International Top Pharmaceutical Awards

No doubt about it, UTIs suck. They burn, they itch, they come back just when you think you’re in the clear. But for those dealing with these relentles s infections, there’s finally a new line of defense. A new type of antibiotic is now available for some of the most annoying infections around. This month, the Food and Drug Administration approved              NN Iterum Therapeutics’ Orlynvah for certain kinds of urinary tract infections that aren’t likely to respond to other drugs. UTIs are some of the most common infections that people encounter, particularly women. About 60% of women in the U.S. are estimated to experience at least one uncomplicated UTI (a UTI with no signs of structural damage or other health conditions) in their lifetime. While UTIs were once easily treatable with just about any simple course of antibiotics, many infections nowadays are resistant to at least one or more front-line drugs used against them. This added hardiness no...

Managing chronic myeloid leukemia (CML) can be challenging, especially when it comes to accurately tracking how well patients respond to treatment and spotting any resistance. Traditional testing methods often miss uncommon gene variations and small mutations. ICON Specialty Laboratory Solutions has created a new, advanced test using next-generation sequencing (NGS) technology. Based on RNA technology, ICON’s new assay offers huge improvements on how CML is monitored. Understanding the challenges in CML molecular monitoring Approximately 98% of CML patients exhibit the common e14a2 or e13a2 BCR::ABL1 transcript types, while the remaining have atypical transcripts like e1a2 or e19a2. Despite the effectiveness of tyrosine kinase inhibitors (TKIs) in treating CML, resistance often develops due to mutations in the tyrosine kinase domain (TKD) or the myristoyl binding site of BCR-ABL1. This necessitates precise identification and monitoring of BCR::ABL1 transcripts and potential resista...

Diagnosis is the first step into the care journey. To support a timely and accurate dementia diagnosis, it is critical to develop diagnostic tools, such as blood-based biomarker tests, that can support the health-care workforce in their clinical decision-making, and that can be readily implemented in low-resource settings. WHO Preferred Product Characteristics (PPCs) are technical documents that define the preferred attributes of diagnostic products for regulatory purposes, policy, and programmatic implementation. PPCs are intended to encourage innovation and development of products for use in settings more relevant to the global unmet public health need . This PPC defines the preferred parameters of blood-based biomarker diagnostics for Alzheimer disease to ensure that these products will not only exhibit optimal clinical performance but are also relevant to different populations, affordable and available to those who need these products. Considerations for implementation include ...

Swiss pharma giant Roche has received approval from a panel of experts to sell a drug for a rare blood disorder, paroxysmal nocturnal hemoglobinuria (PNH), in India, waiving the requirement to conduct clinical trial s on Indian soil, News18 has learnt. The drug – a humanised complement inhibitor C5 monoclonal antibody – is indicated for the treatment of PNH which is a rare and life-threatening condition leading to the premature destruction of red blood cells. This disorder impacts red blood cells (responsible for transporting oxygen), white blood cells (which defend the body against infections), and platelets (crucial for blood clotting). PNH can develop at any age but generally, it is diagnosed among young adults. The condition leads to acute anaemia, fatigue, blood clots, and kidney disease. According to an Indian study published in 2021, the incidence of PNH in the country among people with blood disorders is between 11.8% and 35.3% with a mean age of presentation of 34 years. In Eu...

At IDWeek 2024, Moderna presented interim results from an ongoing Phase I/II, randomised, observer-blind, placebo-controlled, dose-ranging trial (NCT05992935) for mRNA-1403, a prophylactic vaccine candidate under investigation for norovirus (NoV) infections. As one of the leading causes of acute gastroenteritis worldwide, NoV is associated with a substantial healthcare burden. Symptoms include vomiting and diarrhoea, which can be severely dehydrating, and the risk of severe outcomes from NoV is greatest in young children and older adults.To date, no vaccines that protect NoV have been licensed. A key reason for this is the virus’ broad and shifting genotype diversity and limited cross-genotype protection, which poses challenges for the development of an effective vaccine. Epidemiology data from the past two decades indicates that there are, however, a limited number of prevalent strains and recurring patterns in terms of the strains that circulate. Therefore, a multivalent mRNA vacci...

US-based biopharmaceutical company Nurix Therapeutics has reported positive clinical data from its ongoing Phase Ia/Ib trial of NX-5948, a BTK degrader designed to treat relapsed/refractory Waldenstrom’s macroglobulinaemia (WM) . The findings were shared at the 12th International Workshop on Waldenstrom’s Macroglobulinemia in Prague, Czech Republic, which was held from 17 to 19 October. WM is a rare non-Hodgkin’s lymphoma marked by abnormal lymphocytes and monoclonal immunoglobulin M (IgM) production. As of 17 April, the trial data showed NX-5948’s safety across all dosages from 50mg to 600mg administered orally daily. In the trial, the therapy showed an objective response in seven out of nine evaluable patients (77.8%), with two patients showing stable disease. See Also:MSD mulls 2025/26 launch . Seven responses were recorded at the first eight-week assessment while five patients are still undergoing treatment, with two of these patients having been treated for more than a yea...

  Glioblastomas are aggressive brain tumors with a median survival time of less than 22 months despite standard therapy including surgery, irradiation, and chemotherapy. It has become clear in recent years that not all cells within the brain tumor have an equal potential to divide and drive tumor growth. As such, a fraction of tumor cells called brain tumor stem cells (BTSCs) are thought to be the primary origin of tumor re-growth after surgery, in addition to being resistant to standard treatments including chemotherapy and irradiation. Therefore , targeting BTSCs may be a way to effectively treat glioblastomas. In an effort to rapidly identify new treatments effective against BTSCs, a team of researchers from the University of Ottawa, Canada, tested Edaravone , an FDA-approved drug, for its efficacy against BTSCs, knowing that Edaravone blocks cellular processes which are important for the growth and survival of BTSCs. The results were published in  Stem Ce...

  A University of Texas at Dallas chemist and his colleagues have developed a new chemical reaction that will allow researchers to synthesize selectively the left-handed or right-handed versions of "mirror molecules " found in nature and assess them for potential use against cancer, infection, depression, inflammation and a host of other conditions. The results are important because, while the left- and right-handed versions, or enantiomers, of chemical compounds have identical chemical properties, they differ in how they react in the human body. Developing cost-effective ways to synthesize only the version with a desired biological effect is critical to medicinal chemistry. In a study published in the Oct. 11 issue of the journal  Science , the researchers describe how their chemical synthesis method can quickly, efficiently and in a scalable manner produce a sample that is purely one enantiomer of a mirror-image pair of molecules, as opposed to a mixture of the two. Th...