Iloprost

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.

Pictured: FDA signage at its office in Maryland/iStock, hapabapa Eicos Sciences on Wednesday secured the FDA’s approval for its vasodilator iloprost, now to carry the brand name Aurlumyn, for the treatment of severe frostbite and to decrease the risk of amputation. Norman Stockbridge, director of the Division of Cardiology and Nephrology at the FDA’s Center for Drugs and Evaluation Research, in a statement said Aurlumyn is the “first-ever treatment option for severe frostbite.” “Having this new option provides physicians with a tool that will help prevent the lifechanging amputation of one’s frostbitten fingers or toes,” Stockbridge said. Iloprost is a vasodilator whose mode of action is to loosen up blood vessels and prevent clots from forming. The drug was originally approved in 2004 in an inhalable form called Ventavis, manufactured by CoTherix and indicated for pulmonary arterial hypertension. In frostbites, vasodilation could help counter frozen tissues and keep blood flowing to the extremities, preventing amputation. The FDA had previously given iloprost its Priority Review and Orphan Drug designation for severe frostbite. Wednesday’s approval is backed by data from an open-label and controlled study that enrolled 47 severely frostbitten adults who were treated with intravenous aspirin plus standard of care. Patients were randomly assigned to receive Aurlumyn intravenously for six hours a day for up to eight days. The two other groups received unapproved frostbite interventions with or without Aurlumyn. Treatment efficacy was measured using a bone scan, which was highly predictive of the risk of amputation. The results, published in January 2011 in a correspondence article in The New England Journal of Medicine, demonstrated that patients treated with Aurlumyn alone saw a 0% risk of amputation, whereas those who did not receive Eicos’ vasodilator had a 60% likelihood of amputation. Patients who received Aurlumyn plus another unapproved therapy showed a 19% risk of amputation. This risk reduction was validated by the actual number of amputated digits, according to the study. Of the 183 frozen fingers 22 were eventually amputated, nearly half of which belonged to patients who did not receive Aurlumyn infusions. In the Aurlumyn-alone group, no fingers were amputated. Of the 224 frozen toes, 25 required amputations, 34% of which were in the group that did not receive Aurlumyn treatment. As in the case of fingers, none of the Aurlumyn-treated patient required toe amputations. In terms of safety, Aurlumyn’s most common side effects are headache, palpitations, dizziness, flushing, hypotension, nausea, fast heart rate and vomiting. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.