 1/2 trial’s endpoints of overall response rate (ORR) and duration of response (DoR). LY3484356, an oral SERD, is currently being studied in a Phase 1/2 clinical trial. Pipeline Highlights.

Research 52

Research Clinical Trials FDA Approval Trials 52

The Pharma Data

JANUARY 6, 2021

The vaccine was shown in clinical trials to be safe and effective at preventing symptomatic COVID-19, with no severe cases and no hospitalisations more than 14 days after the second dose. The approval in India is an important milestone as it will enable to supply India but also a large number of countries around the world.

Vaccine 52

Vaccine Virus Clinical Trials Government 52

The Pharma Data

JULY 21, 2021

mg, 5 mg, and 10 mg) is indicated for the treatment of symptomatic chronic heart failure in adult patients with reduced ejection fraction who are stabilized after a recent decompensation event requiring intravenous (IV) therapy. Bayer also issued a news release earlier today announcing the EC approval. In the EU, VERQUVO (2.5

Treatment 52

Treatment Production Hospitals FDA Approval 52

The Pharma Data

DECEMBER 18, 2020

. “Guided by science and data, the agency’s career staff determined that the vaccine’s known and potential benefits clearly outweigh its known and potential risks, and although not an FDA approval, the FDA’s expectations described in our June and October guidance documents have been met,” said Peter Marks , M.D.,

Vaccine 52

Vaccine FDA Clinical Trials FDA Approval 52

The Pharma Data

JULY 22, 2021

The approval for this population is based on results from the pivotal Phase 3 KEYNOTE-775/Study 309 trial, in which KEYTRUDA plus LENVIMA demonstrated statistically significant improvements in overall survival (OS), reducing the risk of death by 32% (HR=0.68 [95% CI, 0.56-0.84];

Therapies 52

Therapies Treatment Disease Trials 52

The Pharma Data

OCTOBER 15, 2020

The second study, presented by Tayyab Shah, MD, of the Yale School of Medicine, analyzed data collected from the RECOVER III trial between 2017-2019. This is an interesting result from an observational study with meaningful clinical implications, which need to be further explored in the upcoming RECOVER IV randomized controlled trial.”.

Hospitals 52

Hospitals FDA Approval FDA Treatment 52

The Pharma Data

OCTOBER 25, 2020

SQI intends to submit RALI-dx for EUA to FDA in late Q4 2020. The RALI-dx COVID-19 Severity Triage Test is expected to be used primarily in hospital emergency departments upon FDA approval. SQI intends to submit RALI- fast for EUA to FDA in late Q1 2021. . Source link.

FDA 52

FDA Hospitals Clinical Development FDA Approval 52

Agency IQ

SEPTEMBER 15, 2023

BY AMANDA CONTI SEP 13, 2023 1:58 PM CDT Quick background on nonprescription drug regulation Nonprescription drugs, also known as over-the-counter (OTC) drugs, are regulated differently than traditional prescription drugs. The FDA will follow these procedures for both agency-initiated operations (e.g.,

Science 40

Science FDA Clinical Trials Pharmacy 40

The Pharma Data

NOVEMBER 16, 2020

We are pleased to submit these extended stability conditions for mRNA-1273 to regulators for approval. government, as well as global stakeholders to be prepared for distribution of mRNA-1273, in the event that it receives an Emergency Use Authorization and/or similar global authorizations. Source: Moderna, Inc. .

Vaccine 52

Vaccine Pharmacy FDA Clinical Trials 52

PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

Clinical Trials 98

Clinical Trials Trials Clinical Research Disease 98

The Pharma Data

SEPTEMBER 7, 2020

Novartis will present 48 abstracts from its leading MS portfolio, including new data on recently FDA-approved Kesimpta ® (ofatumumab) — the first and only self-administered, targeted B-cell therapy for relapsing forms of MS (RMS )— Mayzent ® (siponimod) and Gilenya ® (fingolimod) .

Therapies 52

Therapies Clinical Trials Treatment Disease 52

The Pharma Data

JUNE 18, 2021

1,2,4,5 Additionally, the rates of events of special interest were generally comparable between the RINVOQ and HUMIRA groups. 1 Serious adverse events occurred at 10.9 events per 100 patient years (100PY) for RINVOQ compared to 14.1 events/100PY for HUMIRA. events/100PY on RINVOQ and 3.5 events/100PY on HUMIRA.

Treatment 40

Treatment Disease Therapies Clinical Trials 40

The Pharma Data

FEBRUARY 8, 2021

1 Secondary endpoints included time to cytotoxic chemotherapy, time to pain progression, time to chronic opioid use, and time to skeletal-related events. [3] Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019. ERLEADA ® received U.S.

Treatment 52

Treatment Pharmaceutical Companies Therapies Pharmaceuticals 52

The Pharma Data

JUNE 18, 2021

1 The full, long-term results from the Phase 3 SELECT-PsA 2 clinical trial will be presented at the EULAR 2021 Virtual Congress. 1 The full, long-term results from the Phase 3 SELECT-PsA 2 clinical trial will be presented at the EULAR 2021 Virtual Congress. events/100PY on RINVOQ 15 mg. The approved dose for RINVOQ is 15 mg.

Treatment 40

Treatment Therapies Clinical Trials Disease 40

Agency IQ

JULY 21, 2023

BY RACHEL COE, MSC JUL 18, 2023 11:14 PM CDT Oncology endpoints: The growing pains of an evolving field FDA approval of a drug or biologic relies on whether there is “substantial evidence” of safety and effectiveness derived from “adequate and well-controlled clinical investigations” according to the Federal Food, Drug, and Cosmetic (FD&C) Act.

FDA 40

FDA Clinical Trials Treatment DNA 40

Drug Discovery World

AUGUST 10, 2022

Finally, in 2017, after years of improving T cell engineering techniques, the first chimeric antigen receptor (CAR) T cell therapy received FDA approval. Since 2017, five CAR T cell therapies have been approved and have shown incredible success in the clinic. CAR T cells are T cells that have been trained to attack tumours.

Therapies 246

Therapies Virus Treatment Engineering 246

The Pharma Data

APRIL 28, 2021

This EC approval closely follows the U.S. Food and Drug Administration (FDA) approval of Sarclisa for a similar indication in March 2021. Fatal adverse events were reported in 3.4% For more information on Sarclisa clinical trials please visit www.clinicaltrials.gov. . of patients treated with Kd. In the U.S.,

Therapies 40

Therapies Clinical Trials Treatment Disease 40

Codon

JULY 14, 2024

However, the subsequent death of another patient thrust Denys into a contentious trial. Despite the high costs, researchers in Kyoto generated enough cultured platelets in 2022 to transfuse a 55-year-old woman for an initial human trial. The results of the trial are expected at the end of 2024. Data from Rousseau G.F.

Production 133

Production Clinical Trials Trials Therapies 133

The Pharma Data

JANUARY 24, 2021

1 Sairiyo has an exclusive license from a research and development organization to develop and commercialize reformulated Cepharanthine for all diseases and exclusive rights to the patent, method of manufacturing, clinical supply, pre-clinical data and know-how to support FDA clinical trials. References: Bailly, C.,

FDA 52

FDA Drugs Clinical Development Pharmaceutical Companies 52

Vial

DECEMBER 5, 2023

Among others, NGS has led to the identification of disease-causing variants and novel drug targets and an improved understanding of complex biological events, e.g., the heterogeneity of tumors. In addition, NGS facilitates transcriptomics , epigenomics, metagenomics, and other omics studies. Fountzilas et al.

Clinical Trials 52

Clinical Trials Trials RNA Bioinformatics 52

The Pharma Data

JUNE 18, 2021

May 24, 2021 /PRNewswire/ — AbbVie (NYSE: ABBV) today announced The Lancet published primary analysis results from the pivotal global Phase 3 clinical trials – Measure Up 1, Measure Up 2 and AD Up – evaluating RINVOQ ® (upadacitinib) in adults and adolescents with moderate to severe atopic dermatitis who were candidates for systemic treatment.

Treatment 40

Treatment Disease FDA Vaccine 40

The Pharma Data

JANUARY 21, 2021

Results from these trials were presented at the 2019 Conference on Retroviruses and Opportunistic Infections (CROI). These events may have been associated with inadvertent (partial) intravenous administration and began to resolve within a few minutes after the injection. Hepatotoxicity: Hepatic adverse events were reported.

FDA Approval 52

FDA Approval Treatment RNA FDA 52

Agency IQ

OCTOBER 20, 2023

” Interchangeable products can be substituted for the reference product at the pharmacy level, without prescriber intervention, within state and local regulations. The size of the trials was comparable to those of new molecular entities—drugs that have never before been approved by the FDA.

Science 40

Science Biosimilars FDA Production 40

The Pharma Data

NOVEMBER 4, 2020

REDUCE-IT ® PCI results presented in October 2020 showed through post hoc subgroup analyses that patients in the REDUCE-IT study who had stenting, bypass or other forms of percutaneous cardiovascular intervention (PCI) experienced significantly reduced rates of ischemic events when treated with VASCEPA. commercial team. commercial team. “In

Production 40

Production FDA Trials Licensing 40

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) approved LUMAKRAS for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic NSCLC, as determined by an FDA-approved test, who have received at least one prior systemic therapy. In May, the U.S. Bemarituzumab. Tarlatamab (AMG 757). AMG 451 / KHK4083.

Biosimilars 52

Biosimilars Production Treatment FDA 52

Agency IQ

DECEMBER 1, 2023

What We Expect the FDA to do in December 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

FDA 40

FDA Science Regulations Production 40

The Pharma Data

JANUARY 13, 2021

Fast Track designation is well-timed, as we anticipate starting our Phase 2 clinical trial in hospitalized COVID-19 patients this month, and should help bring Brilacidin to patients faster in these dire times.”. Brilacidin, a versatile compound with broad therapeutic potential, is in a new chemical class called defensin-mimetics.

Clinical Trials 52

Clinical Trials Trials Pharmaceuticals FDA 52

Agency IQ

SEPTEMBER 29, 2023

What We Expect the FDA to do in October 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more. The remaining 19% would be furloughed.

FDA 40

FDA Science Regulations Production 40

Agency IQ

OCTOBER 27, 2023

What We Expect the FDA to do in November 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

FDA 40

FDA Science Regulations Production 40