A meeting was held between the Center's management and representatives of the sponsor and applicant companies regarding clinical trials of medicines for patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and severe blood disease.
The investigational drug against PNH has successfully passed preclinical trials and studies involving healthy volunteers. The meeting discussed the possibilities of conducting further clinical trials (phase 1-3) and optimizing the treatment that patients currently need. In addition, the sponsor promises lifelong free provision of the necessary treatment to patients.
The discussion was attended by the Director of the Center Mykhailo Babenko, Deputy Director for Clinical Matters Mykhailo Lobas, Director of the Department of HTA and Rational Pharmacotherapy of the Center Oresta Piniazhko, and specialists of the Department of Expert Evaluation of Preclinical and Clinical Trial Materials of the Center. From the sponsor and applicant companies – Vice President of Omeros Corporation (USA) Steve Whitaker and Regional Manager of LLC “ARENSIA EXPLORATORY MEDICINES” (Germany/Ukraine) Yevhen Levenko.
Director of the Center Mykhailo Babenko thanked foreign sponsors for their attention to Ukraine and for defending the interests of patients with orphan diseases.
“Despite the war, the Center is doing everything possible to ensure that clinical trials of medicines in Ukraine are conducted with quality, on time, and in accordance with the requirements and principles of good clinical practice. We thank the American company ‘Omeros Corporation’ and the German company in Ukraine ARENSIA Exploratory Medicine for continuing to conduct studies in Ukraine and making efforts so that patients receive the necessary treatment in these difficult times,” emphasized Mykhailo Babenko, Director of the Center, during the meeting.
For his part, Deputy Director for Clinical Affairs Mykhailo Lobas gave assurances that all expert procedures on the part of the Center would be completed within the timeframes provided by law, with due regard for the protection of the rights, safety, and well-being of trial subjects, so that patients with PNH could start treatment as soon as possible.
PNH is a rare genetic blood disease that causes the breakdown of red blood cells (hemolysis) and releases hemoglobin into the bloodstream. Patients suffer from hemolytic anemia, chronic fatigue, and thrombosis, which are the most life-threatening. To sustain life, patients need to be systematically administered a drug that makes it possible to suppress hemolysis and maintain a stable level of hemoglobin in the blood. According to statistics, there are about 40 such patients in Ukraine.
