Novartis drug Signifor® recommended by FDA advisory committee for approval to treat patients with Cushing's disease

Novartis drug Signifor® recommended by FDA advisory committee for approval to
                    treat patients with Cushing's disease

- Committee votes unanimously in favor of Signifor (pasireotide) as the first
medication to treat US patients with Cushing's disease

- Pasireotide represents the first targeted approach for this potentially
debilitating endocrine disorder caused by a pituitary tumor that triggers
excess cortisol1,2

- Majority of patients in the Phase III clinical trial experienced a rapid and
sustained decrease in mean cortisol levels with subset of patients achieving

PR Newswire

EAST HANOVER, N.J., Nov. 7, 2012

EAST HANOVER, N.J., Nov. 7, 2012 /PRNewswire/ --The US Food and Drug
Administration's (FDA) Endocrinologic and Metabolic Drugs Advisory Committee
(EMDAC) has voted unanimously in support of the use of
Signifor^®(pasireotide) for the treatment of patients with Cushing's disease
who require medical therapeutic intervention.

"We are encouraged by today's favorable advisory committee recommendation for
pasireotide in Cushing's disease and will work closely with the FDA as it
completes its review of our application," said Herve Hoppenot, President,
Novartis Oncology. "There is a significant unmet medical need for Cushing's
disease patients and Novartis is committed to providing the endocrinology
community with a novel therapeutic approach for this rare and debilitating
endocrine disorder."

The recommendation was based on data from clinical trials of pasireotide,
including PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio - CUSHING'S
disease), the largest randomized Phase III study to evaluate a medical therapy
in patients with Cushing's disease. Although not obliged to follow the
recommendation, the FDA can seek the advice of its advisory committees as it
reviews and decides whether to approve treatments^1,4.

Results from the PASPORT-CUSHINGS study found that mean urinary-free cortisol
(UFC), the key measure of biochemical control of the disease, was rapidly
decreased and sustained in a majority of patients, with a subset of patients
reaching normalized levels. The study also showed that, on average, as UFC
levels were reduced, clinical manifestations of Cushing's disease improved.
The most frequently reported adverse events (AEs) (>/=10%) by investigators
for pasireotide were diarrhea, nausea, hyperglycemia, cholelithiasis,
headache, abdominal pain, fatigue, diabetes mellitus, nasopharyngitis,
alopecia and increased glycosylated hemoglobin (HbA1c), with most events being
Grade 1-2. The safety profile of pasireotide was similar to that of other
somatostatin analogs (SSA) with the exception of the greater degree of

Cushing's syndrome is an endocrine disorder caused by excessive cortisol, a
vital hormone that regulates metabolism, maintains cardiovascular function and
helps the body respond to stress. Cushing's disease is a form of Cushing's
syndrome, in which excess cortisol production is triggered by an
adrenocorticotropic hormone (ACTH)-secreting pituitary adenoma. It is a rare
but serious disease that affects approximately one to two patients per million
per year. Cushing's disease most commonly affects adults as young as 20 to 50
years and affects women three times more often than men. It may present with
weight gain, central obesity, a round, red full face, severe fatigue and
weakness, striae (purple stretch marks), high blood pressure, depression and
anxiety. The first line and most common treatment approach for Cushing's
disease is surgical removal of the tumor^1,2,5,6,7.

About pasireotide

Pasireotide is a multireceptor targeting somatostatin analog (SSA) that binds
with high affinity to four of the five somatostatin receptor subtypes (sst 1,
2, 3 and 5)^2. In April 2012, the European Commission approved pasireotide
under the brand name Signifor ^ for the treatment of adult patients with
Cushing's disease for whom surgery is not an option or for whom surgery has
failed. Other worldwide regulatory filings for pasireotide for this use are
also underway.

For the treatment of Cushing's disease, pasireotide has been studied as a
twice-daily subcutaneous (sc) injection and is currently being evaluated as a
long-acting release (LAR), once-monthly intramuscular (IM) injection as part
of a global Phase III program in Cushing's disease and acromegaly^8,9.

There is no guarantee that pasireotide will become commercially available
anywhere else in the world. As an investigational compound, the safety and
efficacy profile of pasireotide has not yet been established in all countries
for the treatment of Cushing's disease or any other indications. Access to
pasireotide outside of the approved indications has been carefully controlled
and monitored in clinical trials designed to better understand the potential
benefits and risks of the compound.

Information about Novartis clinical trials for pasireotide can be obtained by
healthcare professionals at


The foregoing release contains forward-looking statements that can be
identified by terminology such as "recommended," "potentially," "encouraged,"
"will," "committed," "recommendation," "underway," "potential," or similar
expressions, or by express or implied discussions regarding potential
marketing approvals for Signifor or regarding potential future revenues from
Signifor. 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 Signifor to be materially different
from any future results, performance or achievements expressed or implied by
such statements. There can be no guarantee that Signifor will be approved for
sale in any market, or at any particular time. Nor can there be any guarantee
that Signifor will achieve any particular levels of revenue in the future. In
particular, management's expectations regarding Signifor 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; unexpected manufacturing issues; 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 Pharmaceuticals Corporation researches, develops, manufactures and
markets innovative prescription drugs used to treat a number of diseases and
conditions, including cardiovascular, dermatological, central nervous system,
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1. National Endocrine and Metabolic Diseases Information Service. US
National Institutes of Health. Cushing's Syndrome. Available at:
Accessed October 2012.

2. Pedroncelli, A. Medical Treatment of Cushing's Disease: Somatostatin
Analogues and Pasireotide. Neuroendocrinology. 2010;92(suppl1):120-124.

3. Colao, A. A 12-Month Phase III Study of Pasireotide in Cushing's
Disease. New Engl J Med. 2012; 366(10):914-924.

4. US Food and Drug Administration. The Federal Advisory Committee Act.
Available at
Accessed October 2012.

5. Lindholm, J., et al. Incidence and Late Prognosis of Cushing's
Syndrome: A Population-Based Study. J Clin Endocrinol Metab.

6. Newell-Price, J., et al. The Diagnosis and Differential Diagnosis of
Cushing's Syndrome and Pseudo-Cushing's States. Endocrine

7. Bertanga, X., et al. Cushing's Disease. Best Practice & Research
Clinical Endocrinology & Metabolism. 2009;23:607-623.

8. US National Institutes of Health. Safety and Efficacy of Pasireotide
Long Acting Release (LAR) vs. Octreotide LAR in Patients With Active
Acromegaly. Available at:
Accessed October 2012.

9. US National Institutes of Health. Efficacy and Safety of Pasireotide
Administered Monthly in Patients With Cushing's Disease. Available at: Accessed October 2012.

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