Resume af teksten:
Der er indsendt ansøgninger til FDA og EMA om godkendelse af etavopivat til seglcellesygdom.
Ansøgningerne bygger på data fra fase 3-studiet HIBISCUS med unge og voksne.
FDA og Europa-Kommissionen forventes at træffe afgørelse om ansøgningerne i 2027.
FDA har tildelt etavopivat status som Fast Track, Rare Pediatric Disease og Orphan Drug.
Europa-Kommissionen har tildelt lægemidlet Orphan Drug-status.
Etavopivat er et undersøgende, oral behandling, der tages én gang dagligt.
HIBISCUS sammenlignede etavopivat med placebo over 52 uger hos omkring 440 personer på 12 år eller derover.
Studiet vurderede blandt andet årlig forekomst af vaso-okklusive kriser og hæmoglobinrespons.
Fra Novo Nordisk:
The FDA New Drug Application (NDA) and EMA Marketing Authorization Application (MAA) submissions for etavopivat are based on data from the Phase 3 HIBISCUS trial showing efficacy and safety results in adolescents and adults living with sickle cell disease 1 Sickle cell disease is a progressive, life-threatening, debilitating blood disease with limited disease-modifying therapeutic options that affects an estimated 100,000 Americans, 110,000 Europeans – and around 8 million people worldwide 2-4 Etavopivat is designed to help reduce the sickling of red blood cells and is being evaluated for potential improvement of haemoglobin response (
The FDA has previously granted etavopivat Fast Track, Rare Pediatric Disease and Orphan Drug designations and is expected to take action on the NDA in 2027. Etavopivat was also granted Orphan Drug designation by the European Commission, which is expected to take action on the MAA in 2027. An MAA is the formal application for seeking approval to market a medicine in the European Union (EU).
“For people living with sickle cell disease, severe vaso-occlusive crises and other symptoms can have a profound impact on everyday life, and treatment options remain limited,” said Martin Holst Lange, executive vice president, chief scientific officer and head of Research & Development at Novo. “If approved, we are hopeful that etavopivat can provide a new disease-modifying treatment option for people living with sickle cell disease, and we will continue to work alongside the community to help advance health equity and overcome the longstanding disparities that impact those affected by this disease.”
The NDA and MAA submissions are grounded in the results from the HIBISCUS study program, which was designed to establish the efficacy and safety profile of etavopivat, and evaluated the annualised rate of vaso-occlusive crises (VOCs) at week 52 and haemoglobin response (
What is sickle cell disease? Sickle cell disease (SCD) is driven by polymerisation of mutated sickle haemoglobin (HbS) in red blood cells. The two main hallmarks of SCD are hemolytic anaemia and vaso-occlusive crises (VOCs). Around 8 million people worldwide live with SCD, with the majority in low- and middle-income countries. 4 Approximately 100,000 people living with SCD are in the United States, as well as approximately 110,000 in Europe, 5.7 million in sub-Saharan Africa, 1.28 million in India, and 618,000 in the Middle East and North Africa. 3-5 The disease is characterised by several associated acute and chronic symptoms and complications such as acute chest syndrome, stroke and kidney, liver and heart disease. 3 Despite recent advances in treatment, many people with SCD experience significant levels of pain and reduced lifespan by approximately 20 years compared to the general population. 3
What is etavopivat? Etavopivat is an investigational, once-daily, orally available, selective activator of pyruvate kinase red blood cell isozyme (PKR), a key enzyme in glycolysis. In SCD, PKR activation reduces 2,3-diphosphoglycerate (2,3-DPG) levels and increases adenosine triphosphate (ATP) production. Reduction of 2,3-DPG improves haemoglobin (Hb)-oxygen affinity, averting sickle haemoglobin polymerisation and subsequent sickling. The increase in ATP production helps preserve RBC membrane integrity and deformability, improving RBC survival. These disease-modifying effects have the potential to reduce VOC events and improve Hb levels.
The US Food and Drug Administration has granted etavopivat Fast Track, Rare Pediatric Disease and Orphan Drug designations. Additionally, etavopivat was granted Orphan Drug designation by the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency for the treatment of patients with SCD.
Etavopivat was acquired by Novo as part of the 2022 acquisition of Forma Therapeutics.
What is the HIBISCUS programme? The HIBISCUS clinical development programme investigates etavopivat as a disease-modifying treatment for people with SCD. The pivotal programme includes:
HIBISCUS (NCT04624659) – a 52-week efficacy and safety design phase 2/3 trial, comparing once-daily etavopivat versus placebo, enrolling approximately 440 people aged 12 years and over with SCD who had 2 to 15 VOCs within 12 months prior to enrollment. Participants were allowed to receive standard-of-care treatment throughout the trial. Following the completion of the 12-week phase 2 interim readout, the phase 3 dose was selected, and participants were randomised to the phase 3 main phase of the trial, which continued for 52 weeks. 1
HIBISCUS 2 (NCT06612268) – a 52-week efficacy and safety phase 3b trial, comparing once-daily etavopivat versus placebo, with expected enrollment of 510 people aged 12 years and older with SCD. 1
FLORAL (NCT06609226) – an open-label extension following participation in etavopivat trials. The study allows the collection of long-term safety data. 1 Novo is the global healthcare company that believes lasting health starts now. For over a century, we’ve combined leading scientific expertise with a deep understanding of people’s lives. We develop treatments and support that help millions of people make progress they can see, feel and sustain now and in the future. Every day, over 66,500 employees around the world advance our purpose to drive change for lasting health. Through our partnerships, programmes and investments, we’re working to prevent disease, expand access to treatments and reduce our environmental impact to help even more people live healthier lives. In the United States, Novo has a 40-year presence, is headquartered in New Jersey and employs approximately 10,000 people. For more information, visit novonordisk.com and novonordisk-us.com and follow us on Instagram , LinkedIn , TikTok , Facebook , X and YouTube .
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