Issued: 29 January 2024, London
UK
European Commission authorises GSK's
Omjjara
(momelotinib)
· Omjjara
is the first medicine in the EU specifically
indicated for treating splenomegaly (enlarged spleen) or
symptoms in adult myelofibrosis patients with moderate to severe
anaemia
· Authorisation may address high unmet need, with nearly all
myelofibrosis patients estimated to develop anaemia over
the course of the disease1,2,3,4
· Indicated in both newly diagnosed patients and those
previously treated with existing standard of
care
GSK plc (LSE/NYSE: GSK) today
announced the European Commission* granted marketing authorisation
for Omjjara (momelotinib),
a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1)
inhibitor. Omjjara is the
first authorised medicine in the EU 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 thrombocythaemia
myelofibrosis and who are Janus kinase (JAK) inhibitor naïve or
have been treated with ruxolitinib.
Nina
Mojas, Senior Vice President, Oncology Global Product Strategy,
GSK, said: "The challenges of living
with myelofibrosis can be burdensome, and symptomatic patients can
experience spleen enlargement, fatigue, night sweats and bone pain.
Until now, there have been no options specifically indicated to
treat these symptoms in patients who also experience anaemia. The
authorisation of Omjjara
brings a new treatment option with a differentiated mechanism of
action to these patients in the EU."
Myelofibrosis is estimated to affect
1 in 10,000 people in the EU.5,6 About 40% of patients have
moderate to severe anaemia at the time of diagnosis and nearly all
patients are estimated to develop anaemia over the course of the
disease.1,2,3,4 Myelofibrosis
patients with anaemia require additional supportive care, including
transfusions, and more than 30% will discontinue treatment due to
anaemia.7 Patients who are
transfusion dependent have a poor prognosis and shortened
survival.8,9,10,11,12,13,14,15,16
Francesca Palandri, MD, PhD, IRCCS S. Orsola-Malpighi, Bologna
University Hospital, Italy, said: "The EU authorisation of Omjjara represents a meaningful
advancement for eligible patients with myelofibrosis, and
particularly for those with moderate to severe anaemia who need new
treatment options that may reduce their disease-related burden. The
availability of a single therapy for key manifestations of
myelofibrosis is a clear step forward for eligible
patients."
The authorisation of momelotinib is
based on the MOMENTUM pivotal phase III trial and a subpopulation
of adult patients with moderate to severe anaemia (haemoglobin
<10 g/dL) from the SIMPLIFY-1 phase III
trial.17,18 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.
In these clinical trials, the most
common adverse reactions were diarrhoea, thrombocytopaenia, nausea,
headache, dizziness, fatigue, asthenia, abdominal pain, and
cough.17,18
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).8,17,19,20
Inhibition of JAK1 and JAK2 may improve
constitutional symptoms and splenomegaly.8,17,20 Additionally,
inhibition of ACVR1 leads to a decrease in circulating hepcidin
levels, potentially contributing to anaemia-related
benefit.8,17,19,20
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/)
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 thrombocythaemia), in adults with
anaemia.
Important Information for Omjjara in the EU
Indication
Omjjara is indicated 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.
Refer to the Omjjara EMA Reference Information
(https://www.ema.europa.eu/en/medicines/human/EPAR/omjjara)
for a full list of adverse events and the complete important safety
information in the EU.
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), progressive
anaemia and debilitating constitutional symptoms, such as fatigue,
night sweats and bone pain, attributable to ineffective
haematopoiesis and excessive production of proinflammatory
cytokines.21
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%).17 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,22,23 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.24,25
SIMPLIFY-1 was a multicentre,
randomised, double-blind, phase III study that compared the safety
and efficacy of momelotinib to ruxolitinib in patients (n=432) with
myelofibrosis who had not received prior treatment with a JAK
inhibitor. Safety and efficacy results for SIMPLIFY-1 were based
upon a subset of patients (n=181) with anaemia at baseline. The
efficacy of momelotinib in the treatment of patients with
myelofibrosis in SIMPLIFY-1 was based on spleen volume response
(reduction of spleen volume by 35% or greater: 31% momelotinib, 33%
ruxolitinib p = 0.026).18
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.
* The European Commission has the
authority to authorise medicines for European Union member states,
as well as in the European Economic Area (EEA) countries Iceland,
Norway and Liechtenstein, and Northern Ireland.
GSK
enquiries
|
|
|
|
Media:
|
Tim Foley
|
+44 (0) 20 8047 5502
|
(London)
|
|
Dan Smith
|
+44 (0) 20 8047 5502
|
(London)
|
|
Kathleen Quinn
|
+1 202 603 5003
|
(Washington DC)
|
|
Lyndsay Meyer
|
+1 202 302 4595
|
(Washington DC)
|
|
Alison Hunt
|
+1 540 742 3391
|
(Washington DC)
|
|
|
|
|
Investor Relations:
|
Nick Stone
|
+44 (0) 7717 618834
|
(London)
|
|
James Dodwell
|
+44 (0) 20 8047 2406
|
(London)
|
|
Mick Readey
|
+44 (0) 7990 339653
|
(London)
|
|
Josh Williams
|
+44 (0) 7385 415719
|
(London)
|
|
Camilla Campbell
|
+44 (0) 7803 050238
|
(London)
|
|
Steph Mountifield
|
+44 (0) 7796 707505
|
(London)
|
|
Jeff McLaughlin
|
+1 215 751 7002
|
(Philadelphia)
|
|
Frannie DeFranco
|
+1 215 751 4855
|
(Philadelphia)
|
Cautionary statement regarding forward-looking
statements
GSK cautions
investors that any forward-looking statements or projections made
by GSK, including those made in this announcement, are subject to
risks and uncertainties that may cause actual results to differ
materially from those projected. Such factors include, but are not
limited to, those described under Item 3.D "Risk factors" in the
company's Annual Report on Form 20-F for 2022, and Q3 Results for
2023.
Registered in England & Wales:
No. 3888792
Registered Office:
980 Great West Road
Brentford, Middlesex
TW8 9GS
[1] Tefferi A, Lasho
TL, Jimma T, et al. One thousand patients
with primary myelofibrosis: the mayo clinic experience.
Mayo Clin Proc.
2012;87(1):25-33. doi:10.1016/j.mayocp.2011.11.001
[2] Bose P, et al.
Curr Hematol Malign Rep.
2018;13:164-172. doi:
https://doi.org/10.3109/10428194.2013.813500
[3] Scherber, RM,
Mesa, R. Management of challenging myelofibrosis after JAK
inhibitor failure and/or progression. Blood Rev. 2020;42:100716.
https://doi.org/10.1016/j.blre.2020.100716
[4] Bassiony S,
Harrison CN, McLornan DP. Evaluating the Safety, Efficacy, and
Therapeutic Potential of Momelotinib in the Treatment of
Intermediate/High-Risk Myelofibrosis: Evidence to Date.
Ther Clin Risk Manag.
2020;16:889-901. Published 2020 Sep 25.
doi:10.2147/TCRM.S258704
[5] Orphanet. Primary
Myelofibrosis. 2019. Accessed 01 February 2023.
https://www.orpha.net
[6] Junker, S, et al.
"PB2203: INCIDENCE AND PREVALENCE OF MYELOFIBROSIS IN GERMANY: A
RETROSPECTIVE CLAIMS DATA ANALYSIS." HemaSphere
7.S3 (2023): e32222e6.
[7] Kuykendall AT,
Shah S, Talati C, et al. Between a rux and a hard place: evaluating
salvage treatment and outcomes in myelofibrosis after ruxolitinib
discontinuation. Ann
Hematol. 2018;97(3):435-441.
[8] Chifotides, HT,
Bose, P, Verstovsek, S. Momelotinib: an emerging treatment for
myelofibrosis patients with anemia. J Hematol Oncol.
2022;15(7):1-18.
[9] Tefferi A, et al.
Use of the Functional Assessment of Cancer Therapy--anemia in
persons with myeloproliferative neoplasm-associated myelofibrosis
and anemia. Clin Ther.
2014;36(4):560-566.
https://doi.org/10.1016/j.clinthera.2014.02.016
[10] Tefferi A.
Primary myelofibrosis: 2021 update on diagnosis,
risk-stratification and management. Am J
Hematol. 2021;96(1):145-162.
https://doi.org/10.1002/ajh.26050
[11] Rumi E, et
al. The Genetic Basis of Primary
Myelofibrosis and Its Clinical Relevance. Int J Mol Sci. 2020;21(23):8885.
https://doi.org/10.3390/ijms21238885
[12] How J,
Hobbs GS. A Practical Guide for Using Myelofibrosis Prognostic
Models in the Clinic. J Natl
Compr Canc Netw. 2020;18(9):1271-1278.
https://doi.org/10.6004/jnccn.2020.7557
[13] QxMD. DIPSS
prognosis in myelofibrosis. Accessed September 12, 2022.
https://qxmd.com/calculate/calculator_187/dipss-prognosis-in-myelofibrosis.
[14] QxMD. DIPSS
plus score for prognosis of myelofibrosis. Accessed September 12,
2022.
[15] Nicolosi M,
et al. Sex and degree of severity influence the prognostic impact
of anemia in primary myelofibrosis: analysis based on 1109
consecutive patients. Leukemia. 2018;32(5):1254-1258.
https://doi.org/10.1038/s41375-018-0028-x
[16] Elena C, et
al. Red blood cell transfusion-dependency implies a poor survival
in primary myelofibrosis irrespective of IPSS and DIPSS.
Haematologica.
2011;96(1):167-170.
https://doi.org/10.3324/haematol.2010.031831
[17] Verstovsek
S, et al. MOMENTUM: momelotinib vs danazol in patients with
myelofibrosis previously treated with JAKi who are symptomatic and
anemic. Future Oncol.
2021;17(12):1449-1458.
[18] Mesa RA,
Kiladjian JJ, Catalano JV, et al. SIMPLIFY-1: A Phase III
Randomized Trial of Momelotinib Versus Ruxolitinib in Janus Kinase
Inhibitor-Naïve Patients With Myelofibrosis. J Clin Oncol.
2017;35(34):3844-3850.
[19] Asshoff M,
et al. Momelotinib inhibits ACVR1/ALK2, decreases hepcidin
production, and ameliorates anemia of chronic disease in rodents.
Blood.
2017;129(13):1823-1830.
[20] Oh S, et
al. ACVR1/JAK1/JAK2 inhibitor momelotinib reverses transfusion
dependency and suppresses hepcidin in myelofibrosis phase 2 trial.
Blood Adv.
2020;4(18):4282-4291.
[21] Atallah E,
Verstovsek S. Emerging drugs for myelofibrosis. Expert Opin Emerg Drugs. 2012
Dec;17(4):555-70. doi: 10.1517/14728214.2012.748748. PMID:
23186315; PMCID: PMC5009610.
[22] Mesa R, et
al. Presented at: American Society of Clinical Oncology; June 2022.
Abstract 7002.
[23] Verstovsek
S, et al. Momelotinib versus danazol in symptomatic patients with
anaemia and myelofibrosis (MOMENTUM): results from an
international, double-blind, randomised, controlled, phase 3 study.
The Lancet.
2023;401(10373):269-280.
[24] Gerds AT,
et al. Presented at: American Society of Hematology; December 2022.
Abstract 627.
[25] Gerds AT,
et al. Momelotinib versus danazol in symptomatic patients with
anaemia and myelofibrosis previously treated with a JAK inhibitor
(MOMENTUM): an updated analysis of an international, double-blind,
randomised phase 3 study. The
Lancet Haematology. 2023;10(9):E735-E746.
https://doi.org/10.1016/S2352-3026(23)00174-6