Danazol

GSK’s Omjjara (momelotinib) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat adult myelofibrosis patients with moderate or severe anaemia. Myelofibrosis is a rare blood cancer that affects one in every 500,000 people worldwide, 40% of whom have moderate-to-severe anaemia at the time of diagnosis. Nearly all patients develop anaemia at some point during the course of their disease. The agency’s decision was supported by results from the pivotal MOMENTUM phase 3 trial, which evaluated Omjjara in comparison to another anaemia treatment called danazol. Approximately 195 patients with myelofibrosis and anaemia who had previously been treated with a Janus Kinase (JAK) inhibitor were given either 200mg of Omjjara once daily or 300mg of danazol twice daily for 24 weeks. The treatment works to inhibit the action of enzymes known as JAKs (JAK1, JAK2), which control the activity of cytokines. Cytokines are proteins involved in inflammation in the body, the production of different blood components and regulating the immune system. Omjjara also inhibits the activin A receptor type 1 (ACVR1), which increases the production of hepcidin, a protein that traps iron in the liver and reduces absorption from food, leading to anaemia. Myelofibrosis causes these proteins to act abnormally. The treatment works to block the overproduction of cytokines and hepcidin, reducing inflammation and increasing iron and haemoglobin levels in the blood. The trial met all of its primary and key secondary endpoints, showing that Omjjara was more effective than danazol in reducing symptoms and spleen size. Julian Beach, interim executive director, healthcare quality and access, MHRA said: “We’re assured that the appropriate regulatory standards for the approval of this medicine have been met. “Keeping patients safe and enabling their access to high-quality, safe and effective medical products are key priorities for us.” The MHRA has assured that it will continue to keep the safety and effectiveness of Omjjara under close review. Omjjara has already received approval from the European Commission following a recommendation by the Committee for Medicinal Products for Human Use. The treatment is the first medicine specifically authorised in the EU to treat splenomegaly (enlarged spleen) or symptoms in adult myelofibrosis patients with moderate-to-severe anaemia.

The drug is for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus kinase (JAK) inhibitor naïve or have been treated with ruxolitinib. Professor Claire Harrison, deputy chief medical officer in research, data, and analytics, at Guy’s and St Thomas’, said: “Myelofibrosis is a rare blood cancer with limited treatment options. The approval of Omjjara (momelotinib) is the culmination of many years of collaboration and participation by hundreds of patients in clinical trials. We are very grateful to those who took part as it has enabled this new option for treating myelofibrosis patients with moderate to severe anaemia.” Myelofibrosis is a rare type of blood marrow cancer that disrupts the body’s normal production of blood cells. In its research, GSK found that every year, approximately 380 people are diagnosed with myelofibrosis in the UK. It can lead to splenomegaly, constitutional symptoms such as fatigue, night sweats, and bone pain, as well as low blood counts, including anaemia (deficiency of red blood cells) and thrombocytopenia (deficiency of platelets). The company said nearly all people living myelofibrosis are estimated to develop anaemia over the course of the disease, and may require additional supportive care, including red blood cell (RBC) transfusions. googletag.cmd.push(function () { googletag.display('text-ad1'); }); Innovation for unmet clinical needs Jack Harris, vice-president UK Oncology, at GSK, said: “We are committed to driving innovation for those living with unmet needs. We welcome today’s decision. As the first JAK-inhibitor treatment licensed in Great Britain specifically indicated for adult myelofibrosis patients with moderate to severe anaemia, this milestone could have an impact on this patient population living with myelofibrosis across Great Britain. We look forward to working with all stakeholders to help allow eligible myelofibrosis patients to access this treatment on the NHS as soon as possible.” This MA approval is supported by data from the Momentum phase 3 trial and a subpopulation of adult patients with moderate to severe anaemia (haemoglobin <10 g/dL) from the Simplify-1 phase III trial. The Momentum trial was designed to evaluate the safety profile and efficacy of momelotinib versus danazol for the key manifestations of myelofibrosis (such as splenomegaly, constitutional symptoms, and RBC transfusions) in an anaemic, symptomatic, JAK inhibitor-experienced population. The SIMPLIFY-1 trial was designed to evaluate the efficacy and safety profile of momelotinib versus ruxolitinib in myelofibrosis patients who had not received a prior JAK inhibitor therapy.4