BioPharma Drive: Drug Pricing

MARCH 24, 2025

The company expects next quarter to read out initial data from its Heart-2 trial, which is testing a one-time treatment for people with genetically elevated cholesterol.

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BioPharma Drive: Drug Pricing

SEPTEMBER 27, 2023

Expert advisers to the agency voted 17-1 that Brainstorm's clinical trial data did not show the company's stem cell treatment was effective for treating ALS.

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BioPharma Drive: Drug Pricing

AUGUST 8, 2024

Analysts at Evercore ISI called pegcetacoplan’s data in two kidney conditions a “left-field hit” that compare favorably to results for a Novartis treatment.

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New Drug Approvals

APRIL 11, 2025

2] The label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran. [2] The FDA granted the approval of Qfitlia to Sanofi. Fitusiran 1711.0g/mol, Fitusiran 1711.0g/mol,

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Clinical Trials FDA FDA Approval Trials 106

Science Daily: Pharmacology News

AUGUST 17, 2023

A single injection of a novel CRISPR gene-editing treatment safely and efficiently removes SIV -- a virus related to the AIDS-causing agent HIV -- from the genomes of non-human primates, scientists now report.

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Virus Treatment Clinical Trials FDA 189

Antidote

SEPTEMBER 25, 2023

Clinical trials are how researchers advance their knowledge about potential new treatments, including medications, medical devices, and lifestyle interventions. Clinical trials are divided into phases , each with a distinct duration, purpose, and number of volunteers needed.

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Clinical Trials Trials FDA Treatment 128

BioPharma Drive: Drug Pricing

APRIL 29, 2024

The company will soon start a Phase 1/2 study of its treatment for chronic granulomatous disease, a milestone for the search-and-replace gene editing technology.

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Trials FDA Treatment Disease 157

Drug Target Review

APRIL 7, 2025

A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms.

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Clinical Development Clinical Trials Clinical Pharmacology Trials 59

BioPharma Drive: Drug Pricing

DECEMBER 22, 2023

The biotech is asking the agency to clear its treatment Elevidys in more patients with the disease, despite a confirmatory trial that missed its main goal.

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FDA Trials Treatment Disease 176

Conversations in Drug Development Trends

AUGUST 23, 2023

Patients are the backbone of clinical trials, playing an essential role in the drug development process. However, patients also play a vital role in engaging directly with the FDA. Her fellow panelists represented a diverse group of stakeholders across clinical research, including patient advocates, sponsors, sites, and the FDA itself.

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Drug Target Review

NOVEMBER 25, 2024

As the CEO of iOnctura, an innovative oncology biopharmaceutical company she co-founded in 2017, Catherine has played a key role in advancing the development of highly targeted small molecules aimed at revolutionising cancer treatment. Additionally, we are preparing to launch several other trials over the coming months.

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Treatment Small Molecule Clinical Trials Trials 52

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis.

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FDA Approval Treatment FDA DNA 96

FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. By Sarah Wicks & James E. Valentine & Frank J. those reviewed by the CDER Division of Rare Diseases and Medical Genetics).

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FDA Disease Clinical Pharmacology Science 105

New Drug Approvals

APRIL 2, 2025

2] [6] Suzetrigine is the first medication to be approved by the US Food and Drug Administration (FDA) in this new class of pain management medicines. [2] 2] Medical uses Suzetrigine is indicated for the treatment of moderate to severe acute pain in adults. [1] The FDA has long supported development of non-opioid pain treatment.

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FDA Therapies Trials Treatment 62

National Institute on Drug Abuse: Nora's Blog

MARCH 6, 2025

Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&

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Treatment Trials Clinical Trials Drugs 69

Drug Discovery Today

NOVEMBER 7, 2023

The trial showed that KT-110 has superior efficacy in reducing alcohol consumption. Kinnov Therapeutics expects to have clear guidance from the FDA in early 2024 on the phase III trial KT-110 will be deliverable in a once-daily tablet (proprietary formulation).

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Treatment Trials FDA 113

PPD

JUNE 7, 2023

As therapies for the treatment of NASH in adult patients go this year to the U.S. Food and Drug Administration (FDA) for approval , much work remains for drug developers aiming to advance treatments for pediatric populations. Partnership with an experienced CRO is essential to your trial’s success. Hispanic: 11.8%

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Trials Clinical Trials Clinical Research Disease 98

Conversations in Drug Development Trends

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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FDA Law Blog: Drug Discovery

MARCH 7, 2024

Clissold — The trio of CDER, CBER, and CDRH released a new draft guidance titled “ Use of Data Monitoring Committees in Clinical Trials ” that revises the 2006 guidance “Establishment and Operation of Clinical Trial Data Monitoring Committees” and, when final, will replace the 2006 guidance.

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Clinical Trials Trials FDA Regulations 105

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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Therapies FDA Clinical Trials Disease 105

PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

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Clinical Trials Trials Clinical Research Disease 98

Conversations in Drug Development Trends

AUGUST 9, 2024

Authors: Matt Cooper, PhD, Executive Director, Therapeutic Strategy Lead, Oncology; Megan Morrison, Vice President, Asia Pacific Strategy Lead Adaptive trial designs have become essential in oncology, offering a flexible and efficient approach for conducting clinical trials.

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Trials Clinical Trials Regulations FDA 80

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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ProRelix Research

NOVEMBER 5, 2024

Clinical trials are necessary in advancing medical research and health care options by allowing new treatments to be introduced in the market through safety and efficacy testing in humans. Successful […] The post Clinical Trials and Human Subject Protection as per US FDA appeared first on ProRelix Research.

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Clinical Trials FDA Trials Treatment 52

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.

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Small Molecule Drugs Clinical Pharmacology Trials 145

ProRelix Research

NOVEMBER 23, 2022

Biological products have unlocked the potential for the management of several diseases such as cancers and autoimmune diseases for which treatment with small molecule, chemically synthesized drug molecules remain suboptimal. […]. The post Biosimilar Clinical Trials and US FDA Guidance appeared first on ProRelix Research.

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Clinical Trials Biosimilars FDA Trials 52

Alta Sciences

MARCH 4, 2025

This was in 2006, at a time when the FDA guidances on these topics had not yet been published. Over time, this group of professionals evolved and grew, having regular stakeholder interactions with the FDA and Controlled Substances staff to discuss requirements for drug developers. corticosteroids, beta-blockers, antidepressants).

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Clinical Trials FDA Drug Development Trials 52

Conversations in Drug Development Trends

JANUARY 21, 2025

Regulatory bodies such as the FDA oversee clinical trials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. Any delays or missteps in bioanalysis during a Phase I trial can derail the trajectory of a promising drug.

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The Premier Consulting Blog

OCTOBER 21, 2024

The FDA defines a serious condition as “a disease or condition associated with morbidity that has a substantial impact on day-to-day functioning.” Concerns have arisen over delays—sometimes spanning over 7–8 years—that may expose patients to risks before confirmatory trials are completed.

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The Pharma Data

AUGUST 31, 2021

Food and Drug Administration (FDA) has converted this indication from an accelerated to a full (regular) approval. Previously, KEYTRUDA was indicated for the treatment of patients with locally advanced or mUC who were not eligible for cisplatin-containing chemotherapy and whose tumors expressed PD-L1 (Combined Positive Score [CPS] ?10),

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FDA Approval Treatment FDA Research Laboratories 52

Conversations in Drug Development Trends

NOVEMBER 5, 2024

First, the beginning of the FDA’s START program, with the goal of accelerating the development of novel drugs and biological products for rare diseases. Selected sponsors will be able to obtain frequent advice and enhanced communication from FDA staff to address program-specific development issues within rare disease.

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Disease Clinical Trials Therapies Clinical Research 52

The Pharma Data

OCTOBER 29, 2021

Novartis blazoned moment that the US Food and Drug Administration (FDA) approved Scemblix ® (asciminib) for the treatment of habitual myeloid leukemia (CML) in two distinct suggestions. In cases with Ph CML-CP who had endured resistance or dogmatism to at least two TKIs, the ASCEMBL trial showed that1-3.Scemblix

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FDA Approval Treatment FDA Trials 52

The Pharma Data

MAY 16, 2022

Food and Drug Administration (FDA) has lifted the clinical hold placed on the company’s Investigational New Drug Application (IND) to evaluate injectable lenacapavir for HIV treatment and HIV pre-exposure prophylaxis (PrEP). (Nasdaq: GILD) today announced the U.S. There is no cure for HIV or AIDS. About Lenacapavir.

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Treatment FDA Science Therapies 52

PPD

SEPTEMBER 16, 2024

This can be converted to a clinical trial simulator, which can model a clinical trial before the trial design is finalized and initiated. AI approaches are optimizing trial designs With a significant increase in the number of regulatory submissions to the U.S.

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Clinical Development Clinical Trials Clinical Research Trials 98

Vial

JUNE 12, 2024

PROs in clinical trials are important as they capture the patient’s perspective and ensure that the impact of an intervention is comprehensively evaluated. Food and Drug Administration (FDA) increasingly look to patients to understand how they describe their health status. What are PROs in clinical trials?

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Clinical Trials Trials FDA Clinical Research 52

The Pharma Data

JUNE 2, 2023

Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA. In the U.S.,

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FDA Approval FDA Therapies Treatment 52

H1 Blog

JULY 19, 2023

Food and Drug Administration (FDA) issued draft guidance, Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics , regarding clinical trial design specific to oncology with considerations to support accelerated approval applications.

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Clinical Trials Trials FDA Treatment 52