Clinical trials for rare diseases
European Pharmaceutical Review » Clinical Trials
by Mandy Parrett
7h ago
Measuring treatment effect in rare disease populations presents many methodological challenges due to small sample sizes and heterogeneity of the study population. Additionally, negative or ambiguous results from clinical trials may leave regulators and clinical trial participants wondering whether the failure was due to the drug’s lack of efficacy or to methodological flaws. As participants and families devote years of their lives to clinical trial participation, it is crucial that any changes in function that patients experience are sensitively detected during a clinical trial. Heterogeneo ..read more
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Positive CHMP recommendation for antibiotic combination
European Pharmaceutical Review » Clinical Trials
by Caroline Peachey
3d ago
The Committee for Medicinal Products for Human Use (CHMP) has recommended granting of a marketing authorization for antibiotic combination Emblaveo® (aztreonam-avibactam) for treatment of complicated intra-abdominal and urinary tract infections, hospital-acquired pneumonia and infections caused by certain types of aerobic Gram-negative bacteria. “Gram-negative bacteria are often resistant to multiple drugs, leaving infected patients very ill and at high risk of severe complications, including mortality,” said James Rusnak, Senior Vice President and Chief Development Officer, Internal Medicine ..read more
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Novo Nordisk to buy Cardior Pharmaceuticals
European Pharmaceutical Review » Clinical Trials
by Caroline Peachey
3d ago
Novo Nordisk has agreed to acquire Cardior Pharmaceuticals, a leader in the discovery and development of RNA-based therapies for heart failure for up to €1.025 billion. The agreement includes Cardior’s lead compound CDR132L, currently being investigated in a Phase II clinical trial (HF-REVERT) for the treatment of heart failure. The deal comprises an upfront payment, plus additional payments if certain development and commercial milestones are achieved. CDR132L is designed to halt and partially reverse cellular pathology by selectively blocking abnormal levels of the microRNA molecule miR-132 ..read more
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Combination treatment could minimise cancer relapse risk
European Pharmaceutical Review » Clinical Trials
by Catherine Eckford
6d ago
Research suggests that combining ribociclib with hormone therapy improved invasive-free survival in stage 2 or 3 HR-positive, HER2-negative early breast cancer. The study data showed that distant disease-free survival and recurrence-free survival improved when the small molecule CDK4/6 inhibitor ribociclib was used alongside a nonsteroidal aromatase inhibitor (NSAI). A significant reduction in the rate of disease recurrence “We found that adding ribociclib to the standard hormone therapy resulted in a relative reduction in the recurrence rate by as much as 25 percent,” stated first author of ..read more
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Novartis acquisition to address inflammation-driven diseases
European Pharmaceutical Review » Clinical Trials
by Catherine Eckford
1w ago
Under a new deal, Novartis has agreed to acquire US-based biopharma company IFM Therapeutics, for a total of up to $835 million. As part of this new agreement, Novartis has exercised its option to acquire all outstanding capital stock of IFM Due, a subsidiary company of IFM. Therefore, under the acquisition, Novartis has full rights to IFM Due’s portfolio of small molecule Stimulator of Interferon Genes (STING) antagonists, IFM confirmed. Through the full acquisition, IFM will be eligible for potential milestone payments of a combined value of up to $835 million. These treatments have potenti ..read more
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AstraZeneca agrees novel $1 billion endocrine acquisition
European Pharmaceutical Review » Clinical Trials
by Catherine Eckford
1w ago
For a total of up to $1.05 billion, AstraZeneca has agreed to acquire the biotech Amolyt Pharma, to advance a novel treatment for hypoparathyroidism, a rare disease. CEO of Alexion, AstraZeneca Rare Disease, Marc Dunoyer, explained that the proposed acquisition will facilitate the company’s expansion into rare endocrinology. Under the terms of the agreement, Alexion will be able to add eneboparatide (AZP-3601), which has a novel mechanism of action, to its pipeline. The Phase III investigational therapeutic peptide eneboparatide “has the potential to lessen the often debilitating impact of lo ..read more
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Parkinson’s infusion treatment demonstrates advantage over oral delivery
European Pharmaceutical Review » Clinical Trials
by Catherine Eckford
1w ago
A Phase III trial has shown that compared oral drug delivery, administering levodopa through an infusion pump led to nearly two hours of day (1.72) of additional time in which the medicine reduced symptoms in Parkinson’s patients. There were 381 Parkinson’s patients enrolled in the trial. Levodopa was delivered for 24 hours via a subcutaneous infusion pump, which was shown to be a safe method of drug delivery, the research found. Participants were either were given levodopa subcutaneously through the infusion pump or via standard oral treatment. While oral levodopa is effective and normally e ..read more
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FDA approval could shape liver disease treatment landscape
European Pharmaceutical Review » Clinical Trials
by Catherine Eckford
1w ago
The US Food and Drug Administration (FDA) has approved the first treatment for noncirrhotic non-alcoholic steatohepatitis (NASH), also known as fatty liver disease, in these patients who also have fibrosis. Rezdiffra (resmetirom) is indicated for adults with moderate to advanced fibrosis), alongside diet and exercise. The treatment is a thyroid hormone receptor (THR)-β agonist. FDA highlighted that it works by helping to reduce fat accumulating in the liver.  “Previously, patients with NASH who also have notable liver fibrosis did not have a medication that could directly address th ..read more
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Developing a new treatment paradigm for IgAN
European Pharmaceutical Review » Clinical Trials
by Catherine Eckford
2w ago
In recent months there have been important development in the kidney disease treatment space. For IgA nephropathy (IgAN), key highlights include the US Food And Drug Administration (FDA) approval of Calliditas Therapeutics’ TARPEYO and the Committee for Medicinal Products for Human Use (CHMP)’s recommendation of a conditional marketing authorisation for Filspari (sparsentan). Additionally, there have been notable clinical developments for the treatment of kidney disorders. For example, in January, Phase III trial data showed that the immunotherapy drug pembrolizumab post-surgery was capable o ..read more
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J&J reveal long-term data for novel oral peptide
European Pharmaceutical Review » Clinical Trials
by Catherine Eckford
2w ago
The first and only investigational targeted oral peptide has been shown to maintain high rates of skin clearance in adults with moderate-to-severe plaque psoriasis through 52 weeks, new data shows. In the long-term extension FRONTIER 2 study, JNJ-2113 demonstrated sustained efficacy from Week 16 to Week 52, based on data presented at the American Academy of Dermatology (AAD) 2024 Annual Meeting. Johnson & Johnson stated that JNJ-2113 is the first targeted oral peptide designed to block the IL-23 receptor. IL-23 has a key role in pathogenic T-cell activation in moderate-to-severe plaq ..read more
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