The Pharma Data

JANUARY 14, 2021

Xalkori is a tyrosine kinase inhibitor indicated since 2011 for patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are ALK- or ROS1-positive. Xalkoria received Breakthrough Therapy designation from the FDA in May 2018 for ALK-positive ALCL. It has also been approved for that indication in more than 90 countries.

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New Drug Approvals

JANUARY 31, 2024

In the CPRC stage, disease is characterized by high AR expression, AR amplification and persistent activation of the AR signalling axis by residual tissue/tumor androgens and by other steroid hormones and intermediates of steroid biosynthesis. 71(20), 6503-6513, 2011). 7 H 8 O 3 S Molecular Weight 590.71 et al, Cancer Res.,

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The Pharma Data

APRIL 27, 2023

“Obstructive hypertrophic cardiomyopathy can be a debilitating and life-changing heart disease, reducing physical functioning and overall well-being, and often runs in families. Senior Vice President, Cardiovascular Development at Bristol Myers Squibb. “We

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Creative Biolabs

JUNE 20, 2022

There are already targeted therapies for the BRAF V600E mutation, such as vemurafenib, which was approved by the U.S. Food and Drug Administration (FDA) in 2011. The challenge is: for a given mutation, how can we rapidly translate this knowledge into more mechanistic understanding and genotype-driven approaches to cancer therapy?

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The Pharma Data

DECEMBER 16, 2020

Food and Drug Administration (FDA) has approved Benlysta to treat lupus nephritis (lupus-related kidney disease) in adults. The decision makes Benlysta the first lupus therapy authorized to treat this potentially life-threatening complication of the disease. The FDA first approved Benlysta for lupus in 2011.

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The Pharma Data

AUGUST 9, 2020

Etrolizumab failed to meet its primary endpoint versus placebo as maintenance therapy in people with ulcerative colitis. Pivotal phase III stud y of etrolizumab in Crohn’s disease is ongoing. Roche is studying additional investigational medicines in inflammatory bowel diseases.

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FDA Law Blog: Drug Discovery

FEBRUARY 28, 2024

This is the third withdrawal of an accelerated approval FDA has performed, following Avastin in 2011 and Makena in 2023. In contrast , the two previous withdrawals, Makena in 2023 and Avastin in 2011, took 30 months and 11 months, respectively.

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The Pharma Data

JANUARY 20, 2021

21, 2021 /PRNewswire/ — Dynacure , a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today the appointment of Jean M. STRASBOURG, France and PHILADELPHIA , Jan. Before these roles, Ms.

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Agency IQ

APRIL 12, 2024

Draft guidance on potency assays for CGT products garners extensive stakeholder input Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapy products, unveiling a major shift in approach to the issue. How does “potency” apply to cell and gene therapy (CGT) products?

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The Pharma Data

DECEMBER 17, 2020

Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. Goater is CEO at Surface Oncology, an immuno-oncology company developing next-generation antibody therapies.

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Broad Institute

JANUARY 14, 2025

By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. When I received my genetic test report in 2011, the world had never heard of base editing.

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The Pharma Data

NOVEMBER 5, 2020

Velders is presently chairman of HollandBIO, the association of Dutch biotech companies, and provides years of experience, specifically in the cell and gene therapy areas. Taveras will lead all research and non-clinical development functions supporting the company’s pipeline of investigational therapies.

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The Pharma Data

NOVEMBER 15, 2020

iSTAR Medical was founded in 2011 and is headquartered in Wavre, Belgium. About Glaucoma Glaucoma is a progressive disease and the second leading cause of adult blindness, 3 affecting over 100 million people globally. 3 IOP reduction, through medication or surgery, helps delay disease progression.

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New Drug Approvals

SEPTEMBER 13, 2024

2] It first appeared in the scientific literature in 2010 or 2011. [16] 19] As of 2016, aticaprant has reached phase II clinical trials as an augmentation to antidepressant therapy for treatment-resistant depression. [20] 2] It first appeared in the scientific literature in 2010 or 2011. [16] December 2011). nM vs. 24.0

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The Pharma Data

NOVEMBER 15, 2020

This prestigious group of professionals will help lead the Company as it prepares for potential commercialization of its investigational cell therapy, MultiStem ®. Prior to Celgene, she held leadership roles in marketing, sales and new business development from 2002 to 2011 at Johnson & Johnson. Before that, Ms. About MultiStem ®.

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Agency IQ

JUNE 7, 2024

Active medical devices fall into these categories: MD 1100 general active medical devices, MD 1200 devices for imaging, MD 1300 monitoring devices, MD 1400 devices for radiation and thermo therapy. Series AIMD 0100 describes all active implantable device types.

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Agency IQ

SEPTEMBER 15, 2023

This new body of data includes environmental exposure studies and clinical trials from Merck and Johnson and Johnson collected between 2011 and 2018. Since the 2007 NDAC meeting, several clinical trials have been conducted by industry to assess new phenylephrine doses and formulations—consistently failing to outperform placebo.

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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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Sygnature Discovery

APRIL 7, 2025

Since 2011, Sygnature Discovery has delivered 56 novel pre-clinical and 34 clinical compounds, with its scientists named on over 225 patent applications. Therapeutic areas of expertise include oncology, inflammation and immunology, neuroscience, metabolic diseases, infectious diseases, fibrotic diseases, and more.

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PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.

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Codon

APRIL 9, 2024

“Cumulatively the deadliest of the human infectious diseases,” malaria has been documented throughout recorded history. In this light, scouring old Chinese remedies for new treatments for a disease specifically troublesome during war was entirely intuitive for Chinese biomedical scientists like Tu Youyou in the mid-20th century.

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The Pharma Data

DECEMBER 17, 2020

Food and Drug Administration (FDA) has approved belimumab (Benlysta®), the first-ever treatment for adults with lupus nephritis (LN) who are currently receiving standard therapy. LN, or inflammation of the kidneys, is a common system of systemic lupus erythematosus (SLE), one of the most prevalent autoimmune diseases. MacroGenics Inc.

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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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Drug Target Review

SEPTEMBER 4, 2023

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

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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

NOVEMBER 10, 2020

According to data published on the WHO website, more than 46 million people have been tested positive for COVID-19, with over 1 million patients in more than 200 counties died from the disease as of November 2 nd , 2020. SHANGHAI , Nov.

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Agency IQ

SEPTEMBER 22, 2023

Since that time, the FDA’s use of single pivotal trials has become more common , especially due to FDA’s expanded reviews of products intended to treat, cure or prevent rare diseases or life-threatening conditions for which there is an unmet clinical need.

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The Pharma Data

DECEMBER 3, 2020

The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease. BEDFORD, Mass.–( –( BUSINESS WIRE )– Stoke Therapeutics , Inc. Chief Medical Officer of Stoke Therapeutics. Devices, Technologies, Stem Cells.

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Drug Target Review

JUNE 8, 2023

An important research area at AbbVie involves blood cancers, where our ultimate goal is to transform standards of care across these diseases and advance a dynamic cancer research and treatment pipeline. But we learn much from scientific dead-ends and always remain inspired, as the stakes are very high for those suffering from cancer.

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The Pharma Data

MAY 5, 2021

Farxiga use expanded for reducing risks in patients with chronic kidney disease. Farxiga received fast track, breakthrough therapy and priority review designations for the current indication. . controlled trial had not received previous systemic therapy for metastatic disease. Patients in the multicenter, double?blind,

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Common Sense for Drug Policy Blog

JANUARY 5, 2024

This estimate is more than 5 times the most recent US estimate of ∼774,000 from 2011 [ 25 ]. Much of this increase is likely attributable to increases in IDU, but it is important to consider methodological differences in the creation of this 2018 estimate vs the 2011 estimate. during the past decade.

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The Pharma Data

JUNE 26, 2021

Current guidelines are limited and recommend treating pediatric patients with or at risk for reoccurring blood clots with standard anticoagulant therapy, which often requires painful injections, dietary restrictions and regular laboratory monitoring. for use in pediatric patients. WHAT IS XARELTO ® (rivaroxaban)?

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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). SOUTH SAN FRANCISCO, Calif.,

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Agency IQ

JULY 22, 2024

As such, it doesn’t review things like vaccines, blood products or gene therapies – those products are instead reviewed by CBER. While the total number of novel drugs dipped to 37 in 2022, the agency recovered with 55 approvals in 2023, the second highest in a calendar year since 2011. [ population.

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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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The Pharma Data

JANUARY 6, 2021

In two exploratory cohorts, up to 100 patients with moderate Chronic Kidney Disease (CKD) and up to 100 patients with COVID-19 will be enrolled. No singular effective therapy to alter the progression of these devastating conditions has been approved, while the healthcare burden for sepsis in the US alone is $16.7 7, 2021 10:00 UTC.

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The Pharma Data

SEPTEMBER 8, 2021

Updated Phase 3 CheckMate -577 disease-free survival (DFS) results for adjuvant Opdivo treatment vs. placebo for patients with resected esophageal or gastroesophageal junction cancer following neoadjuvant chemoradiation therapy (CRT). On March 25, 2011, the U.S. Summary of Presentations. Early Development. Dr. Quincy Chu.

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