Novartis drug Votubia® approved as first medication in Switzerland for SEGA, a benign brain tumor associated with tuberous sclerosis
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Novartis drug Votubia® approved as first medication in Switzerland for SEGA, a
benign brain tumor associated with tuberous sclerosis
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The issuer is solely responsible for the content of this announcement.
* Subependymal giant cell astrocytomas (SEGAs) associated with tuberous
sclerosis (TS) primarily affect children and adolescents[1],[2]
* Approval based on Phase II US study of 28 patients showing 75% of patients
had 30% or greater reduction in the size of their largest SEGA at six
months[3]
* Prior to the approval of Votubia, brain surgery was the only treatment
option in Switzerland for patients with growing SEGAs[4]
* Worldwide regulatory submissions for everolimus to treat this patient
population are under way; first approval received in the US in 2010 as
Afinitor(® )
Basel, May 11, 2011 - Swissmedic, the Swiss Agency for Therapeutic Products, has
approved Votubia(®) (everolimus) tablets* for the treatment of patients 3 years
of age and older, with subependymal giant cell astrocytoma (SEGA) associated
with tuberous sclerosis (TS), for whom surgery is not a suitable option[3].
Votubia is the first medication approved in Switzerland to treat these patients,
who are primarily children and adolescents[1],[2]. In the US, everolimus is
approved for patients with SEGA under the trade name Afinitor(®) tablets[5].
Tuberous sclerosis is a genetic disorder that may cause benign tumors to form in
vital organs and can affect many different parts of the body, most commonly the
brain[6],[7]. Signs of TS vary depending on which system and which organs are
involved[6]. SEGAs, or benign brain tumors, occur in up to 20% of patients with
TS and may lead to a variety of resulting disorders including seizures, swelling
in the brain, developmental delays and skin lesions[4],[8]. Prior to this
approval, surgery was the only treatment option for Swiss patients with growing
SEGAs associated with TS[4].
The approval is based on a prospective, open-label, single-arm Phase II study of
28 patients. Results showed 75% of patients (21 of 28) experienced a reduction
of 30% or greater in the size of their largest SEGA and 32% (9 of 28)
experienced a reduction of 50% or greater at six months relative to baseline. Of
16 patients with seizures at the start of the study, nine experienced decreases
in seizure frequency, six reported no change and one experienced an increase at
6 months relative to baseline. Facial angiofibromas (red elevated skin lesions)
improved in 87% of patients (13 of 15 evaluated patients) from baseline to six
months[3].
"This approval of Votubia is significant for children and adults who have SEGA
associated with tuberous sclerosis and, until now, have had limited treatment
options," said Hervé Hoppenot, President, Novartis Oncology. "This milestone
represents our first approval in Europe for Votubia and underscores our
commitment to help patients worldwide improve their management of this
difficult-to-treat disease."
Everolimus targets mTOR, a protein that acts as an important regulator of tumor
cell division, blood vessel growth and cell metabolism[9]. Tuberous sclerosis is
caused by defects in the TSC1 and TSC2 genes[6]. When these genes are defective,
mTOR activity is increased, which can cause uncontrolled tumor cell growth and
proliferation, blood vessel growth and altered cellular metabolism, leading to
the formation of benign tumors throughout the body, including the brain[4]. By
inhibiting mTOR activity in this protein pathway, everolimus may reduce cell
proliferation, blood vessel growth and glucose uptake related to SEGA associated
with TS[4].
Regulatory approvals have also been granted in this disease setting in the
United States, Brazil, Guatemala and the Philippines. Submissions to the
European Medicines Agency (EMA) and other global regulatory agencies are under
review.
Tuberous sclerosis affects approximately one to two million people worldwide[6].
In Europe, the prevalence in the general population is estimated to be nearly
nine cases per 100,000[10]. SEGAs occur in up to 20% of patients with TS[8].
About the Phase II study
In a prospective, open-label, single-arm study, 28 patients aged three years and
above (median age=11, range 3-34) with evidence of SEGA growth initially
received everolimus orally at a dose of 3 mg/m[2]( )daily or every other day. In
total, 16 of the 28 patients were treated with Votubia for at least
21 months[3].
In the study, 75% of patients (21 of 28) experienced a reduction of 30% or
greater in the size of their largest SEGA and 32% (9 of 28) experienced a
reduction of 50% or greater at six months relative to baseline[3].
Of 16 patients with seizures at the start of the study for whom 24-hour video
electroencephalograms (EEG) were available, nine experienced decreases in
seizure frequency, six reported no change and one experienced an increase at six
months relative to baseline. Facial angiofibromas improved in 87% of patients
(13 of 15 evaluated patients) from baseline to six months. None of the patients
developed new symptoms of intracranial pressure or an increase in hydrocephalus
(swelling in the brain). No patient underwent surgery[3].
The most common adverse events reported (incidence >=30%) in the prospective,
open-label, single-arm trial were mouth sores, upper respiratory tract
infections, sinusitis, middle ear infections and fever[3]. However, the
reliability of the frequency of adverse reactions and laboratory abnormalities
reported in this trial is limited because of the small number of patients.
All data from the study submitted to Swissmedic are based on the cut-off date of
December 9, 2009.
About everolimus
Votubia(®) (everolimus) tablets is approved in Switzerland for the treatment of
patients 3 years of age and older, with SEGA associated with tuberous sclerosis
(TS), for whom surgery is not a suitable option. Should everolimus be approved
in the EU, the trade name will be Votubia. In the US, Afinitor(®) (everolimus)
tablets is approved to treat patients with SEGA associated with tuberous
sclerosis who require therapeutic intervention but are not candidates for
curative surgical resection. The effectiveness of everolimus is based on an
analysis of change in SEGA volume. Clinical benefit such as improvement in
disease-related symptoms or increase in overall survival has not been shown.
Afinitor is approved in the US for the treatment of progressive neuroendocrine
tumors of pancreatic origin in patients with unresectable, locally advanced or
metastatic disease. The FDA determined that the safety and effectiveness of
Afinitor in the treatment of patients with carcinoid tumors have not been
established.
Afinitor is approved in the European Union (EU) for the treatment of patients
with advanced renal cell carcinoma (RCC) whose disease has progressed on or
after treatment with vascular endothelial growth factor (VEGF)-targeted therapy
and also in the US for the treatment of patients with advanced RCC after failure
of treatment with sunitinib or sorafenib.
In the EU, everolimus is available in different dosage strengths for the non-
oncology patient population under the trade name Certican(®) for the prevention
of organ rejection in heart and kidney transplant recipients. In the US,
everolimus is available in different dosage strengths under the trade name
Zortress(®) for the prophylaxis of organ rejection in adult patients at low-
moderate immunologic risk receiving a kidney transplant.
Everolimus is exclusively licensed to Abbott and sublicensed to Boston
Scientific for use in drug-eluting stents.
Not all indications are available in every country. Because of the uncertainty
of clinical trials, there is no guarantee that everolimus will become
commercially available for SEGAs anywhere else in the world.
Important Safety Information about Votubia/Afinitor
Votubia can cause serious side effects including lung or breathing problems,
infections, and renal failure which can lead to death. Mouth ulcers and mouth
sores are common side effects. Votubia can affect blood cell counts, kidney and
liver function, blood sugar and cholesterol levels. Votubia may cause fetal harm
in pregnant women. Women taking Votubia should not breast feed.
The most common adverse drug reactions (incidence >=15%) are mouth ulcers, rash,
diarrhea, fatigue, acneiform dermatitis, infections, weakness, nausea,
peripheral swelling, decreased appetite, headache, pneumonitis, abnormal taste,
nose bleeds, mucosal inflammation, weight decreased and vomiting. The most
common grade 3-4 adverse drug reactions (incidence >=2%) are mouth ulcers,
fatigue, decreased white blood cell count, diarrhea, infections, pneumonitis and
diabetes mellitus. Cases of hepatitis B reactivation and pulmonary embolism have
been reported.
Disclaimer
The foregoing release contains forward-looking statements that can be identified
by terminology such as "commitment," "will," or similar expressions, or by
express or implied discussions regarding potential new indications or labeling
for everolimus or regarding potential future revenues from everolimus. You
should not place undue reliance on these statements. Such forward-looking
statements reflect the current views of management regarding future events, and
involve known and unknown risks, uncertainties and other factors that may cause
actual results with everolimus to be materially different from any future
results, performance or achievements expressed or implied by such statements.
There can be no guarantee that everolimus will be submitted or approved for any
additional indications or labeling in any market. Nor can there be any guarantee
that everolimus will achieve any particular levels of revenue in the future. In
particular, management's expectations regarding everolimus could be affected by,
among other things, unexpected regulatory actions or delays or government
regulation generally; unexpected clinical trial results, including unexpected
new clinical data and unexpected additional analysis of existing clinical data;
government, industry and general public pricing pressures; competition in
general; the company's ability to obtain or maintain patent or other proprietary
intellectual property protection; the impact that the foregoing factors could
have on the values attributed to the Novartis Group's assets and liabilities as
recorded in the Group's consolidated balance sheet, and other risks and factors
referred to in Novartis AG's current Form 20-F on file with the US Securities
and Exchange Commission. Should one or more of these risks or uncertainties
materialize, or should underlying assumptions prove incorrect, actual results
may vary materially from those anticipated, believed, estimated or expected.
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 healthcare solutions that address the evolving needs of
patients and societies. Focused solely on healthcare, Novartis offers a
diversified portfolio to best meet these needs: innovative medicines, eye care,
cost-saving generic pharmaceuticals, consumer health products, preventive
vaccines and diagnostic tools. Novartis is the only company with leading
positions in these areas. In 2010, the Group's continuing operations achieved
net sales of USD 50.6 billion, while approximately USD 9.1 billion (USD 8.1
billion excluding impairment and amortization charges) was invested in R&D
throughout the Group. Headquartered in Basel, Switzerland, Novartis Group
companies employ approximately 119,000 full-time-equivalent associates and
operate in more than 140 countries around the world. For more information,
please visithttp://www.novartis.com.
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* Known as Afinitor(®) (everolimus) tablets for this patient population outside
Switzerland. If approved in the EU for this patient population, the trade name
will be Votubia.
References
1. Nabbout R, et al. Early diagnosis of subependymal giant cell astrocytoma in
children with tuberous sclerosis. J Neurol Neurosurg Psychiatry 1999;66:370-375.
2. Medkour A, et al. Neonatal Subependymal Giant Cell Astrocytoma. Pediatr
Neurosurg 2002;36:271-274.
3. Votubia Swiss Prescribing Information. Novartis. May 2011.
4. Krueger, et al. Everolimus for Subependymal Giant-Cell Astrocytomas in
Tuberous Sclerosis. New Eng J Med 2010;363:1801-11.
5. Afinitor US Prescribing
Information.http://www.pharma.us.novartis.com/product/pi/pdf/afinitor.pdf.
Accessed May 2011.
6. National Institute of Neurological Disorders and Stroke. Tuberous Sclerosis
Fact Sheet. Available
athttp://www.ninds.nih.gov/disorders/tuberous_sclerosis/detail_tuberous_sclerosi
s.htm. Accessed May 2011.
7. Inoki, et al. Tuberous sclerosis complex, implication from a rare genetic
disease to common cancer treatment. HM Genetics, 2009; 18:R94-R100.
8. Adriaensen ME, et al. Prevalence of subependymal giant cell tumors in
patients with tuberous sclerosis and a review of the literature. Eur J Neurol
2009;16:691-6.
9. Motzer, et. al. Phase 3 Trial of Everolimus for Metastatic Renal Cell
Carcinoma. Cancer 2010 Sep;116(18):4256-4265.
10. Orphanet Report Series."Prevalence of rare diseases: Bibliographic Data."
Available
athttp://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_a
lphabetical_list.pdf. Accessed May 2011.
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Datum: 11.05.2011 - 07:15 Uhr
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