Iloprost

Aurlumyn injection is a new treatment option for adults with severe frostbite. Credit: movchanzemtsova / Shutterstock.com. The US Food and Drug Administration (FDA) has approved Eicos Sciences’s Aurlumyn (iloprost) injection, a new treatment for adults with severe frostbite to lower the risk of amputation of fingers or toes. Aurlumyn contains iloprost, a vasodilator that opens blood vessels and stops blood clotting, as its active ingredient. The efficacy of iloprost in treating severe frostbite was established through an open-label controlled trial in 47 adult subjects. Participants were randomised into three groups, with group one receiving iloprost intravenously for six hours daily for up to eight days along with aspirin and standard of care. Groups two and three were administered other medications, with and without iloprost, which are not approved for frostbite treatment. See Also: Otsuka wins FDA breakthrough therapy designation for IgAN drug QBiotics wins FDA orphan status for rainforest-derived cancer drug The trial’s primary efficacy measure was bone scans on day seven following the frostbite incident to predict the need for amputation. The scans showed that none of the subjects who received iloprost alone (group one) required amputation compared to 19% in group two and 60% in group three. This indicated a significant reduction in the presence of bone scan abnormalities in groups receiving iloprost. Most subjects had follow-up data available to confirm whether they underwent amputation. This aligned with the bone scan findings. Headache, flushing, heart palpitations, nausea and hypotension were common side effects linked to Aurlumyn treatment. The treatment carries a warning for potential symptomatic hypotension. Aurlumyn was granted priority review and orphan drug designations for this indication, highlighting its significance in addressing severe frostbite, a condition that can escalate from mild to severe, potentially leading to permanent tissue damage and the cessation of blood flow. Iloprost originally received approval in 2004 for pulmonary arterial hypertension. FDA Center for Drug Evaluation and Research Division of Cardiology and Nephrology director Norman Stockbridge stated: “This approval provides patients with the first-ever treatment option for severe frostbite. “Having this new option provides physicians with a tool that will help prevent the life-changing amputation of one’s frostbitten fingers or toes.”

Diamyd is an antigen-specific immunotherapy for the preservation of beta cells – insulin-producing cells. Image Credit: Image Point Fr / Shutterstock. Swedish biotech Diamyd has won fast track designation for its eponymous type 1 diabetes (T1D) immunotherapy Diamyd (rhGAD65/alum) from the US Food and Drug Administration (FDA). Diamyd is an antigen-specific immunotherapy for the preservation of beta cells, which produce insulin. It is being developed as a therapy for improving glycaemic control in T1D with the HLA DR3-DQ2 genotype – a gene associated with increased susceptibility to insulin-dependent diabetes. The therapy is being evaluated in a randomised, placebo-controlled Phase III DIAGNODE-3 trial (NCT05018585). The study is expected to enrol approximately 330 participants aged 12–28 years who were recently diagnosed with T1D and carry the HLA DR3-DQ2 gene. The trial’s primary endpoints include evaluating a change in blood glucose levels measured using haemoglobin A1c (HBA1c) and stimulated C-peptide during a mixed meal tolerance test (MMTT) that checks how well the beta cells are working, at 24 months compared to baseline. The trial suffered a setback when the US FDA placed a partial clinical hold on the trial for approximately 14 months starting September 2021, but the trial did continue recruitment and treatment across its European sites. The trial is expected to complete enrolment in H2 of this year, as per the company’s 2024 financials. Diamyd is also being investigated as a preventive therapy for children with the HLA DR3-DQ2 genotype who are at risk of developing T1D. The open-label DiaPrecise trial (2022-002356-39) is being conducted in partnership with a Swedish research centre DiaUnion. The study will enrol approximately 16 pre-diabetic participants ages 8–18 years with HLA DR3-DQ2 genotype. See Also: FDA maps out process for sponsors to charge for drugs in clinical trials FDA approves Eicos’s Aurlumyn for severe frostbite treatment The burden of T1D is forecasted to increase in the coming years. A modelling study estimated the prevalence of T1D to be approximately 13.5–17.4 million in 2040. In 2022, the FDA approved the first and only treatment to delay the onset of T1D, Provention Bio’s Tzield (teplizumab). Following this, Diamyd CEO Ulf Hannelius noted the Tzield approval “creates clarity around the regulatory pathway for disease-modifying therapies in type 1 diabetes and will set a reference for the field regarding pricing and reimbursement”, in an 18 November 2022 press release.