REG - GSK PLC - Omjjara approved in Japan for myelofibrosis

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24/06/2024 07:15

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RNS Number : 4903T  GSK PLC  24 June 2024

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  (#_edn1)
))(,( 2  (#_edn2) )),( 3  (#_edn3) )

 

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  (#_edn4) ))(,( 5 
(#_edn5) )), 6  (#_edn6) 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  (#_edn7) (, 8  (#_edn8) , 9  (#_edn9) , 10 
(#_edn10) ) Myelofibrosis patients with anaemia require additional supportive
care, including transfusions, and more than 30% will discontinue treatment
with established therapies due to anaemia.(( 11  (#_edn11) )) Patients who are
anaemic and transfusion dependent have a poor prognosis and shortened
survival. 12  (#_edn12) (, 13  (#_edn13) , 14  (#_edn14) , 15  (#_edn15) , 16 
(#_edn16) , 17  (#_edn17) , 18  (#_edn18) , 19  (#_edn19) , 20  (#_edn20) )

 

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  (#_edn21) , 22  (#_edn22) , 23  (#_edn23) )
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
(https://www.gsk.com/en-gb/media/press-releases/ojjaara-momelotinib-approved-in-the-us-as-the-first-and-only-treatment-indicated-for-myelofibrosis-patients-with-anaemia/)
 24  (#_edn24) 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
(https://www.gsk.com/en-gb/media/press-releases/european-commission-authorises-gsk-s-omjjara-momelotinib/)
 25  (#_edn25) 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
(https://www.gov.uk/government/news/omjjara-licensed-for-anaemic-myelofibrosis-patients-to-treat-the-symptoms-of-their-disease)
 26  (#_edn26) 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
(https://www.info.pmda.go.jp/psearch/html/menu_tenpu_base.html)  27  (#_edn27)
.

 

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  (#_edn28) (, 29  (#_edn29) )

 

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  (#_edn30) 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  (#_edn31)
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  (#_edn32) (, 33  (#_edn33) ) 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  (#_edn34) )

 

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  (#_edn35) (,) 36  (#_edn36) 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 
(#_edn37)

 

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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GSK's Q1 Results for 2024.

 

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 22  (#_ednref22) Asshoff M, et al. Momelotinib inhibits ACVR1/ALK2, decreases
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 24  (#_ednref24) GSK press release issued 15 September 2023: Ojjaara
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 26  (#_ednref26) MHRA press release issued 31 January 2024: Omjjara licensed
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