Issued: 24 June 2024, London
UK
GSK's Omjjara
(momelotinib) approved in Japan for treatment of
myelofibrosis
· Omjjara
approved for use in both newly diagnosed or
previously treated myelofibrosis patients
· Differentiated mechanism of action addresses key
manifestations of myelofibrosis, namely anaemia, constitutional
symptoms and splenomegaly
· In Japan, about 70% of patients diagnosed with primary
myelofibrosis have moderate to severe anaemia at the time of
diagnosis[1],[2],[3]
GSK plc (LSE/NYSE: GSK) today
announced that Japan's Ministry of Health, Labour and Welfare
(MHLW) has approved Omjjara (momelotinib) for the
treatment of myelofibrosis. Omjjara is a once-a-day, oral
JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor. The
approval is based on data from the pivotal phase III MOMENTUM and
SIMPLIFY-1 trials.
This is the fourth major regulatory
approval for GSK's momelotinib in the treatment of myelofibrosis,
following approval under the brand name Ojjaara from the US Food and Drug
Administration and authorisations under the brand name Omjjara from the European Commission
and the Medicines and Healthcare products Regulatory Agency in the
UK.
Nina Mojas, Senior Vice President, Oncology Global Product
Strategy, GSK, said: "Myelofibrosis
has a heavy disease burden, with symptomatic patients experiencing
spleen enlargement, fatigue, night sweats and bone pain, along with
anaemia which can lead to treatment discontinuation and dependence
on regular blood transfusions. With the approval of Omjjara, myelofibrosis patients in
Japan will have a new treatment option for this complex blood
cancer."
Myelofibrosis is a blood cancer that
affects approximately 1 in 500,000 people worldwide, with up to
5,000 patients impacted in Japan.[4],[5],[6] In Japan,
about 70% of patients diagnosed with primary myelofibrosis, and
about half of those patients diagnosed with secondary
myelofibrosis, have moderate to severe anaemia at the time of
diagnosis.1,2,3 Nearly all patients are estimated to develop anaemia over the
course of the disease.[7],[8],[9],[10] Myelofibrosis patients with anaemia require additional
supportive care, including transfusions, and more than 30% will
discontinue treatment with established therapies due to
anaemia.[11]
Patients who are anaemic and transfusion dependent
have a poor prognosis and shortened survival.[12],[13],[14],[15],[16],[17],[18],[19],[20]
The approval is based on data from
the MOMENTUM and SIMPLIFY-1 pivotal phase III trials. MOMENTUM was
designed to evaluate the safety and efficacy of momelotinib versus
danazol for the treatment and reduction of key manifestations of
myelofibrosis in an anaemic, symptomatic, JAK inhibitor-experienced
population. SIMPLIFY-1 was designed to evaluate the efficacy and
safety of momelotinib versus ruxolitinib in myelofibrosis patients
who had not received a prior JAK inhibitor therapy.
About Omjjara
(momelotinib)
Momelotinib has a differentiated
mechanism of action, with inhibitory ability along three key
signalling pathways: Janus kinase (JAK) 1, JAK2, and activin A
receptor, type I (ACVR1).1,[21],[22],[23] Inhibition of JAK1
and JAK2 may improve constitutional symptoms and
splenomegaly.1,21,23 Additionally,
inhibition of ACVR1 leads to a decrease in circulating hepcidin
levels, potentially contributing to anaemia-related
benefit.1,21,22,23
In September 2023, the US Food and
Drug Administration
licensed[24] momelotinib under the brand name Ojjaara for the treatment of
intermediate or high-risk myelofibrosis, including primary
myelofibrosis or secondary myelofibrosis (post-polycythaemia vera
and post-essential thrombocythemia), in adults with
anaemia.
In January 2024, the European
Commission
granted marketing authorisation[25] for Omjjara for 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 thrombocythemia
myelofibrosis and who are Janus kinase (JAK) inhibitor naïve or
have been treated with ruxolitinib. Omjjara was also
approved[26] by the Medicines and Healthcare products Regulatory Agency
(MHRA) in the United Kingdom to treat the symptoms experienced by
adult myelofibrosis patients who have moderate or severe
anaemia.
Please refer to the updated Product
Information (PI) for precautions concerning indication and
important dosage, administration, and safety information in Japan
which will shortly be updated at this link:
Japan
Pharmaceuticals and Medical Devices
Agency[27].
About myelofibrosis
Myelofibrosis is a rare blood cancer
that disrupts the body's normal production of blood cells because
of dysregulated JAK-signal transducer and activator of
transcription protein signalling. The clinical hallmarks of
myelofibrosis are splenomegaly (enlarged spleen), severely low
blood counts, including anaemia and thrombocytopenia, and
debilitating constitutional symptoms, such as fatigue, night sweats
and bone pain, attributable to ineffective haematopoiesis and
excessive production of proinflammatory cytokines.[28],[29]
About the pivotal clinical trials
MOMENTUM was a phase III, global,
multicentre, randomised, double-blind study investigating
momelotinib versus danazol in patients (n=195) with myelofibrosis
who were symptomatic and anaemic and had been previously treated
with a licensed JAK inhibitor. The trial was designed to evaluate
the safety and efficacy of momelotinib for treating and reducing
key hallmarks of the disease: symptoms, blood transfusions (due to
anaemia) and splenomegaly. The MOMENTUM trial met all its primary
and key secondary endpoints, demonstrating statistically
significant response with respect to constitutional symptoms,
splenic reduction and transfusion independence in patients treated
with momelotinib versus danazol (Total Symptom Score reduction of
50% or greater: 25% momelotinib, 9% danazol, p=0.0095; reduction of
spleen volume by 35% or greater: momelotinib 22%, danazol 3%,
p=0.0011; no transfusions and all haemoglobin values ≥8 g/dL in the
12 weeks prior to week 24: momelotinib 30%, danazol
20%).[30] The most common
non-haematological treatment-emergent adverse events in
momelotinib-treated patients over the entire study period as of the
data cutoff were diarrhoea (45 [26%] of 171) and asthenia (28
[16%]); the most common grades 3 and 4 treatment-emergent adverse
events were thrombocytopenia (33 [19%]) and anaemia (19
[11%]).[31] Results from the 24-week
randomised treatment period were presented at the 2022 American
Society of Clinical Oncology (ASCO) Annual Meeting and subsequently
published in The
Lancet,[32],[33] with 48-week data presented at the 64th
American Society of Hematology (ASH) Annual Meeting and Exposition
in December 2022 and subsequently published in The Lancet
Hematology.31,[34]
SIMPLIFY-1 was a multicentre
randomised, double-blind, phase III study that compared the safety
and efficacy of momelotinib to ruxolitinib in patients with
myelofibrosis who had not received prior treatment with a JAK
inhibitor (momelotinib: n=215 and ruxolitinib: n=217).
SIMPLIFY-1 met its primary endpoint, demonstrating non-inferiority
of momelotinib to ruxolitinib in spleen volume response (reduction
by 35% or greater) with a difference of 9% (95% CI 2%-16%),
and substantial improvements in transfusion independence rates
(66.5% for momelotinib compared to 49.3% for ruxolitinib), a
difference of 18% (95% CI 9%-26%).[35],[36] The most common grade 3 or
higher haematologic abnormalities in either group were
thrombocytopenia and anaemia. Grade 3 or higher infections occurred
in 7% of patients who received momelotinib and 3% of patients who
received ruxolitinib.[37]
GSK
in oncology
GSK is committed to maximising
patient survival through transformational medicines, with a current
focus on breakthroughs in immuno-oncology and tumour-cell targeting
therapies, and development in haematologic malignancies,
gynaecologic cancers, and other solid tumours.
About GSK
GSK is a global biopharma company
with a purpose to unite science, technology, and talent to get
ahead of disease together. Find out more at gsk.com.
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2024.
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