DUBLIN, Nov. 8, 2018 /PRNewswire/ -- Jazz Pharmaceuticals
plc (Nasdaq: JAZZ) today announced that the National Institute for
Health and Care Excellence (NICE) has published a Final
Appraisal Determination (FAD) recommending Vyxeos® 44
mg/100 mg powder for concentrate for solution for infusion for
routine use on the National Health Service (NHS) in England and Wales for the treatment of adults with newly
diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML
with myelodysplasia-related changes (AML-MRC)1 – two
types of secondary AML.
"This is the first new chemotherapy in forty years for adults
with specific types of newly diagnosed secondary AML, a
particularly aggressive cancer that typically affects older people
and has a high mortality rate," said Dr. Nigel Russell,
Professor of Haematology, Faculty of Medicine & Health Sciences
at the University of Nottingham. "I am pleased that NICE has
recognised the value of this medicine for adults with secondary
AML. In time, it is expected to become the standard of care for
this specific group of older AML patients."
Patients diagnosed with t-AML or AML-MRC have a very poor
prognosis and have the poorest survival of all AML diagnostic
subgroups.2,3 Thus they are classified as having high
risk disease because of these poor outcomes. In the UK, the
expected number of these high-risk AML cases is 680 per
year.4 Its incidence increases with age and accounts for
approximately 25% of all AML cases in the UK.4
"A diagnosis of acute myeloid leukaemia can have a huge
emotional impact on the lives of patients, as well as their family
and friends," said Zack
Pemberton-Whiteley, Patient Advocacy Director at Leukaemia
Care. "We welcome the decision to recommend this treatment for
adults with high-risk AML. Leukaemia Care has been working with
NICE to enable patients to access this important new medicine,
where options have been previously limited."
"Jazz is delighted that Vyxeos will now be made routinely
available on the NHS in England
and Wales as people with
therapy-related AML or AML with myelodysplasia-related changes have
had limited treatment options until now," said Iain McGill, senior vice president, Europe and Rest of World at Jazz
Pharmaceuticals. "We believe that it is a meaningful
medicine for patients with this rapidly progressing and
life-threatening blood cancer."
Vyxeos is an advanced liposomal formulation that
delivers a synergistic molar ratio of daunorubicin and cytarabine.
It is the first chemotherapy to demonstrate a significant
overall survival advantage versus the current treatment standard,
7+3 chemotherapy, in a Phase 3 study of older adult patients with
newly diagnosed t-AML or AML-MRC,5 and the first
chemotherapy treatment option specifically for people who have
these types of high-risk AML.
Notes to Editors
About Vyxeos®
It is an advanced liposomal
formulation that has been shown in vitro to deliver a synergistic
combination of daunorubicin and cytarabine to leukaemia cells for a
prolonged period of time. Based on data in animals, the liposomes
accumulate and persist in high concentration in the bone marrow,
where they are preferentially taken up intact by leukaemia
cells.6 It is the first product developed with the
company's proprietary CombiPlex® platform, which enables
the consideration and design of various combinations of
therapies.
It received Orphan Drug Designation (ODD) by the European
Commission in January 2012 with
retention of the ODD reaffirmed in July
2018 following assessment by the Committee for Orphan
Medicinal Products (COMP), and by the U.S. Food and Drug
Administration (FDA) in September
2008 for the treatment of AML. It received Promising
Innovative Medicine (PIM) designation from the Medicines and
Healthcare Products Regulatory Agency in the United Kingdom. In August 2018, the European Commission approved it
for the treatment of adults with newly diagnosed t-AML or AML-MRC
in all European Union Member States, as well as
Iceland, Norway and Liechtenstein.
View the Summary of Product Characteristics here.
About CombiPlex®
The CombiPlex proprietary
technology enables the design and rapid evaluation of various
combinations of therapies to deliver enhanced anti-cancer activity.
The CombiPlex technology seeks to identify the most synergistic
ratio of drugs in vitro and fix this ratio in a nano-scale delivery
complex that maintains the synergistic combination after
administration. CombiPlex utilizes two proprietary nano-scale
delivery platforms: liposomes to control the release and
distribution of water-soluble drugs and drugs that are both water-
and fat-soluble (amphipathic), and nanoparticles to control the
release and distribution of non-water-soluble (hydrophobic)
drugs.
About AML
Acute myeloid leukaemia (AML) is a blood
cancer that begins in the bone marrow, which produces most of the
body's new blood cells. AML cells crowd out healthy cells and move
into the bloodstream to spread cancer to other parts of the
body.7 The median age at diagnosis is 72 years old, with
rising age associated with a progressively worsening
prognosis.4 There is also a reduced tolerance for
intensive chemotherapy as patients age.8 Patients with
t-AML or AML-MRC have the lowest survival rates compared with
people with other forms of leukaemia.2,3 A
haematopoietic stem cell transplant (HSCT) may be a curative
treatment option for patients.9
About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals
plc (Nasdaq: JAZZ) is an international biopharmaceutical company
focused on improving patients' lives by identifying, developing and
commercializing meaningful products that address unmet medical
needs. The company has a diverse portfolio of products and
product candidates with a focus in the areas of sleep and
hematology/oncology.
"Safe Harbor" Statement under the Private Securities
Litigation Reform Act of 1995
This press release contains
forward-looking statements, including, but not limited to,
statements related to Vyxeos becoming the standard of care
for patients with newly diagnosed t-AML or AML-MRC, and other
statements that are not historical facts. These forward-looking
statements are based on the company's current plans, objectives,
estimates, expectations and intentions and inherently involve
significant risks and uncertainties. Actual results and the
timing of events could differ materially from those anticipated in
such forward-looking statements as a result of these risks and
uncertainties, which include, without limitation, risks and
uncertainties associated with: the company's ability to effectively
commercialize Vyxeos; delays or problems in the supply or
manufacture of Vyxeos; maintaining appropriate pricing and
reimbursement for Vyxeos; complying with applicable
regulatory requirements; and other risks and uncertainties
affecting the company, including those described from time to time
under the caption "Risk Factors" and elsewhere in Jazz
Pharmaceuticals plc's Securities and Exchange Commission filings
and reports (Commission File No. 001-33500), including the
company's Quarterly Report on Form 10-Q for the quarter ended
September 30, 2018 and future filings
and reports by the company. Other risks and uncertainties of which
the company is not currently aware may also affect the company's
forward-looking statements and may cause actual results and the
timing of events to differ materially from those anticipated. The
forward-looking statements herein are made only as of the date
hereof or as of the dates indicated in the forward-looking
statements, even if they are subsequently made available by the
company on its website or otherwise. The company undertakes no
obligation to update or supplement any forward-looking statements
to reflect actual results, new information, future events, changes
in its expectations or other circumstances that exist after the
date as of which the forward-looking statements were made.
References:
1. NICE Final Appraisal Document Vyxeos
November 2018 www.nice.org.uk
2. Roman E et al. Myeloid malignancies in the real-world:
Occurrence, progression and survival in the UK's population-based
Haematological Malignancy Research Network. 2004-2015. Cancer
Epidemiology 2016; 42:186-198.
3. Boddu P et al. Treated Secondary Acute Myeloid Leukemia: A
Distinct High-Risk Subset of AML with Adverse Prognosis. The
American Society of Hematology. 2017; 1(17)
4. HMRN Incidence. AML. Available at:
https://www.hmrn.org/statistics/incidence Last accessed
October 2018
5. Lancet JE et al. CPX-351 (cytarabine and daunorubicin) Liposome
for Injection Versus Conventional Cytarabine Plus Daunorubicin in
Older Patients with Newly Diagnosed Secondary Acute Myeloid
Leukemia. J Clin Oncol. 2018; 36(26):2684-2692.
6. Vyxeos Summary of Characteristics Available at
https://www.medicines.org.uk/emc/product/9442 Last accessed
October 2018
7. National Cancer Institute. General Information About Adult Acute
Myeloid Leukemia
https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq
Accessed July 23, 2018
8. Klepin HD. Hematology Am Soc Hematol Educ Program. 2014;
(1):8-13
9. Peccatori J and Ciceri F. Haematologica. 2010; 95(6):
857–859. doi: 10.3324/haematol. 2010.023184.
Date of preparation: October
2018
Job bag number: EURW-UKVYX-0058
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