Novartis drug Revolade® recommended by CHMP for EU approval to treat patients with severe aplastic anemia, a serious blood disorder
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Novartis drug Revolade® recommended by CHMP for EU approval to treat patients
with severe aplastic anemia, a serious blood disorder
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* If approved, Revolade would be the first treatment option in its class in
the EU for certain patients with SAA
* Approximately 40% of SAA patients unresponsive to initial immunosuppressive
therapy (IST) die from infection or bleeding within five years of
diagnosis[1]
* No approved therapies exist for patients with SAA who have not responded to
IST or are ineligible for hematopoietic stem cell transplantation[2]
Basel, July 24, 2015 - The Committee for Medicinal Products for Human Use (CHMP)
of the European Medicines Agency (EMA) adopted a positive opinion for
Revolade(®) (eltrombopag) for the treatment of adult patients with severe
aplastic anemia (SAA) who have had an insufficient response to immunosuppressive
therapy (IST) and are not eligible to receive a hematopoietic stem cell
transplant.
"Today's CHMP opinion marks a key milestone towards Revolade becoming the first
approved therapy in its class in the European Union for patients with severe
aplastic anemia who have not responded to or are ineligible for existing
treatments," said Alessandro Riva, MD, Global Head, Novartis Oncology
Development and Medical Affairs. "Our commitment to oncology includes continuing
to work towards new treatments for patients impacted by rare diseases like SAA,
especially when limited options exist."
SAA is a blood disorder where the bone marrow fails to make enough red blood
cells, white blood cells and platelets[3]. Two out of every one million people
in Europe and North America are diagnosed with aplastic anemia per year, a
portion of which are severe cases[4],[5]. The exact cause of the disease is
still unknown, but most cases of SAA are believed to be triggered by an
autoimmune reaction where the body attacks blood-forming stem cells located in
the bone marrow[3],[6]. As a result, patients with SAA are at risk for life-
threatening infections or bleeding[6].
Treatment of SAA is focused on increasing a patient's blood cell count. The
current standard of care includes IST or hematopoietic stem cell
transplantation[6]. Of patients treated with IST, one-quarter to one-third will
not respond and 30-40% of responders will relapse, causing symptoms to
return[6]. Approximately 40% of SAA patients who don't respond to initial IST
die from infection or bleeding within five years of their diagnosis[1].
The CHMP positive opinion is based on the results of a pivotal open-label Phase
II study (ELT112523) and two supporting Phase II studies (ELT116826 and
ELT116643) conducted by the National Heart, Lung and Blood Institute (NHLBI) at
the National Institutes of Health (NIH) . The pivotal study demonstrated a
hematologic response (40%) in SAA patients treated with Revolade who had an
insufficient response to IST. The most common adverse reactions (>=20%) in the
pivotal single-arm study of 43 patients were nausea (33%), fatigue (28%), cough
(23%), diarrhea (21%), and headache (21%)[7].
The European Commission will review the CHMP recommendation and is expected to
deliver its final decision within three months. The decision will be applicable
to all 28 EU member states plus Iceland, Norway and Liechtenstein. In August of
2014, eltrombopag (marketed as Promacta(®) in the USA), was approved by the US
Food and Drug Administration for once-daily use in patients with SAA who have
had an insufficient response to IST. Eltrombopag is also approved for SAA in
Canada.
About the NIH Study
In the single-arm, single-center, open-label Phase II study (NCT00922883),
eltrombopag was evaluated in 43 patients with SAA who have had an insufficient
response to at least one prior IST and who had a platelet count <=30 x
10(9)/L[8]. At baseline, the median platelet count was 20 x 10(9)/L, hemoglobin
was 8.4 g/dL, absolute neutrophil count (ANC) was 0.58 x 10(9)/L, and absolute
reticulocyte count was 24.3 x 10(9)/L[8]. The treated population had a median
age of 45 years (range 17 to 77 years) and 56% were male. The majority of
patients (84%) received at least two prior immunosuppressive therapies[8].
Eltrombopag was administered at an initial dose of 50 mg once daily for two
weeks and increased over two-week periods up to a maximum dose of 150 mg once
daily[8].The primary endpoint was hematologic response, which was initially
assessed after 12 weeks of treatment with eltrombopag[8]. Treatment was
discontinued after 16 weeks if no hematologic response was observed. Additional
efficacy assessments included median duration of response in months[8].
About Revolade(®) (eltrombopag)
Revolade is approved in more than 100 countries worldwide for the treatment of
thrombocytopenia in adult patients with chronic immune (idiopathic)
thrombocytopenic purpura (ITP) who have had an inadequate response or are
intolerant to other treatments, and in over 45 countries worldwide for the
treatment of thrombocytopenia (low blood platelet counts) in patients with
chronic hepatitis C to allow them to initiate and maintain interferon-based
therapy. Eltrombopag (marketed as Promacta(®) in the USA), is approved by the US
Food and Drug Administration for once-daily use in patients with SAA who have
had an insufficient response to IST and was also recently approved for the
treatment of children six years and older with chronic ITP who have had an
insufficient response to corticosteroids, immunoglobulins or splenectomy.
Important Safety Information for Revolade(®) (eltrombopag)
Revolade may cause serious side effects, such as liver problems, high platelet
counts and a higher chance for blood clots, bleeding after stopping treatment,
and bone marrow problems.
Revolade may damage the liver and cause serious, even life threatening, illness.
Blood tests to check the liver are needed before taking Revolade and during
treatment. When certain antiviral treatments are given together with Revolade
for the treatment of thrombocytopenia due to hepatitis C virus (HCV) infections,
some liver problems can get worse.
A doctor will order the blood tests and any other tests required. In some cases
Revolade treatment may need to be stopped. Patients should tell a doctor right
away if they have any of these signs and symptoms of liver problems: yellowing
of the skin or the whites of the eyes (jaundice), unusual darkening of the
urine, unusual tiredness, right upper stomach area pain.
Patients have a higher chance of getting a blood clot if their platelet count is
too high during treatment with Revolade, but blood clots can occur with normal
or even low platelet counts. Patients who have cirrhosis of the liver are at
risk of a blood clot in a blood vessel that feeds the liver. Patients may have
severe complications from some forms of blood clots, such as clots that travel
to the lungs or that cause heart attacks or strokes. A doctor will check the
patient's blood platelet counts, and change the dose or stop Revolade if
platelet counts get too high. Patients should tell their doctor right away if
they have signs and symptoms of a blood clot in the leg, such as swelling or
pain/tenderness of one leg.
When patients stop taking Revolade, their blood platelet count will drop back
down to what it was before they started taking Revolade. These effects are most
likely to happen within 4 weeks after patients stop taking Revolade. The lower
platelet counts may increase risk of bleeding. A doctor will check platelet
counts for at least 4 weeks after patients stop taking Revolade. Patients should
tell their doctor or pharmacist if they have any bruising or bleeding after they
stop taking Revolade.
Patients being treated for the disease may have problems with their bone marrow.
Medicines like Revolade could make this problem worse. Signs of bone marrow
changes may show up as abnormal results in blood tests. A doctor may also carry
out tests to directly check the bone marrow during treatment with Revolade.
The most common side effects of Revolade when used to treat patients with
chronic ITP include nausea, diarrhea, increase of liver enzymes, dry mouth,
vomiting, unusual hair loss or thinning, rash, back pain, muscle pain, sore
throat and discomfort when swallowing, urinary tract infection.
The most common side effects of Revolade when used to treat patients with
chronic HCV and antiviral agents include fever, feeling very tired, chills,
headache, cough, nausea, diarrhea, unusual hair loss or thinning, muscle pain,
itching, feeling weak, difficulty sleeping, loss of appetite, flu-like symptoms,
and swelling of the hands, ankles or feet.
The most common side effects of Revolade when used to treat patients with severe
aplastic anemia (SAA) include cough, headache, shortness of breath, pain in the
nose and throat, runny nose, abdominal pain, diarrhea, bruising, joint pain,
muscle spasms, pain in extremities, dizziness, feeling very tired, fever,
inability to sleep (insomnia). Common side effects that may show up in blood
tests include increase in some liver enzymes and laboratory tests that may show
abnormal changes to the cells in the bone marrow.
Please see full EU Summary of Product Characteristics for Revolade
(eltrombopag).
Disclaimer
The foregoing release contains forward-looking statements that can be identified
by words such as "recommended," "would," "positive opinion," "milestone,"
"commitment," "continuing," "will," "expected to," or similar terms, or by
express or implied discussions regarding potential new indications or labeling
for Revolade, or regarding potential future revenues from Revolade and Promacta.
You should not place undue reliance on these statements. Such forward-looking
statements are based on the current beliefs and expectations of management
regarding future events, and are subject to significant known and unknown risks
and uncertainties. Should one or more of these risks or uncertainties
materialize, or should underlying assumptions prove incorrect, actual results
may vary materially from those set forth in the forward-looking statements.
There can be no guarantee that Revolade will be submitted or approved for any
additional indications or labeling in any market, or at any particular time. Nor
can there be any guarantee that Revolade or Promacta will be commercially
successful in the future. In particular, management's expectations regarding
Revolade and Promacta could be affected by, among other things, the
uncertainties inherent in research and development, including unexpected
clinical trial results and additional analysis of existing clinical data;
unexpected regulatory actions or delays or government regulation generally; the
company's ability to obtain or maintain proprietary intellectual property
protection; general economic and industry conditions; global trends toward
health care cost containment, including ongoing pricing pressures; unexpected
manufacturing issues, and other risks and factors referred to in Novartis AG's
current Form 20-F on file with the US Securities and Exchange Commission.
Novartis is providing the information in this press release as of this date and
does not undertake any obligation to update any forward-looking statements
contained in this press release as a result of new information, future events or
otherwise.
About Novartis
Novartis provides innovative healthcare solutions that address the evolving
needs of patients and societies. Headquartered in Basel, Switzerland, Novartis
offers a diversified portfolio to best meet these needs: innovative medicines,
eye care and cost-saving generic pharmaceuticals. Novartis is the only global
company with leading positions in these areas. In 2014, the Group achieved net
sales of USD 58.0 billion, while R&D throughout the Group amounted to
approximately USD 9.9 billion (USD 9.6 billion excluding impairment and
amortization charges). Novartis Group companies employ approximately 120,000
full-time-equivalent associates. Novartis products are available in more than
180 countries around the world. For more information, please visit
http://www.novartis.com.
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References
[1] Valdez JM, Scheinberg P, Nunez O, et al. Decreased infection-related
mortality and improved survival in severe aplastic anemia in the past two
decades. Clin Infect Dis. 2011; 52(6):726-735.
[2] Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood.
2012;120(6):1185-1196.
[3] National Heart, Lung and Blood Institute. What is aplastic anemia? Accessed
on May 26, 2015. Available at: http://www.nhlbi.nih.gov/health/health-
topics/topics/aplastic.
[4] Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia.
Haematologica. 2008; 93(4):489-492.
[5] Montané E, Ibáñez L, Vidal X, et al. Epidemiology of aplastic anemia: a
prospective multicenter study. Haematologica. 2008; 93(4):518-523.
[6] Townsley DM, Desmond R, Dunbar CE, et al. Pathophysiology and management of
thrombocytopenia in bone marrow failure: possible clinical applications of TPO
receptor agonists in aplastic anemia and myelodysplastic syndromes. Int J
Hematology. 2013; 98(1):48-55.
[7] Promacta Prescribing Information.
[8] US National Institutes of Health. ClinicalTrials.gov Identifier:
NCT00922883. A pilot study of a thrombopoietin-receptor agonist (TPO-R agonist),
eltrombopag, in aplastic anemia patients with immunosuppressive-therapy
refractory thrombocytopenia. Accessed on May 26 2015. Available at:
https://clinicaltrials.gov/ct2/show/NCT00922883?term=eltrombopag+and+saa&rank=1.
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Datum: 24.07.2015 - 13:12 Uhr
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