So far, the 2024 medical insurance catalog adjustment has announced the drugs and related information that have passed the formal review. From the draft for comments, rare disease drugs are still one of the focuses of the medical insurance catalog adjustment. Rare diseases are also called “orphan diseases”. Most of them are congenital diseases, chronic diseases, and may be life-threatening. Although the incidence of rare diseases is low, there are many types of diseases and the number of patients cannot be underestimated. At present, there are more than 7,000 confirmed rare diseases in the world, and there are about 20 million rare disease patients in China1. For a long time, the clinical phenotypes of rare diseases and common diseases overlap greatly, and early diagnosis is difficult, so the misdiagnosis and missed diagnosis rates are high. According to statistics from the National Organization for Rare Diseases in the United States, among ...
In the field of tumors, any breakthrough is not easy. Although humans have discovered cancer for a long time, chemotherapy has long been the core treatment method. Of course, after countless failures and dashed hopes, once there is a breakthrough, the progress is often unexpected. For example, after decades of silence, the treatment of small cell carcinoma seems to have ushered in a period of accelerated breakthroughs. On August 15, AstraZeneca announced that its drug durvalumab has been accepted by the FDA for the indication of limited small cell lung cancer (LS-SCLC). If approved for marketing, this will be the first immunotherapy in 40 years to show survival benefits in this field, representing an important breakthrough. For limited-stage SCLC, the current first-line treatment is still chemoradiotherapy, especially the treatment of cisplatin combined with etoposide, which occupies an absolute dominant position in treatment. Amgen has also made progress in the field ...
Johnson & Johnson’s (J&J) Rybrevant (amivantamab-vmjw) plus Lazcluze (lazertinib) has secured US Food and Drug Administration (FDA) approval as a first-line treatment for patients with some types of non-small cell lung cancer (NSCLC). The indication covers patients with locally advanced or metastatic NSCLC with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test. The approval is based on data from the Phase III MARIPOSA study (NCT04487080), which compared the Rybrevant/Lazcluze combination to AstraZeneca’s Tagrisso (osimertinib). Data showed that Rybrevant plus Lazcluze reduced the risk of disease progression or death by 30% compared to Tagrisso alone. Additionally, the median duration of response was nine months longer with the combination therapy compared to Tagrisso. The study met its primary endpoint of progression-free survival (PFS) and additional secondary endpoints, including participant race, type of EGFR mutation, and history of brain metastasis. These ...
Researchers from the Universities of Cambridge, Bristol and Edinburgh have suggested that COVID-19 vaccinations could lower the incidence of arterial thromboses. The study, published in Nature Communications, was supported by the British Heart Foundation (BHF) Data Science Centre at Health Data Research UK. Researchers analysed the de-identified health records of 46 million adults from GP practices, hospital admissions and death records in England between December 2020 and January 2022, provided by NHS England. The team compared the incidence of cardiovascular diseases (CVDs) following COVID-19 vaccination with the incidence before or without vaccination during the first two years of the vaccination programme. CVD affects around seven million people in the UK and is a significant cause of disability and death. Overall, the study revealed that the incidence of arterial thromboses, such as heart attacks and strokes, which affect 200,000 people in the UK every year combined, was up to 10% lower ...
Chinese pharmaceutical industry insiders have warned that foreign takeovers may damage the sector and even threaten national security. Since December, five Chinese biotech drug makers have been sold to global pharmaceutical giants. Industry insiders have warned that this could have a negative impact, especially given the risk that the United States could extend its technology sanctions to essential medicines. John Cai, chairman of China Healthcare Innovation Platform Academy, a healthcare think tank in Shanghai, said: “When national conflicts occur and drugs are sanctioned as strategic products, it will affect the health of a country’s population. “Considering that China’s biopharmaceutical and broader healthcare industry are now facing international competition and restrictions, Beijing should act with a sense of urgency.” He also said that research and development was a long process and if innovative products were sold as soon as they were developed, it would be difficult for China to cultivate world-class ...
NHS Scotland, AstraZeneca (AZ) and the Universities of Glasgow and Dundee have entered into a partnership to accelerate research into treating chronic kidney disease (CKD). The collaboration aims to develop new medicines to slow down the progression of CKD, build infrastructure and enhance expertise in renal clinical trial delivery in Scotland to improve patient outcomes. Affecting nearly 850 million people worldwide, CKD is a serious, progressive condition caused by decreased kidney function, commonly caused by diabetes, hypertension and glomerulonephritis. By preventing the progression of CKD, patients will live longer, better lives, free from other consequences of the condition, including heart failure, cardiovascular disease and stroke, while also reducing pressure on the NHS. In addition to slowing the progression of CKD, NHS Scotland, AZ and the Universities of Glasgow and Dundee will work to reduce the risk of patients potentially needing dialysis, a procedure to remove waste products and excess fluid ...
By Don Tracy, Associate Editor FDA’s Oncologic Drugs Advisory Committee acknowledged that Imfinzi met its primary endpoint of event-free survival in the treatment of resectable non-small cell lung cancer. The FDA’s Oncologic Drugs Advisory Committee (ODAC) has found that AstraZeneca’s Imfinzi (durvalumab) achieved the primary endpoint of event-free survival (EFS) in the treatment of resectable non-small cell lung cancer (NSCLC) in the Phase III AEGEAN trial. However, AstraZeneca noted that although the contribution by phase of neoadjuvant and adjuvant components of the perioperative regimen could not be clearly assigned based on the trial design, its potential should not be overlooked. Results of the trial were published in The New England Journal of Medicine. “The majority of patients with resectable lung cancer face recurrence of their disease even after surgery and neoadjuvant chemotherapy. The Committee acknowledged the potential to address this urgent unmet need with durvalumab both before and after surgery, ...
The US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) has called for changes in how clinical trials are conducted for treatments involving surgery for early-stage non-small cell lung cancer (NSCLC). In the 25 July meeting, the ODAC highlighted that it’s important to separately evaluate the effectiveness of each part of the treatment process, which includes pre-operative and post-operative phases. The committee meeting – which ended with a 11–0 vote in favour of changing how perioperative trials are run– came after AstraZeneca proposed to use Imfinzi (durvalumab) as a treatment with chemotherapy both before and after surgery. The pharma giant conducted the Phase III AEGEAN study (NCT03800134) to support this label expansion. The committee did not vote on Imfinzi’s approval in this indication, but the overall majority suggested that it should be approved. Following a lengthy discussion as to whether there should be another trial, Ravi Madan, medical ...
Recent studies have allowed the unexplored field of bronchiectasis to be further investigated by shedding light on the mechanisms behind the disease’s pathophysiology. In recent years, bronchiectasis has garnered a significant amount of attention, with pharmaceutical companies prioritising their investments in the development of promising pipeline agents to treat the disease through various mechanisms of action (MOAs). Pharmaceutical companies involved in the field of bronchiectasis include Insmed, Boehringer Ingelheim, Sanofi, Renovion, Armata Pharmaceuticals, Chiesi, CSL, BioAegis Therapeutics, and Reistone Biopharma, all of which are shaping the competitive landscape of the field, according to GlobalData. Current research focuses on disease mechanisms, such as inflammation, further uncovering potential targets for pharmaceutical companies. Such targets include neutrophil-mediated inflammation components but also general inflammation components, such as interleukins and bacteriophages. Interestingly, the pipeline landscape is filled with Phase I and Phase II agents, with only Insmed’s brensocatib in Phase III. These pipeline agents have ...
European Medicines Agency approval of Dupixent in chronic obstructive pulmonary disease makes the drug the first biologic therapy approved for treating the prevalent respiratory condition. In COPD, Dupixent addresses what’s called type 2 inflammation. By Frank Vinluan Inflammation that develops in chronic obstructive pulmonary disorder stems from different causes. A new regulatory decision in Europe makes Sanofi and Regeneron Pharmaceuticals drug Dupixent the first biologic therapy approved for COPD driven by one particular type of inflammation. The European Medicines Agency (EMA) has approved Dupixent for adults with uncontrolled COPD that is also characterized by raised blood levels of eosinophils, a type of white blood cell. COPD patients typically take multiple medications to manage the chronic condition. The new Dupixent approval, announced just prior to the Independence Day holiday in the U.S., covers use of the injectable drug as an add-on maintenance treatment for patients whose COPD is uncontrolled by available therapies. ...
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