Eye on FDA

AUGUST 4, 2021

Below is a chart that looks at 3 numbers – the number of AdComms; the number of new molecular entities approved each year, and the number of drugs that had breakthrough therapy designation. Fewer meetings – more approvals.

FDA 122

FDA Clinical Pharmacology FDA Approval Therapies 122

Drug Target Review

JANUARY 25, 2024

The peptide in development is based on a 2018 Cleveland Clinic discovery and serves as a proof-of-concept for this type of drug for triple-negative breast cancer. Currently, they are exploring the drug’s potential for clinical trials. This study was published in Molecular Cancer Therapeutics. References 1 Marqus S, Pirogova E, Piva TJ.

Treatment 105

Treatment Clinical Trials Science Research 105

The Pharma Data

DECEMBER 22, 2020

Tagrisso Approved as Adjuvant Therapy for NSCLC With EGFR Mutations. Tagrisso was approved in 2018 for first-line treatment of patients with metastatic non-small cell lung cancer with EGFR exon 19 deletions or exon 21 L858R mutations. Professional. TUESDAY, Dec. Food and Drug Administration announced Friday.

Therapies 52

Therapies Treatment FDA Disease 52

Advarra

OCTOBER 19, 2023

The clinical trial startup process has seen significant shifts over the past five years: growth in decentralized trials, improved technology, and an increased demand for accelerated timelines in getting therapies to market. Sponsor Technology and Training The role of technology in clinical trials has never been more pronounced.

Clinical Trials 52

Clinical Trials Trials Clinical Research Research 52

Drug Target Review

NOVEMBER 6, 2023

2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).

Small Molecule 111

Small Molecule Therapies Treatment Disease 111

The Pharma Data

NOVEMBER 9, 2020

Talaris Chief Executive Officer Scott Requadt sees FCR001, the company’s investigational cell therapy, as a potential pipeline in a product (an experimental treatment that could have multiple uses across a number of indications). The trial is expected to enroll 120 adult patients who will receive kidney donations from living donors.

Therapies 52

Therapies Production Disease Treatment 52

Drug Target Review

OCTOBER 11, 2023

1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Symptoms were reversed in mouse models and a clinical trial is planned for later this year. Gene therapy, of course, also holds great promise. 2018 [cited 2023 Sep 5].

Disease 113

Disease Treatment Small Molecule Therapies 113

PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

FDA Approval 52

FDA Approval Therapies FDA Disease 52

Codon

JULY 14, 2024

While this departs from the reductionist, single-component therapies that have dominated transfusion medicine since World War II, emerging data underscores that whole blood transfusions—blood with all its parts—yield better outcomes following severe blood loss than transfusions involving discrete blood components. Always free.

Production 133

Production Clinical Trials Trials Therapies 133

Conversations in Drug Development Trends

FEBRUARY 28, 2023

Over the next few decades, that drug made its way through clinical trials, securing approval in 2007—just 36 months before Rob was diagnosed in 2010. If we don’t fund this research, we never have the chance for treatments, cures, or innovative therapies. But what was truly remarkable? It really has to start from square one,” says Rob.

Clinical Trials 52

Clinical Trials Disease Trials Research 52

PPD

SEPTEMBER 6, 2023

Approaches to outsourcing clinical trials have changed significantly in recent years. Mixing of service models — a strategy that drug developers are leveraging now more than ever — can bring life-changing therapies to market faster. Growth of the FSP market is steadily increasing. Overseen by an insourced project manager.

Drug Development 98

Drug Development Clinical Trials Drugs Clinical Research 98

The Pharma Data

JANUARY 13, 2021

PITTSBURGH–( BUSINESS WIRE )– Knopp Biosciences LLC today announced positive top-line results in a Phase 2 dose-ranging trial of the novel oral drug dexpramipexole in patients with moderate-to-severe eosinophilic asthma. The trial was conducted at 28 U.S. 14, 2021 11:00 UTC. study centers. ABOUT THE EXHALE STUDY.

Trials 52

Trials Small Molecule Clinical Trials Treatment 52

Codon

NOVEMBER 3, 2024

The experience showed me that new therapies are needed not only to meet the targets laid out by the End TB Strategy but also to prevent drug resistance from negating the effectiveness of current therapeutics. A 1994 review of 14 prospective trials and 12 case-control studies revealed that the BCG vaccine reduced the risk of TB by 50 percent.

Vaccine 85

Vaccine Trials Therapies Treatment 85

The Pharma Data

JANUARY 31, 2021

Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immune responses in a phase 1 trial. Clover plans to initiate a global phase 2/3 trial in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021.

Clinical Trials 52

Clinical Trials Vaccine Trials Virus 52

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 110

Vaccine Trials Disease Treatment 110

Drug Target Review

JUNE 21, 2023

Enabling the systematic discovery of these largely uncharted targets can be a valuable opportunity for development of novel therapies. Genetic-based therapeutics hold significant promise in the clinic Despite the growing number of genes linked to human disease, the proportion of genes targeted by approved therapies remains low.

Disease 111

Disease Clinical Trials Therapies Science 111

The Pharma Data

OCTOBER 20, 2020

In hopes to advance the best and brightest, MD Anderson, in partnership with The Focus Fund GP, has launched the Cancer Focus Fund to propel compelling investigational cancer therapies from preclinical development through Phase II clinical trials. But now they’ll be choosing the therapies themselves. . Cancer is still the No.

Clinical Trials 52

Clinical Trials Therapies Pharma Companies Trials 52

The Pharma Data

JANUARY 14, 2021

(NASDAQ: FPRX) today announced clinical results from the global, randomized, double-blind placebo-controlled Phase 2 FIGHT trial evaluating first-in-class targeted therapy bemarituzumab in advanced gastric or gastroesophageal junction (GEJ) cancer. Phase 2 FIGHT Trial: Summary of Efficacy*. Wainberg, M.D.,

Trials 52

Trials Treatment Therapies Clinical Trials 52

Drug Target Review

AUGUST 24, 2023

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo. in North America) in late 2017.

Treatment 98

Treatment Therapies Clinical Development Cell Biology 98

The Pharma Data

DECEMBER 9, 2020

9, 2020 — More women with early-stage breast cancer may be able to safely skip chemotherapy after having surgery, according to initial results from a major clinical trial. The trial, conducted in nine countries, found that adding chemotherapy to hormone-blocking drugs brought no added benefit to a particular group of patients.

Trials 52

Trials Therapies Clinical Trials Treatment 52

New Drug Approvals

SEPTEMBER 8, 2024

2222514-07-8 ] Palopegteriparatide , sold under the brand name Yorvipath , is a hormone replacement therapy used for the treatment of hypoparathyroidism. [1] 5] History The effectiveness of palopegteriparatide was evaluated in a 26-week, randomized, double-blind, placebo-controlled trial that enrolled 82 adults with hypoparathyroidism. [5]

FDA 62

FDA International Trials FDA Approval 62

The Pharma Data

OCTOBER 22, 2020

. INTERLINK-1 represents first Phase 3 study examining IO approach in R/M SCCHN patients who have been treatedwith a platinum-based therapy and PD-(L)1 inhibitor. Dosing of the first patient in this trial has triggered a $50 million milestone payment from AstraZeneca to Innate. “W MARSEILLE, France, Oct.

Clinical Trials 40

Clinical Trials Trials Cell Biology Production 40

Drug Target Review

SEPTEMBER 26, 2023

Early detection of neurotoxicity induced by potential new therapies is a major challenge, and hiPSC-neuronal cells may provide a solution. Dr Rockley previously completed an MSc in Cancer Pharmacology and have experience in clinical research from an oncology clinical trial coordinator role. 2018 Mar;17(3):167–81. 2016;81:37–46.

Drugs 111

Drugs FDA Disease Animal Testing 111

The Pharma Data

NOVEMBER 1, 2020

The trial is expected to enroll 160 patients. billion in 2018, and it is expected to be up to US$48.9 I am very delighted to learn the swift IND clearance by the US FDA, and we are initiating the phase II clinical trial immediately,” said Dr. Jasmine Cui, the co-founder, chairman, and Chief Executive Officer of InnoCare.

Clinical Trials 40

Clinical Trials Trials FDA Treatment 40

The Pharma Data

JANUARY 14, 2021

The approval was based on data from Study ADVL0912, a multicenter, single-arm, open-label trial in 121 patients ranging in ages from one to 21, and included 26 patients with r/r/, systemic ALK-positive ALCL after at least one systemic treatment. For the full year of 2019, Xalkoria brought in $530 million, an increase of 1% from 2018. “We

FDA Approval 52

FDA Approval FDA Therapies Treatment 52

Drug Target Review

APRIL 3, 2024

Our company has identified a new role for Growth Differentiation Factor 15 (GDF-15) as a potent local immunosuppressor mediating cancer resistance to therapy. She joined the company in November 2018 with more than 10 years of experience in drug discovery and non-clinical development of immunomodulatory drugs in the immuno-oncology space.

Science 105

Science Clinical Development Treatment DNA 105

New Drug Approvals

SEPTEMBER 10, 2024

2] [3] It is the first approval by the US Food and Drug Administration (FDA) of a systemic therapy for people with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation. [2] April 2018). mol), (R)-1,1,1-trifluoropropan-2-amine hydrochloride (0.39 6 August 2024.

FDA Approval 62

FDA Approval FDA Clinical Trials Trials 62

FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

An Opportunity to Convene this New Advisory Committee for All Rare Disease Therapies In 2018, HP&M attorneys Frank Sasinowski and James Valentine proposed a Rare Disease Center of Excellence (which we blogged about here ). Note that FDA is currently soliciting applications to staff this committee.

Disease 59

Disease Production FDA Drug Development 59

The Pharma Data

MAY 5, 2022

1 Clinical trials for new therapies in AML are becoming increasingly lengthy to complete based on the traditional clinical endpoint of survival. “Measuring MRD as a surrogate endpoint may help forge a path to bring new AML therapies to patients quickly.”

Disease 52

Disease Clinical Trials Drug Development Pharmaceutical Companies 52

The Pharma Data

DECEMBER 31, 2020

Tabibiazar co-founded private Aravive Biologics and served as the Chairman of its board of directors and as President and Chief Executive Officer from its inception to April 2017 and as Executive Chairman from May 2017 until October 2018. since October 2018. to form the combined company, Aravive, Inc.

Clinical Trials 52

Clinical Trials Trials Disease Therapies 52

The Pharma Data

JULY 22, 2021

ARV-471 is currently in a Phase 2 dose expansion clinical trial for the treatment of patients with estrogen receptor (ER) positive / human epidermal growth factor receptor 2 (HER2) negative (ER+/HER2-) locally advanced or metastatic breast cancer. The estrogen receptor is a well-known disease driver in most breast cancers.

Targeted Protein Degradation 52

Targeted Protein Degradation Therapies Clinical Trials Treatment 52

The Pharma Data

JANUARY 20, 2021

The combination therapy involves an injectable medication called extended-release naltrexone and a daily generic pill called bupropion. But between 2017 and 2019, more than 400 adults with moderate-to-severe methamphetamine use disorder participated in a combination treatment trial, and the results suggest the combination works.

Drugs 52

Drugs Therapies Treatment Clinical Trials 52

The Pharma Data

NOVEMBER 24, 2020

Lynparza is the first biomarker-selected targeted treatment option approved in Canada , based on the Phase III PROfound trial of men with Homologous Recombination Repair mutated (HRRm) mCRPC. Approximately 32 patients from 12 Canadian centres have participated in the PROfound trial to date. 3, 4, 5, 6 . ” .

Treatment 52

Treatment DNA Trials Therapies 52

The Pharma Data

AUGUST 5, 2020

Ofatumumab is a targeted B-cell therapy that delivers superior efficacy with a similar safety profile when compared with teriflunomide, a commonly prescribed oral treatment for multiple sclerosis 1 . Low-dose, subcutaneous anti-CD20 therapy effectively depletes B-cells and ameliorates CNS autoimmunity. Eur J Neurol. 2020;27(S1).

Trials 52

Trials Treatment Disease Therapies 52

The Pharma Data

MARCH 3, 2022

Pfizer will publish outcomes from this clinical trial at a future date. 1 This decision follows the FDA’s November 2018 decision to grant Fast Track status to RSVpreF. 1 This decision follows the FDA’s November 2018 decision to grant Fast Track status to RSVpreF. Pfizer Inc. 4,5,6,7 In the United States alone, approximately 2.1

Virus 52

Virus Vaccine Clinical Trials Trials 52

The Pharma Data

DECEMBER 3, 2020

The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing eight weeks of treatment with either maribavir or investigator assigned treatment (IAT) in transplant recipients with CMV infection refractory or resistant to existing antiviral treatments (i.e.,

Treatment 52

Treatment Pharmaceutical Companies Therapies Virus 52

The Pharma Data

NOVEMBER 8, 2020

The New Drug Application (NDA) for the drug is based on data from four Phase III trials in pediatric patient populations from the age of 6 to 17 years, two Phase II trials, several Phase I trials, a long-term open label extension study, preclinical testing, and drug manufacturing data. This is under Priority Review.

FDA 52

FDA Trials Therapies Pharmacokinetics 52