Clinical News
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wAIHA | Rilzabrutinib Receives FDA Breakthrough Therapy Designation
Warm autoimmune haemolytic anaemia is a rare autoimmune disorder characterized by the premature destruction of red blood cells, leading to anemia and other serious complications, such as significant fatigue, dizziness,…
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wAIHA | Sovleplenib Demonstrates Positive Phase III Clinical Results
wAIHA is a rare autoimmune disorder in which the immune system produces antibodies that mistakenly target and destroy red blood cells, leading to anemia and significant clinical burden. The…
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SICKLE CELL DISEASE | Alzheimer’s Drug Demonstrates Potential Therapeutic Benefit
The study explores the potential of memantine, a medication that has been safely used for many years in the treatment of Alzheimer’s disease. Researchers investigated whether this well-known, affordable drug…
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FDA APPROVES AQVESME™ (mitapivat) | First Oral Therapy for Anaemia Across All Forms of Thalassaemia
This regulatory decision marks a landmark milestone in the management of thalassaemia, as AQVESME is now the first and only therapy approved in the United States indicated for treating anaemia…
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PUTTING PATIENTS FIRST IN CLINICAL RESEARCH | New EU Recommendations to Reduce Trials Bureaucracy
As Prof. Martin Dreyling, Chair of the Coalition for Reducing Bureaucracy, explains: “The current safety requirements for investigators are disproportionately burdensome, to the point that they may obscure relevant safety…
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NEW STUDY | Pegcetacoplan Shows Potential in Managing CAD and wAIHA
This investigational therapy, which targets complement component C3, has demonstrated the ability to increase haemoglobin levels, reduce haemolysis, and improve fatigue scores, according to the study’s authors. Although not all…
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WHAT YOU NEED TO KNOW | EMA Recommends Suspension of Pfizer’s Sickle Cell Disease Drug Oxbryta
Oxbryta, which was designed to treat sickle cell disease, has been a crucial medication for patients with mild-to-moderate symptoms. Approved in the US under an accelerated process in 2019 and…
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SICKLE CELL DISEASE | FDA Approves Two Landmark Gene Therapies for the Treatment of the Disorder
The two therapies are Casgevy (exa-cel) by Vertex Pharmaceuticals and CRISPR Therapeutics – the world’s first drug to utilize the revolutionary CRISPR gene-editing system which earned its creators the 2020…
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TOP STORY | EU Approves Luspatercept for Anemia in Patients with Non–Transfusion-Dependent β-Thalassaemia
The approval was based on findings from the phase 2 BEYOND trial, which demonstrated that 77.1% of patients treated with luspatercept (n = 74/96) experienced a mean haemoglobin (Hb) increase…
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IRON CONTROL | Disc Medicine And Mabwell Sign Deal For Licence To Antibodies Portfolio
Disc plans to initiate a phase 1 trial in healthy volunteers in the second half of 2023. MWTX-003 has the potential to address a wide range of hematologic disorders including…
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