Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

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Disease Clinical Trials Therapies Science 111

The Pharma Data

JANUARY 14, 2021

Food and Drug Administration (FDA) approved Pfizer ’s Xalkori (crizotinib) for pediatric patients one year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive. Manuel Esteban/Shutterstock. Most Read Today. Source link.

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The Pharma Data

AUGUST 3, 2021

end-stage renal disease (ESRD) bundled payment system, dialysis clinics rapidly implemented new treatment protocols, switching patients from Parsabiv to generic oral cinacalcet. LUMAKRAS received accelerated approval based on overall response rate and duration of response. With Parsabiv’s inclusion in the U.S. In May, the U.S.

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Agency IQ

OCTOBER 6, 2023

Taking a closer look, nearly half of the products were reviewed by the Office of Oncologic Diseases. The largest portion of reviews after that were 14% (3 NMEs) each with the Office of Neuroscience and the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine. of all NME approvals this year.

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Broad Institute

OCTOBER 11, 2023

By Allessandra DiCorato October 11, 2023 In 2011, Robert Manguso was working in a cell biology lab when his mother was diagnosed with Merkel cell carcinoma, a rare and aggressive skin cancer. His mother had a presentation of the disease that suggested her immune system was already on the job.

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Research Immune Response Clinical Trials Cell Biology 137

The Pharma Data

AUGUST 11, 2020

.–( BUSINESS WIRE )– Bristol Myers Squibb (NYSE: BMY) today announced the Phase 3 CheckMate -577 trial evaluating Opdivo (nivolumab) as an adjuvant therapy for patients with resected esophageal or gastroesophageal junction (GEJ) cancer met its primary endpoint of disease-free survival (DFS) at a pre-specified interim analysis.

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The Pharma Data

DECEMBER 21, 2020

While up to 30% of all patients with NSCLC may be diagnosed early enough to have potentially curative surgery, disease recurrence is still common in early-stage disease and nearly half of patients diagnosed in Stage IB, and over three quarters of patients diagnosed in Stage IIIA, experience recurrence within five years.

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Treatment Trials Disease FDA 52

The Pharma Data

AUGUST 17, 2021

Applications based on positive results from the Phase 3 CheckMate -648 trial, in which both Opdivo -based combinations demonstrated a significant survival benefit over chemotherapy alone. Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -648 trial. About CheckMate -648. About Esophageal Cancer.

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Treatment Immune Response Trials FDA Approval 52

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] Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019. All rights reserved.

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Pharmaceutical Companies Treatment Clinical Trials Trials 52

Codon

MAY 22, 2023

This gene is shared by monkeys and humans and, when it’s mutated in juuuust the right way, can lower LDL and reduce heart disease risk. Verve already started a Phase 1b trial in New Zealand last July. halted a Verve trial in the U.S. An important proof-of-concept for human trials. Again, this is a big deal.

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DNA Engineering Science Trials 55

Codon

MAY 22, 2023

This gene is shared by monkeys and humans and, when it’s mutated in juuuust the right way, can lower LDL and reduce heart disease risk. Verve already started a Phase 1b trial in New Zealand last July. halted a Verve trial in the U.S. An important proof-of-concept for human trials. Again, this is a big deal.

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DNA Engineering Science Trials 52

The Pharma Data

SEPTEMBER 8, 2021

CheckMate -743 remains the only Phase 3 trial to show a survival benefit with first-line immunotherapy treatment in patients with MPM. First-line nivolumab + cabozantinib vs sunitinib in patients (pts) with advanced renal cell carcinoma (aRCC) in subgroups based on prior nephrectomy in the CheckMate 9ER trial. Dr. Camillo Porta.

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Research Immune Response Treatment Trials 52

The Pharma Data

AUGUST 11, 2020

The safety profiles of Opdivo and chemotherapy in this trial are reflective of the known safety profiles of Opdivo and chemotherapy in first-line gastric and esophageal cancers. The company remains blinded to data from this arm and the trial continues in follow-up to allow the data to mature. PRINCETON, N.J.–( Janjigian, M.D.,

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Treatment Trials Therapies Clinical Trials 52

The Pharma Data

OCTOBER 25, 2020

Real-world effectiveness data on Oxbryta ® (voxelotor) in the treatment of sickle cell disease featured as oral presentation. Two abstracts have been accepted for presentation, including real-world effectiveness data of Oxbryta ® (voxelotor) tablets in the treatment of sickle cell disease (SCD). FDA approval. 26-31, 2020.

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Treatment Disease FDA FDA Approval 52

The Pharma Data

JUNE 26, 2021

If approved, XARELTO ® will be the first and only oral Factor Xa inhibitor indicated in the U.S. There are currently no FDA-approved anticoagulation therapies for pediatric patients with congenital heart disease who have undergone the Fontan procedure, a surgical procedure that redirects blood flow from the lower body to the lungs.

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Doctors Hospitals Clinical Trials Treatment 52

Drug Discovery World

FEBRUARY 22, 2024

According to Alzheimer’s Research UK (ARUK): “In the UK, dementia was the leading cause of death in 2022 and has been the leading cause of death for women since 2011 – maintaining its position throughout the Covid-19 pandemic. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In

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Drugs Clinical Trials Disease Therapies 147

Agency IQ

SEPTEMBER 22, 2023

Traditionally, FDA has interpreted the need for “well-controlled investigations” to mean at least two clinical trials for new drugs, or applications for supplemental indications. Three drugs were approved based on no pivotal trials (such as in cases where an approval leveraged literature on use of the drug).

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Drug & Device Law

DECEMBER 21, 2023

Shikada is the first appellate decision creating a state-law warning-based duty (here, via a consumer protection claim, brought by the state rather than anyone actually claiming injury) based solely on pharmacogenomics − racially/ethnically-based genetic variations − allegedly affecting the effectiveness of FDA-approved prescription drugs.

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FDA Approval FDA Drugs Production 52

Drug & Device Law

JULY 24, 2023

To put that in context, the Actos label also carries the FDA’s most serious type of warning – a boxed warning – but for congestive heart failure, not bladder cancer. precipitously” after the 2011 label change adding the bladder cancer language, a “significant number” of claims would not have been reimbursed by TPPs nationwide.

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Testimonials Pharmacy FDA Doctors 52

Drug & Device Law

DECEMBER 29, 2023

Infectious Diseases Society of America , 86 F.4th 2023) (remote trial testimony cannot be compelled beyond Rule 45’s 100-mile limit on subpoenas) ( here ); Carson v. Thus, CEH turned on implied impossibility preemption, specifically an application of Mensing ( 2011+1 ) independence principle. du Pont de Nemours & Co.

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Drug & Device Law

MARCH 16, 2023

In two of these cases, our client won summary judgment at the trial court level and an appellate court ended up creating a new cause of action to accommodate the plaintiff’s theory (and lack of helpful testimony from the prescribing physician). 604 (2011), and Mutual Pharm. Wyeth Ayerst Pharms. , 3d 364 (1st Cir. 3d 1034 (S.D.

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FDA Approval FDA Testimonials Production 59

Drug & Device Law

FEBRUARY 22, 2024

In particular, plaintiffs trying to force clinical trial sponsors to continue providing free drug after the end of the trial—something clinical trial participants are told is not a guaranty—have floundered. We do not even need to call out the emphasis that plaintiff lawyers place on FDA approval when it suits them.

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