Medication Update

The article offers medical briefs. Topics include the authorization of a new drug called Gohibic for critically ill patients with COVID-19;the approval of an over-the-counter naloxone nasal spray for opioid overdose treatment;and the Food and Drug Administration approval of a long-acting antifungal called Rezzayo for the treatment of candidemia and invasive candidiasis for hypertension medications.

Study on the risk signal mining related to lopinavir/ritonavir based on the US FDA Adverse Event Reporting System.

Objective: To explore the clinical safety of lopinavir/ritonavir (LPV/r) by mining the risk signals of adverse events (AEs) related to LPV/r for the safe application of the drug in the treatment of novel coronavirus pneumonia (COVID-19). Method(s): The risk signals related to LPV/r in AE reports of US FDA Adverse Event Reporting System (FAERS) from the first quarter of 2010 to the third quarter of 2019 were mined by reporting odds ratio (ROR). An AE with reports more than 3 and 95% confidence interval (CI) lower limit of ROR greater than 1 was defined as a positive signal. AEs were counted and classified using the preferred system organ class (SOC) and preferred term (PT) of Medical Dictionary for Regulatory Activities (MedDRA). The PTs of top 50 adverse event reports and signal strength were selected and analyzed. Result(s): From the first quarter of 2010 to the third quarter of 2019, a total of 13 335 AE reports with LPV/r as the primary suspicious drug were reported in the FAERS database. Four hundred and fifty-five AE risk signals with reports more than 3 and the 95%CI lower limit of ROR greater than 1 were detected, involving 7 718 AE reports. The top 2 system organs involved in AE reports were "injury, poisoning and procedural complications" [13.6% (1 051/7 718)] and "pregnancy, puerperium and perinatal conditions" [11.7% (899/7 718)]. However, 998 (95.0%) of 1051 AE reports involved in "injury, poisoning and procedural complications" were related to drug exposure during pregnancy. The system organ with the highest signals was "congenital, familial and genetic disorders" [16.3% (74/455)]. In addition, 144 AEs caused by drug interactions were detected, which ranked the 7th in the AE reports. Conclusion(s): The risk signals of fetal, neonatal and infant abnormalities related to LPV/r during pregnancy were detected, suggesting that attention should be paid to the risk of using LPV/r in pregnant women and infants. The interaction between LPV/r and other drugs was also worthy of attention.Copyright © 2020 by the Chinese Medical Association.

Drug News

New HIV drug for experienced patients... New MS drug reduces relapse and brain lesions... Malaria vaccine shows promise... Antibiotic resistance in Western Pacific and South-East Asia... Leukemia pharmacotyping precision medicine blueprint... MIS-C not associated with serious adverse reactions from mRNA vaccines

FDA Streamlining COVID-19 Shot to a Single Formula

The article offers information on how U.S. Food and Drug Administration is streamlining COVID-19 vaccine to a single formula. Topics include information on how FDA's Vaccines and Related Biological Products Advisory Committee (VRBPAC) has voted unanimously to simplify the vaccine process;how multiple vaccine doses and boosters have caused confusion and pandemic apathy;and importance of vaccines.

Pandemic reveals new concerns about facial protection products: Design may be the answer to altering behaviors, habits ushered into standard practice

The article examines what medical suppliers and providers learned about facial protection products after the Covid-19 pandemic of 2020-2022. Topics discussed include remarks from Jason Burnham, Senior Director of Facial Protection at Owens & Minor, evidence of pandemic-relaxed behaviors morphing into workflow acceptance, and statement from Gary Harris, Vice President of Sales and Marketing at Prestige Ameritech, about emergency use guidelines for personal protective equipment (PPE).

Starting the conversation on gene therapy for phenylketonuria: Current perspectives of patients, caregivers, and advocates.

Phenylketonuria (PKU) is a rare genetic condition caused by inborn error(s) in the gene for the enzyme phenylalanine hydroxylase. Resulting loss of phenylalanine (Phe) metabolism requires strict dietary therapy and/or medication to prevent toxic accumulation of Phe. Novel investigational therapies, including gene therapies that aim to address underlying causes of PKU, are now entering clinical trials. However, perceptions of this technology in the PKU community have not been assessed. We conducted a qualitative survey recruiting adult patients, caregivers, and patient advocates from the US and 3 EU countries to assess the impact of living with PKU and the perceptions of gene therapy. Telephone interviews were conducted for up to 60 min following a standardized discussion guide. Interviewers classified each participant by their level of knowledge regarding gene therapy as either: low (little or no prior awareness); moderate (awareness of gene therapy as a concept in PKU); or high (working knowledge of gene therapy, e.g., vectors). In total, 33 participants were recruited (patients, n = 24; caregivers, n = 5; advocates, n = 4). The patient sample was well balanced among age groups, sex, and US/EU geographies. The participants' experiences and burden of living with PKU were largely negative, characterized by frustrations with current management consistent with prior reports. Most participants (n = 18/33) were identified as displaying moderate gene-therapy knowledge, 10/33 as displaying high knowledge, and 5/33 as displaying low knowledge. Both positive and negative perceptions were observed; positive perceptions were often linked to "hope" that gene therapy may represent a cure, whereas negative perceptions were linked to the "uncertainty" of outcomes. High knowledge of gene therapy appeared to trend with negative perceptions; 7/10 participants from this group reported high levels of concern over gene therapy. In contrast, participants who displayed low knowledge reported low (n = 3/5) or moderate (n = 2/5) concern, with predominantly positive perceptions. These data highlight the need for education around the theoretical risk:benefit profile of gene therapy. Despite current unknowns around gene therapy, our study demonstrates the important role of healthcare providers as educators who can use available data to provide balanced information to patients and caregivers.

FDA Coronavirus Treatment Acceleration Program: approved drugs and those in clinical trials

Coronavirus disease is a highly infectious disease caused by the 2019 novel coronavirus, now recognized as severe acute respiratory syndrome coronavirus 2. This pandemic caused great stress in the general public and led to the collapse of healthcare system in some places. Till date, there has not been an effective vaccine or antiviral drug for its treatment. As at the time of write-up, scientists, researchers, and industries in different countries are working collaboratively to develop an effective therapy or alternative to combat this deadly and highly infectious virus. To combat or overcome these difficulties, the United States Food and Drug Administration launched an FDA Coronavirus Treatment Acceleration Program (CTAP) to accelerate the development of new investigational drugs, biological therapies, vaccines, and medical devices for the treatment of COVID-19 patients. This chapter provides details about this, the role of FDA CTAP and the drugs they are evaluating for the treatment of this disease. © 2022 Elsevier Inc. All rights reserved.

Single domain antibodies derived from ancient animals as broadly neutralizing agents for SARS-CoV-2 and other coronaviruses.

With severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) as an emergent human virus since December 2019, the world population is susceptible to coronavirus disease 2019 (COVID-19). SARS-CoV-2 has higher transmissibility than the previous coronaviruses, associated by the ribonucleic acid (RNA) virus nature with high mutation rate, caused SARS-CoV-2 variants to arise while circulating worldwide. Neutralizing antibodies are identified as immediate and direct-acting therapeutic against COVID-19. Single-domain antibodies (sdAbs), as small biomolecules with non-complex structure and intrinsic stability, can acquire antigen-binding capabilities comparable to conventional antibodies, which serve as an attractive neutralizing solution. SARS-CoV-2 spike protein attaches to human angiotensin-converting enzyme 2 (ACE2) receptor on lung epithelial cells to initiate viral infection, serves as potential therapeutic target. sdAbs have shown broad neutralization towards SARS-CoV-2 with various mutations, effectively stop and prevent infection while efficiently block mutational escape. In addition, sdAbs can be developed into multivalent antibodies or inhaled biotherapeutics against COVID-19.

The Role of Blind Justice in U.S. Supreme Court Decisions on Health Care

The article discusses the U.S. Supreme Court's conferment with judicial review and authority on the constitutionality of a law to maintain the balance among the executive, legislative, and judiciary branches of government. Topics covered include its rulings on the coronavirus disease 2019 (COVID-19), societal demands, and personal liberties issues, and the historical moderation of judges' views based on their own biases. Also noted are cases that have had different legal interpretations.

Medication Update

The article offers several medical news briefs. Topics include considered that the FDA approved Mounjaro (tirzepatide) injection to improve blood glucose control in adults with type 2 diabetes mellitus as an addition to diet and exercise;and examines that the FDA approved the use of Qelbree for adults 18 years of age and older with attention deficit hyperactivity disorder (ADHD).

AJN On the Cover

The article focuses on blood donated by blood donor Rich Patternson at ImpactLife in Cedar Rapids, Iowa and mentions blood shortage during pandemic experience by U.S.

A Proposal to Increase Value and Equity in the Development and Distribution of New Pharmaceuticals.

The process of developing and marketing new pharmaceuticals in the United States is driven by a need to maximize returns to shareholders. This results all too often in the production of new medications that are expensive and of marginal value to patients and society. In line with our heightened awareness of the importance of social justice and public health-and in light of our government's alliance with private companies in bringing us COVID-19 vaccines-we need to reconsider how new pharmaceuticals are developed and distributed. Accordingly, we propose the creation of a new agency of the Food and Drug Administration (FDA) that would direct the whole process. This agency would fund the research and development of high-value medications, closely monitor the clinical studies of these new drugs, and manage their distribution at prices that are value-based, fair, and equitable.