PPD

OCTOBER 12, 2023

Since chimeric antigen receptor T cell (CAR-T) therapy was first approved in 2017, there has been a marked increase of cell and gene therapy studies resulting in significant changes in the way diseases are treated as well as patient outcomes. However, key differences between PCR assays and immunotherapeutic assays must be considered.

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FDA Law Blog: Drug Discovery

FEBRUARY 2, 2023

Since 1962, the FD&C Act has authorized FDA to require that sponsors of clinical trials submit data from “preclinical tests (including tests on animals)” in order to demonstrate that their drug is safe enough to advance to testing in humans. For more on FDORA’s other provisions, see HPM’s complete summary here ). FDORA § 3209(a)(1).

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Animal Testing Biosimilars Cell Based Assays FDA 64

The Pharma Data

MARCH 22, 2021

GENERATION HD1 is the largest clinical trial in Huntington’s disease to date and we do know that the data generated will significantly advance our understanding of huntingtin-lowering as a potential treatment approach. About tominersen and the clinical trials. said Levi Garraway, M.D.,

Disease 52

Disease Clinical Trials Pharmacokinetics Treatment 52

The Pharma Data

JANUARY 3, 2021

This trial is the first to evaluate nasally administered Foralumab to improve the immune system’s fight against coronaviruses. The last patients in the trial received their final dose on 21 December 2020. The topline data from the trial is expected to be available in January 2021. NEW YORK and LONDON, Jan.

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New Drug Approvals

OCTOBER 24, 2023

This approval was based on favorable results obtained from Pfizer’s Elevate UC Phase III registrational program, consisting of the Elevate UC 52 and Elevate UC 12 clinical trials, that investigates the efficacy of a 2-mg daily dose regimen of etrasimod, with a 32% and 26% remission rate observed in UC 52 and UC 12 trials respectively.

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FDA FDA Approval International Pharmacokinetics 57

New Drug Approvals

SEPTEMBER 29, 2024

2017 ; Ilievski et al., Findings Two Phase 1 trials of atuzaginstat were completed by June 2019. The pharmacokinetic profiles of COR388 in AD and controls were reported to be similar. A Phase 2/3 trial (GAIN) evaluating a 48-week course of COR388 in 643 people with mild to moderate AD began in April 2019. and Europe.

Small Molecule 62

Small Molecule DNA Disease Trials 62

The Pharma Data

DECEMBER 15, 2020

SetPoint Medical received FDA Investigational Device Exemption (IDE) approval for a multicenter, double-blind, randomized, sham-controlled pivotal trial that will enroll up to 250 patients at 40 clinical trial sites in the U.S. The secondary outcome involves pharmacokinetic endpoints. Small Molecule Inhibitors.

Disease 52

Disease Clinical Trials Small Molecule Trials 52

Alta Sciences

APRIL 17, 2024

The FDA approved the first gene therapy in 2017 for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), and as of December 2023, there are over 30 approved cell and gene therapies on the market in the U.S.A. Several bioanalytical strategies for the quantitation of ASOs are available at Altasciences.

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Drug Development Treatment FDA Approval Therapies 52

New Drug Approvals

JANUARY 31, 2024

citation needed ] The pharmacokinetic properties of obeldesivir and improved was first published by Chinese researchers in May 2022. 2017, 60, 1648-1661) in tetrahydrofuran (THF) was added N,N-dimethylaminopyridine (DMAP) (0.03 Obeldesivir, 2647441-36-7 361.35 mmol) (Siegel et. To the reaction mixture, isobutyric anhydride (1.1

RNA 52

RNA Science Treatment Pharmacokinetics 52

Agency IQ

FEBRUARY 23, 2024

On the first day, discussants looked at how to tackle first-in human trials, weighed the importance of the maximum tolerated dose, and looked at the evolving concept of the clinical utility index. This workshop built on three prior workshops held by the FDA-AACR, which took place in 2015, 2016 and 2017.

Science 40

Science FDA Trials Clinical Pharmacology 40

The Pharma Data

SEPTEMBER 15, 2020

Limited blinded tolerability data from the ongoing Phase 3 trial, confirming the mostly mild to moderate tolerability profile as was observed in Phase 1. In the blinded data presented, 50% of trial participants received placebo and 50% received BNT162b2. A Phase 3 trial is ongoing. Protease Inhibitor Program. Rare Disease.

Vaccine 52

Vaccine Therapies Trials Clinical Trials 52

Agency IQ

DECEMBER 11, 2023

Oncology at FDA in 2023: A quick glance at the numbers First created under the 21st Century Cures Act of 2016 and launched in 2017, FDA’s Oncology Center of Excellence (OCE ) “unites experts across the FDA to conduct expedited review of medical products for oncologic and hematologic malignancies.” What’s ahead? Putting policy into practice.

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The Pharma Data

NOVEMBER 6, 2020

Since October 2017 Biogen and Eisai have collaborated on the development and commercialization of aducanumab globally. Secondary outcomes were: (1) the effect of aducanumab on brain amyloid plaque content as measured by [18F]-florbetapir PET, (2) the pharmacokinetics of aducanumab and (3) the immunogenicity of aducanumab.

Disease 40

Disease FDA Clinical Trials Licensing 40

The Pharma Data

FEBRUARY 8, 2021

Patients in the trial received either a combination of ERLEADA ® and ZYTIGA ® plus prednisone (combination arm) or placebo and ZYTIGA ® plus prednisone (control arm). [i] v] ERLEADA ® is being studied in five Phase 3 clinical trials. For more information about ERLEADA ® , visit www.ERLEADA.com. *© All rights reserved.

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Pharmaceutical Companies Treatment Clinical Trials Therapies 52

The Pharma Data

OCTOBER 27, 2020

The growth in Orenitram revenues resulted primarily from an increase in quantities sold, as the number of patients being treated with Orenitram grew following the update to Orenitram’s labeling to reflect the FREEDOM-EV clinical trial results, partially offset by the impact of the Excess Order. Implantable System for Remodulin (ISR).

FDA 40

FDA Production Licensing Licensing 40

Agency IQ

SEPTEMBER 15, 2023

In 2017, the FDA expanded the restrictions , which include behind-the-counter sale requiring photo identification with a per-month individual purchase quota, to products containing ephedrine and phenylpropanolamine — effectively narrowing the decongestant drugs approved for over-the-counter sale.

Science 40

Science FDA Clinical Trials Clinical Pharmacology 40

Agency IQ

AUGUST 16, 2024

Throughout 2017, Lykos sought feedback on the protocol for its first Phase 3 study, MAPP1, via the Special Protocol Assessment (SPA) process. Following some back-and-forth regarding statistical analyses and endpoints and abuse potential studies, the agency agreed to a revised SPA in July 2017.

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FDA Trials Therapies Regulations 40

The Pharma Data

APRIL 8, 2021

New 2-year findings from Part 2 of the Phase II/III FIREFISH trial show longer-term efficacy and safety of EVRYSDI in infants with symptomatic Type 1 SMA treated with EVRYSDI. P6: Neuromuscular Disorders and Clinical Trials. P6: Neuromuscular Disorders and Clinical Trials. Multiple Sclerosis (MS).

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Clinical Trials Trials Disease Therapies 52

The Pharma Data

NOVEMBER 9, 2020

The HIV Prevention Trials Network study (HPTN 084) on the safety and efficacy of the long-acting injectable antiretroviral drug cabotegravir (CAB LA), for pre-exposure prophylaxis (PrEP) in HIV-uninfected women, was stopped early by the trial Data and. Thirty-eight women in the trial acquired HIV. Implications of the results.

Trials 52

Trials Pharmacokinetics Drugs Therapies 52

The Pharma Data

OCTOBER 6, 2021

.” In August, 2021, AstraZeneca declared high- position results from the PROVENTpre-exposure prophylaxis trial which showed AZD7442 reduced the imminence of developing discriminating COVID-19 by 77 (95 confidence interval (CI) 46, 90), compared to placebo. The trial accrued 25 cases of discriminating COVID-19 at the primary analysis.

Pharmacokinetics 52

Pharmacokinetics Trials Government Vaccine 52

Agency IQ

AUGUST 16, 2024

chemotherapies), sponsors have historically pushed the envelope to find the highest tolerable dose when conducting clinical trials for oncology products, since higher doses generally mean increased efficacy for these products. In early-stage development of traditional cytotoxic oncology drugs (i.e.,

Production 40

Production FDA Science Clinical Trials 40

The Pharma Data

DECEMBER 20, 2020

Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. The primary objective of the trial is to evaluate the safety and tolerability of SAD and MAD of ALS-4 administered orally to healthy subjects. About ALS-4.

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Small Molecule Clinical Trials Trials Treatment 40

The Pharma Data

FEBRUARY 25, 2021

FIREFISH, an open-label, two-part pivotal study, was designed to assess Evrysdi safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) in patients aged 1 to 7 months with Type 1 SMA. Evrysdi has been and continues to be studied in more than 450 people as part of a large and robust clinical trial program in SMA.

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Pharmacokinetics FDA Approval Treatment Disease 52

The Pharma Data

MARCH 16, 2021

More than 2,500 patients now treated with Evrysdi in clinical trial, compassionate use and real-world settings. Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively. The study met its primary endpoint.

Pharmacokinetics 52

Pharmacokinetics Treatment Clinical Trials Disease 52

Agency IQ

FEBRUARY 15, 2024

These topics include administrative, advanced manufacturing, the Animal Rule, antimicrobials, biosimilars, blood products, breakthrough devices, cell and gene therapy products, CGMP, clinical pharmacology, clinical trials, clinical/medical guidance, combination products, communication, compounding, cybersecurity, device software, digital health technologies, (..)

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The Pharma Data

JANUARY 19, 2021

The Phase 1 clinical trial is planned to be conducted in Canada and targeted to recruit up to 48 and 24 healthy volunteers for the single-ascending dose (SAD) and multiple- ascending dose (MAD) cohorts, respectively. About ALS-4. This press release is provided “as is” without any representation or warranty of any kind.

Small Molecule 52

Small Molecule Clinical Trials Trials Pharmacokinetics 52

The Pharma Data

SEPTEMBER 27, 2020

These results build on the efficacy and safety demonstrated by Evrysdi in pivotal trials, and we look forward to continued assessments of both survival and motor function during long-term follow up for this first-of-its-kind treatment.”. Roche’s Chief Medical Officer and Head of Global Product Development. months and the oldest was 45.1

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Pharmacokinetics FDA Approval Treatment Clinical Trials 52