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The evolving role of liver transplantation as enzyme replacement therapy in the era of RNA nanotherapies.

Solid organ transplantation (SOT) is a treatment for monogenic diseases like transthyretin amyloidosis (ATTR) and primary hyperoxaluria (PH). The advent of RNA nanotherapy has introduced new disease-modifying options for ATTR and PH. We examined the prevalence, clinical characteristics, and outcomes of SOT waitlisted candidates (due to ATTR/PH) pre-US Food and Drug Administration (US FDA) and post-US FDA approval of patisiran and lumasiran, respectively. We used the Scientific Registry of Transplant Recipients/United Network of Organ Sharing database, including all waitlisted patients for SOT

A Developer's Perspective on Clinical Evidence and Benefits for Rituximab Biosimilar Uptake, with a Focus on CT-P10.

To date, four rituximab biosimilars have received regulatory approval from the European Medicines Agency and/or US Food and Drug Administration. CT-P10 was the first rituximab biosimilar to be approved by each agency, in 2017 and 2018, respectively. Regulatory approval of CT-P10 followed demonstration of pharmacokinetic equivalence to the reference product in a phase I study in patients with rheumatoid arthritis. Phase III pivotal studies of CT-P10 subsequently demonstrated equivalence or non-inferiority of pharmacokinetics and efficacy between CT-P10 and reference rituximab in patients with r

Innovative Targets For Asthma And COPD: Exploring The Existing And Screening The New!!

The inadequate benefits of the existing therapies and the new insights into the pathophysiology of inflammatory airway diseases like chronic obstructive pulmonary disease (COPD) and asthma have led to the breakthrough of newer targets and innovative compounds as the treatment alternatives. The enhanced interpretation of immune cell signalling and signal transduction pathways at the molecular level involved in this process allows the selection of new therapeutic targets and designing of new molecules to combat such multifactorial diseases. Pertaining to the marked variability in type of inflamm

Management of obesity as a chronic disease: nonpharmacologic, pharmacologic, and surgical options.

The successful management of obesity requires a long-term approach that is tailored to an individual's lifestyle and needs. Initial treatment should focus on lifestyle modifications-dietary interventions and increased physical activity-with behavioral modification strategies used adjunctively. Several antiobesity drugs are approved by the Food and Drug Administration for use in obese patients, as well as in overweight individuals with at least one obesity-related comorbidity. Most are approved only for short-term weight loss, but sibutramine and orlistat are approved for long-term weight loss

Review of embryo-fetal developmental toxicity studies performed for pharmaceuticals approved by FDA in 2024 and 2025.

The FDA approved 96 novel drugs between 2024 and 2025. Of these approvals, 49 were designated for orphan indications. EFD studies were conducted for 78% of approvals. Rats and rabbits were the most common species used (48% and 41% of studies, respectively). For the testing of biopharmaceuticals, non-human primates (40% of EFD studies), rodents (33%) and rabbits (27%) were all used. Most (75%) biopharmaceuticals intended to treat cancer were approved without EFD studies. Amongst the 51 drugs for which both rat and rabbit EFD studies were performed, the rabbit appeared more sensitive species of

Structure and function of therapeutic antibodies approved by the US FDA in 2023.

In calendar year 2023, the United States Food and Drug Administration (US FDA) approved a total of 55 new molecular entities, of which 12 were in the class of therapeutic antibodies. Besides antibody protein drugs, the US FDA also approved another five non-antibody protein drugs, making the broader class of protein drugs about 31% of the total approved drugs. Among the 12 therapeutic antibodies approved by the US FDA, 8 were relatively standard IgG formats, 3 were bivalent, bispecific antibodies and 1 was a trivalent, bispecific antibody. In 2023, no new antibody-drug conjugates, immunocytokin

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… Figure 1 shows a comparison of these 2023 approvals with previous years, indicating that 2023 was a strong year both for novel drug approvals in the USA in general and for antibody-based drugs. … Novel drug approvals for 2023 . U.S Food and Drug Administration, https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2023 . Last accessed January 10, 2023 2. Kaplon, H, Crescioli, S, Chenoweth, A et al. …

The RAG1 N-terminal region regulates the efficiency and pathways of synapsis for V(D)J recombination.

Myelofibrosis (MF) is a myeloproliferative neoplasm characterized by clonal proliferation of differentiated myeloid cells leading to bone ma … especially in high-risk patients. This concern has led to a prolonged clinical hold and delayed the drug approval by several years during which the drug exchanged manufacturers, highlighting the need for meticulous investigation and adjudication of serious, but rare, adverse events in drug development that could end up preventing drugs with favorable risk/benef …

Tumor-Infiltrating Lymphocyte Therapy for the Treatment of Metastatic Melanoma.

Tumor-infiltrating lymphocyte (TIL) therapy is a type of personalized immunotherapy that harnesses the antitumor activity of endogenous immune cells. While TIL therapy has been in clinical investigation since the 1980s and shown promising signs of clinical activity in immunogenic solid tumors such as melanoma, more recent improvements in product manufacturing and characterization have demonstrated consistent efficacy and the feasibility of administering TIL therapy on a larger scale. Lifileucel was granted accelerated approval by the US Food and Drug Administration in February 2024 for the tre

Progression-free survival as a surrogate endpoint in myeloma clinical trials: an evolving paradigm.

Measurement of overall survival (OS) remains the gold standard for interpreting the impact of new therapies for multiple myeloma in phase 3 trials. However, as outcomes have improved, it is increasingly challenging to use OS as the primary endpoint if timely approval of novel agents is to be ensured to enable maximum benefit for patients. Surrogate endpoints of OS, such as progression-free survival (PFS) and response to treatment, have contributed to approval decisions by the Food and Drug Administration (FDA) and European Medicines Agency as endpoints demonstrating clinical benefit, and the F

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… Progression-free survival (PFS), defined as time from start of therapy to disease progression or death, has been used in this way by both the FDA and the European Medicines Agency (EMA) in many drug approvals to date [ 3 , 4 ]. … In our view, implementing these measures will hopefully ensure timely drug approvals in myeloma and subsequently, further improvements in clinical benefit for all our patients. …

Clinical Development, Regulatory, and Reimbursement Review Duration of New Drugs in the US and European Countries.

IMPORTANCE: Rapid access to new drugs is of interest to patients and society. OBJECTIVE: To analyze duration for clinical development, regu … sement of new drugs showed only minimal variation over time. Given recent efforts to accelerate new drug approvals, future research should evaluate potentially modifiable factors that increase clinical development durations. This could help to better target policies to accelerate access to new drugs, ideally adequately balancing safety with speed.

Biocatalytic routes to anti-viral agents and their synthetic intermediates.

With recent outbreaks of COVID-19 and Ebola, health and healthcare have once more shown to be heavily burdened by the lack of generally effective anti-viral therapies. Initial scientific ventures towards finding anti-viral agents are soon to be followed by challenges regarding their mass production. Biocatalysis offers mild, highly selective, and environmentally benign synthetic strategies for the production of pharmaceuticals in a sustainable fashion. Here we summarise biocatalytic methods that have been applied to the production of FDA-approved anti-viral drugs and their intermediates. Exemp

Update on taxane development: new analogs and new formulations.

The taxanes (paclitaxel and docetaxel) represent an important class of antineoplastic agents that interfere with microtubule function leading to altered mitosis and cellular death. Paclitaxel (Taxol(®)) was originally extracted from a yew tree (Taxus spp., Taxaceae) a small slow-growing evergreen, coniferous tree. Due to the initial scarcity of paclitaxel, docetaxel (Taxotere(®)) a semisynthetic analog of paclitaxel produced from the needles of European yew tree, Taxus baccata was developed. Docetaxel differs from paclitaxel in two positions in its chemical structure and this small alteration

Comparators in Pharmacovigilance: A Quasi-Quantification Bias Analysis.

BACKGROUND AND OBJECTIVE: It is unclear which comparator is the most appropriate for bias reduction in disproportionality analyses based on spontaneous reports. We conducted a quasi-quantitative bias analysis using two well-studied drug-event combinations to assess how different comparators influence the directionality of bias in pharmacovigilance. METHODS: We used the US Food and Drug Administration Adverse Event Reporting System focusing on two drug-event combinations with a propensity for stimulated reporting: rivaroxaban and hepatotoxicity, and canagliflozin and acute kidney injury. We as

Sources of pharmacokinetic and pharmacodynamic variability and clinical pharmacology studies of antiseizure medications in the pediatric population.

Multiple treatment options exist for children with epilepsy, including surgery, dietary therapies, neurostimulation, and antiseizure medications (ASMs). ASMs are the first line of therapy, and more than 30 ASMs have U.S. Food and Drug Administration (FDA) approval for the treatment of various epilepsy and seizure types in children. Given the extensive FDA approval of ASMs in children, it is crucial to consider how the physiological and developmental changes throughout childhood may impact drug disposition. Various sources of pharmacokinetic (PK) variability from different extrinsic and intrins

Editorial: Current Status of Oral Antiviral Drug Treatments for SARS-CoV-2 Infection in Non-Hospitalized Patients.

On 4th November 2021, the first oral antiviral drug for COVID-19, molnupiravir (Lagevrio®), received full regulatory approval from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK. Molnupiravir is an orally bioavailable antiviral drug for use at home when a SARS-CoV-2 test is positive. On 22nd December 2022, the FDA granted emergency use authorization (EUA) for the oral antiviral drug, nirmatrelvir/ritonavir (Paxlovid®) for adults and children with mild and moderate COVID-19 at increased risk of progression to severe COVID-19. These regulatory drug approvals come at a c…

Impact of Deuterium Substitution on the Pharmacokinetics of Pharmaceuticals.

OBJECTIVE: Stable heavy isotopes of hydrogen, carbon, and other elements have been incorporated into drug molecules, largely as tracers for quantitation during the drug development process. Studies involving the human use of drugs labeled with deuterium suggest that these compounds may offer some advantages when compared with their nondeuterated counterparts. Deuteration has gained attention because of its potential to affect the pharmacokinetic and metabolic profiles of drugs. Deutetrabenazine (Austedo, Teva Pharmaceutical Industries, Ltd) is the first deuterated drug to receive Food and Drug

Survey analysis on comparative use human factors processes.

Proposed generic drug-device combination products submitted for FDA approval through the Abbreviated New Drug Application (ANDA) pathway require a comparison to the specific brand name version of the drug, or reference listed drug (RLD). The application requires data demonstrating the generic drug is not only pharmaceutically equivalent, but also that any associated device administering the drug is interchangeable from a human factors perspective. In 2017, the FDA made available the draft guidance for industry entitled "Comparative Analyses and Related Comparative Use Human Factors Studies for