Drug approval
View article: History of Erythropoiesis-Stimulating Agents, the Development of Biosimilars, and the Future of Anemia Treatment in Nephrology
History of Erythropoiesis-Stimulating Agents, the Development of Biosimilars, and the Future of Anemia Treatment in Nephrology Open
Background: Exogenous replacement of erythropoietin (EPO) by recombinant human EPO has been considered a standard of care for the treatment of anemia in patients with chronic kidney disease for more than 20 years. Genetically engineered bi…
View article
Identification of Antiviral Drug Candidates against SARS-CoV-2 from FDA-Approved Drugs Open
Drug repositioning is the only feasible option to immediately address the COVID-19 global challenge. We screened a panel of 48 FDA-approved drugs against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) which were preselected b…
View article
Antibodies to watch in 2021 Open
In this 12th annual installment of the Antibodies to Watch article series, we discuss key events in antibody therapeutics development that occurred in 2020 and forecast events that might occur in 2021. The coronavirus disease 2019 (COVID-1…
View article
FDA Approval Summary: Sotorasib for <i>KRAS G12C</i> -Mutated Metastatic NSCLC Open
On May 28, 2021, the FDA granted accelerated approval to sotorasib (Lumakras, Amgen) for the treatment of adults with advanced non–small cell lung cancer (NSCLC) with a Kirsten rat sarcoma proto-oncogene (KRAS) G12C mutation who have recei…
View article
Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval Open
Confirmatory trials for one-fifth (n = 19 of 93) of cancer drug indications approved via the FDA's accelerated approval pathway demonstrated improvements in overall patient survival. Reassessment of the requirements for confirmatory trials…
View article
An Insight into FDA Approved Antibody-Drug Conjugates for Cancer Therapy Open
The large number of emerging antibody-drug conjugates (ADCs) for cancer therapy has resulted in a significant market ‘boom’, garnering worldwide attention. Despite ADCs presenting huge challenges to researchers, particularly regarding the …
View article
Contribution of NIH funding to new drug approvals 2010–2016 Open
Significance This report shows that NIH funding contributed to published research associated with every one of the 210 new drugs approved by the Food and Drug Administration from 2010–2016. Collectively, this research involved >200,000 yea…
View article
FDA Approval of Remdesivir — A Step in the Right Direction Open
secretary of health and human services declared a public health emergency in response to Covid-19.This disease, caused by the SARS-CoV-2 virus, can have severe manifestations, including pneumonia, respiratory failure, multiorgan failure, a…
View article
Sotorasib: First Approval Open
Sotorasib (LUMAKRAS™) is a RAS GTPase family inhibitor being developed by Amgen for the treatment of solid tumours with KRAS mutations, including non-small cell lung cancer (NSCLC) and colorectal cancer. In May 2021, sotorasib was granted …
View article
Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014 Open
Introduction The efficacy of pharmaceuticals is most often demonstrated by randomised controlled trials (RCTs); however, in some cases, regulatory applications lack RCT evidence. Objective To investigate the number and type of these approv…
View article
Revisiting FDA Approval of Aducanumab Open
Interview with Dr. Gil Rabinovici on the FDA's controversial approval of a new treatment for Alzheimer's disease. (16:39)Download Given the scientific, regulatory, and clinical implications of the accelerated FDA approval of aducanumab for…
View article
Potential harmful effects of discontinuing ACE-inhibitors and ARBs in COVID-19 patients Open
The discovery of angiotensin converting enzyme-2 (ACE-2) as the receptor for SARS- CoV-2 (Severe Acute Respiratory Syndrome Coronavirus-2) has implicated the renin-angiotensin-aldosterone system in acute respiratory distress syndrome (ARDS…
View article
The Role of Real‐World Evidence in FDA‐Approved New Drug and Biologics License Applications Open
The US Food and Drug Administration (FDA) is open to accepting real‐world evidence (RWE) to support its assessment of medical products. However, RWE stakeholders lack a shared understanding of FDA’s evidentiary expectations for the use of …
View article
The Pharmaceutical Industry in 2019. An Analysis of FDA Drug Approvals from the Perspective of Molecules Open
During 2019, the US Food and Drug Administration (FDA) approved 48 new drugs (38 New Chemical Entities and 10 Biologics). Although this figure is slightly lower than that registered in 2018 (59 divided between 42 New Chemical Entities and …
View article
Identification of antiviral drug candidates against SARS-CoV-2 from FDA-approved drugs Open
COVID-19 is an emerging infectious disease and was recently declared as a pandemic by WHO. Currently, there is no vaccine or therapeutic available for this disease. Drug repositioning represents the only feasible option to address this glo…
View article
Clinical Perspective of FDA Approved Drugs With P-Glycoprotein Inhibition Activities for Potential Cancer Therapeutics Open
P-glycoprotein (also known as multidrug resistance protein 1 (MDR1) or ATP-binding cassette sub-family B member 1 (ABCB1) plays a crucial role in determining response against medications, including cancer therapeutics. It is now well estab…
View article
The Pharmaceutical Industry in 2020. An Analysis of FDA Drug Approvals from the Perspective of Molecules Open
Although the pharmaceutical industry will remember 2020 as the year of COVID-19, it is important to highlight that this year has been the second-best—together with 1996—in terms of the number of drugs accepted by the US Food and Drug Admin…
View article
Current Status of Companion and Complementary Diagnostics: Strategic Considerations for Development and Launch Open
US Food and Drug Administration (FDA)‐approved diagnostic assays play an increasingly common role in managing patients to prolong lifespan while also enhancing quality of life. Diagnostic assays can be essential for the safe and effective …
View article
FDA Acceptance of Surrogate End Points for Cancer Drug Approval: 1992-2019 Open
This retrospective review assesses the frequency of surrogate measures used for the first time vs subsequent times in a cancer setting and the surrogate’s strength of correlation with patient-centered outcomes.
View article
Abrocitinib: First Approval Open
DeclarationsFunding The preparation of this review was not supported by any external funding.Authorship and Conflict of interest During the peer review process the manufacturer of the agent under review was offered an opportunity to commen…
View article
Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017 Open
In this study, more recent FDA approvals of new drugs and biologics were based on fewer pivotal trials, which, when aggregated by indication, had less rigorous designs but longer trial durations, suggesting an ongoing need for continued ev…
View article
How Much Longer Will We Put Up With $100,000 Cancer Drugs? Open
The spiraling cost of new drugs mandates a fundamentally different approach to keep lifesaving therapies affordable for cancer patients. We call here for the formation of new relationships between academic drug discovery centers and commer…
View article
Regulatory and clinical consequences of negative confirmatory trials of accelerated approval cancer drugs: retrospective observational study Open
Objectives To investigate the regulatory handling of cancer drugs that were granted accelerated approval by the US Food and Drug Administration (FDA) but failed to improve the primary endpoint in post-approval trials and to evaluate the ex…
View article
Pharmaceutical Approval Update. Open
Coagulation factor IX (recombinant), albumin fusion protein (Idelvion) for hemophilia B; captisol-enabled melphalan hydrochloride (Evomela) for multiple myeloma; and antihemophilic factor (recombinant) (Kovaltry) for hemophilia A.
View article
Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study Open
Objective To characterize the therapeutic value of new drugs approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) and the association between these ratings and regulatory approval through expedited prog…
View article
Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review Open
Objectives Non-randomised clinical trial designs involving comparisons against external controls or specific standards can be used to support regulatory submissions for indications in diseases that are rare, with high unmet need, without a…
View article
What Do We Know About Remdesivir Drug Interactions? Open
The global pandemic of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has resulted in a critical need to rapidly develop new pharmacologic interventions and disseminate information. This has led to confusing and conflicting i…
View article
Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy Open
Background Over the past decades, US Congress enabled the US Food and Drug Administration (FDA) to facilitate and expedite drug development for serious conditions filling unmet medical needs with five special designations and review pathwa…
View article
A Comparison of <span>EMA</span> and <span>FDA</span> Decisions for New Drug Marketing Applications 2014–2016: Concordance, Discordance, and Why Open
The Food and Drug Administration ( FDA ) and the European Medicines Agency ( EMA ) have robust scientific and technical collaborations. As a window to the impact of these activities we compared the agencies’ decisions on drug marketing app…
View article
2020 FDA TIDES (Peptides and Oligonucleotides) Harvest Open
2020 has been an extremely difficult and challenging year as a result of the coronavirus disease 2019 (COVID-19) pandemic and one in which most efforts have been channeled into tackling the global health crisis. The US Food and Drug Admini…