The Pharma Data

NOVEMBER 16, 2020

and the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health. principal investigator of the NIAID-funded Infectious Diseases Clinical Research Consortium site at Baylor College of Medicine in Houston; Lindsey R. El Sahly, M.D., Baden, M.D.,

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Drug Target Review

FEBRUARY 21, 2024

This is a time when several promising treatments – including mRNA vaccines, BiTE therapies and CAR-T cell therapy, are essentially in competition with each other – they all have a common goal of treating the same disease, but they are approaching the objective from different angles.

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Advarra

NOVEMBER 17, 2022

In 2016, the Food and Drug Administration (FDA) approved Spinraza (nusinersen). It was the first drug to treat children with spinal muscular atrophy (SMA), a disease affecting children’s muscular strength and movement. While the FDA’s approval of nusinersen may not seem extraordinary, it was. Why This Guidance Now?

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Advarra

JULY 5, 2022

Individuals invest a great deal to participate in clinical research, undertaking risks and burdens, while giving of their time and energies to advance scientific knowledge and the public good. Notably, participants may have an interest in receiving results even if they are not clinically actionable.

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The Pharma Data

JANUARY 22, 2021

22, 2021 /PRNewswire/ — The Lupus Research Alliance (LRA) is excited to share that the U.S. Despite treatment, for as many as 30 percent, lupus nephritis worsens to cause end-stage renal disease when the kidneys can no longer function properly, and dialysis or an organ transplant is required. . NEW YORK , Jan.

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The Pharma Data

MARCH 26, 2021

Global Therapeutic Area Head, Neuroscience, Janssen Research & Development, LLC. If approved, ponesimod will be the first therapy by Janssen for patients living with RMS with active disease defined by clinical or imaging features. About Ponesimod. About Multiple Sclerosis.

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The Pharma Data

MARCH 23, 2023

Dupixent is the first and only biologic to demonstrate a clinically meaningful and highly significant reduction (30%) in moderate or severe acute exacerbations of COPD (rapid and acute worsening of respiratory symptoms), while also demonstrating significant improvements in lung function, quality of life and COPD respiratory symptoms.

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The Pharma Data

MARCH 11, 2021

Session Category: Clinical Research (Excluding Trials). Session Title: Disease-Oriented Innovative Clinical Research and Trials. Session Type: Clinical Trials Minisymposium. FDA-approved oral prescription medicine, 120 mg or 160 mg dependent on weight (<50 kg or ?50 Sintilimab.

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The Pharma Data

OCTOBER 22, 2020

October 22, 2020 — In less than one year, the world has moved from a sense of desperation to understand an unknown disease spreading all too quickly and taking lives, to one of hope that we now have enough knowledge and a growing set of tools to help fight COVID-19.

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Agency IQ

NOVEMBER 3, 2023

In simpler terms, these are live microbes that have actually demonstrated health benefits in clinical research. Keep in mind that this is the definition developed by ISAPP – there is no regulatory body confirming that any product on the market referred to as a “probiotic” has actually demonstrated benefits in clinical research.

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The Anticancer Fund

JUNE 23, 2023

Among 16 patients evaluable for best response, there were one complete response (Ewing sarcoma), three partial responses (Ewing sarcoma, glioblastoma multiforme, and alveolar rhabdomyosarcoma), and five patients with stable disease. Primary outcome was PFS, with OS and disease control and response rate as secondary outcomes.

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The Pharma Data

JUNE 24, 2021

“With the introduction of PARP inhibitors in the metastatic castration-resistant prostate cancer setting, it is important to explore how a combination approach may impact outcomes for men with metastatic castration-sensitive disease,” said Neeraj Agarwal, M.D., Talazoparib is not approved for the treatment of prostate cancer.

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The Pharma Data

JANUARY 24, 2021

Sairiyo is a biotechnology company focused on repurposing and developing improved formulations of naturally derived compounds for serious, rare, and life-threatening diseases with the aim to obtain European Medicines Evaluation Agency and U.S. Food and Drug Administration (“FDA”) approval.

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The Pharma Data

SEPTEMBER 1, 2020

study investigator and Professor of Clinical Neurology, University of California San Francisco (UCSF) Weill Institute for Neurosciences and clinical research director, UCSF MS Center. Presentation Topic: Disease Modifying Therapies – Risk Management. Presentation Topic: Disease Modifying Therapies – Mechanism of Action.

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NIH Director's Blog: Drug Development

JULY 20, 2017

Credit: Brittany Mahoney As NIH Director, I often hear stories of how people with serious diseases—from arthritis to Zika infection—are benefitting from the transformational power of NIH’s investments in basic science. According to preliminary data from one Phase 1 and two Phase 2 clinical trials reported by Vertex Pharmaceuticals Inc.,

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The Pharma Data

JULY 22, 2021

Immunotherapy and Tyrosine Kinase Inhibitor Combination Approved for the Treatment of Patients With Advanced Endometrial Carcinoma That is Not Microsatellite Instability-High or Mismatch Repair Deficient, Who Have Disease Progression Following Prior Systemic Therapy in Any Setting and Are Not Candidates for Curative Surgery or Radiation.

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The Pharma Data

NOVEMBER 16, 2020

and is being conducted in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), and the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S.

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The Pharma Data

JULY 29, 2021

Launch activities continue following the full FDA approval for second-line metastatic triple-negative breast cancer (“mTNBC”) and accelerated approval for metastatic urothelial cancer. Viral Diseases. Importantly, the majority of these responses were associated with undetectable minimal residual disease.

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NIH Director's Blog: Drug Development

FEBRUARY 16, 2021

The program relies on four expert “working groups” with specific charges: Preclinical Working Group : Shares standardized preclinical evaluation resources and accelerate testing of candidate therapies and vaccines for clinical trials. Our work is clearly a marathon, not a sprint.

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Vial

JUNE 12, 2024

Utilizing PROs enables the active participation of patients in their care outcomes – both in a clinical setting and in clinical research. PROs in clinical trials are important as they capture the patient’s perspective and ensure that the impact of an intervention is comprehensively evaluated. For HF, Savarese et al.

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The Pharma Data

JULY 8, 2021

This approval is based on data from the second interim analysis of the Phase 2 KEYNOTE-629 trial, in which KEYTRUDA demonstrated an objective response rate (ORR) of 50% (95% CI, 36-64) (n=54), including a complete response rate of 17% and a partial response rate of 33% in the cohort of patients with locally advanced disease.

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PPD

AUGUST 5, 2024

Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. The clinical sites working on these trials are highly experienced and have a deep understanding of the complexities of successfully running PAH clinical trials. None offers a cure for PAH.

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KIF1A

JULY 1, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. But drug R&D is an uncertain process, and many therapeutics get shelved during preclinical or clinical research, before they ever reach a patient.

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Conversations in Drug Development Trends

MAY 30, 2024

This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. The fight against ALS is a testament to human resilience and the relentless pursuit of scientific advancement.

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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. Research through WGS and WES has provided novel insights into biological processes. Fountzilas et al.

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Advarra

AUGUST 8, 2023

As of June 2023, 416 Phase III studies are listed, representing a growing pipeline of gene therapy products preparing to undergo consideration for FDA approval. Risks and Regulatory Requirements Most clinical researchers are familiar with the regulatory requirements pertaining to the FDA phases of review as well as with IRB review.

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The Pharma Data

JULY 6, 2021

accelerated approval indication for KEYTRUDA for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 [combined positive score (CPS ?1)] Merck has the industry’s largest immuno-oncology clinical research program.

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Drug Target Review

AUGUST 3, 2023

Clinical trials are now underway for various genetic subtypes of both rare and common diseases, on the understanding that medications utilising genetic biomarkers have a significantly higher chance of success. There are clinical trials underway for genetic subtypes of rare and common diseases. That needs to change.

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The Pharma Data

SEPTEMBER 8, 2020

Phase IIIb clinical trial programme of higher-dose OCREVUS (ocrelizumab) to evaluate impact on reducing disability progression in RMS and PPMS. OCREVUS CHIMES study exclusively focused on disease insights and more tailored care for minority populations with MS. and more than 2.3 million people worldwide.

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The Pharma Data

MARCH 30, 2021

“KEYTRUDA has become an important treatment option for certain patients with locally advanced or metastatic bladder cancer in the European Union and other countries around the world,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “We 1] as determined by an FDA-approved test.

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The Anticancer Fund

OCTOBER 18, 2023

Best response was partial response in one patient, and stable disease in 11 (36%) patients. The authors report that of the 5 cases, four showed clinical benefit, including one complete response, one partial response and two stable disease. Adverse events were manageable and reversible.

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The Pharma Data

MARCH 23, 2021

The approval is based on results from the Phase 3 KEYNOTE-590 trial, which demonstrated significant improvements in overall survival (OS), progression-free survival (PFS) and objective response rate (ORR) for KEYTRUDA plus fluorouracil (FU) and cisplatin versus FU and cisplatin alone, regardless of histology or PD-L1 expression status.

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The Pharma Data

APRIL 4, 2023

Patients were treated until disease progression or unacceptable toxicity. Thirty-seven percent of patients had upper tract disease. Merck has the industry’s largest immuno-oncology clinical research program. A total of 121 patients received KEYTRUDA in combination with enfortumab vedotin. At baseline, 97.5%

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The Pharma Data

JULY 15, 2021

0.82]; p=0.00031) versus the chemotherapy-placebo regimen – a statistically significant and clinically meaningful EFS result. EFS was defined as the time from randomization to the first occurrence of either disease progression that precluded definitive surgery, a local/distant recurrence, a second primary cancer, or death from any cause.

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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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The Pharma Data

NOVEMBER 30, 2020

and is being conducted in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), and the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S.

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Agency IQ

OCTOBER 27, 2023

12/29/2023 FDORA, Section 3607 Clinical Trials Modernization : FDA must issue or revise draft guidance regarding “the use of seamless, concurrent, and other innovative clinical trial designs.” Within 180 days, FDA will release a report on the proceedings of the public meeting and any recommendations.

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