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of Contents
11
Neuromuscular Diseases
SMA is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and
progressive muscular atrophy and weakness. Ultimately, individuals with the most severe type of SMA can become
paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing. Due to a loss of, or
defect in, the SMN1 gene, people with SMA do not produce enough survival motor neuron (SMN) protein, which is
critical for the maintenance of motor neurons. The severity of SMA correlates with the amount of SMN protein.
People with Type 1 SMA, the most severe life-threatening form, produce very little SMN protein and do not achieve
the ability to sit without support or live beyond two years without respiratory support. People with Type 2 and Type 3
SMA produce greater amounts of SMN protein and have less severe, but still life-altering, forms of SMA.
In December 2016 the FDA approved SPINRAZA for the treatment of SMA in pediatric and adult patients. In
June 2017 the EC approved SPINRAZA for the treatment of SMA in pediatric and adult patients in the E.U. The
Japanese Ministry of Health, Labor and Welfare approved SPINRAZA for the treatment of infantile SMA in July 2017
and for the treatment of pediatric and adult patients with SMA in September 2017.
Our products for SMA and major markets include:
Product
Indication
Collaborator
Major Markets
SMA
Ionis
U.S.
France
Germany
Japan
Turkey
Biosimilars
Biosimilars are a group of biologic medicines that are similar to currently available biologic therapies known as
originators. Under our
agreement with Samsung Bioepis, we manufacture and commercialize two anti-TNF biosimilars
in certain countries in the E.U.: BENEPALI, an etanercept biosimilar referencing ENBREL, and FLIXABI, an infliximab
biosimilar referencing REMICADE. In August 2017 the EC granted a marketing authorization for IMRALDI, an
adalimumab biosimilar referencing HUMIRA, in the E.U.
Product
Indication
Major Markets
Moderate to severe rheumatoid arthritis
Progressive psoriatic arthritis
Axial spondyloarthritis
Moderate
to severe plaque psoriasis
Germany
Norway
Sweden
U.K.
Rheumatoid arthritis
Moderate to severe Crohn's disease
Severe ulcerative colitis
Severe ankylosing spondylitis
Psoriatic arthritis
Moderate to severe plaque psoriasis
Germany
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13
Patient Support and Access
We interact with patients, advocacy
organizations and healthcare societies in order to gain
insights into unmet needs. The insights gained from
these engagements help us support patients with
services, programs and applications that are designed
to help patients lead better lives. Among other things,
we provide customer service and other related
programs for our products, such as disease and
product specific websites, insurance research
services, financial assistance programs and the
facilitation of the
procurement of our marketed
products.
We are dedicated to helping patients obtain
access to our therapies. Our patient representatives
have access to a comprehensive suite of financial
assistance tools. With those tools, we help patients
understand their insurance coverage and, if needed,
help patients compare and select new insurance
options and programs. In the U.S., we have
established programs that provide co-pay assistance
or free marketed product for qualified uninsured or
underinsured patients, based on specific eligibility
criteria. We also provide charitable contributions to
independent charitable organizations that assist
patients with out-of-pocket expenses associated with
their therapy.
Marketing
and Distribution
Sales Force and Marketing
We promote our products worldwide, including in
the U.S., most of the major countries of the E.U. and
Japan, primarily through our own sales forces and
marketing groups. In some countries, particularly in
areas where we continue to expand into new
geographic areas, we partner with third parties.
We co-promote ZINBRYTA with AbbVie in the
U.S., E.U. and Canadian territories and BENEPALI and
FLIXABI with Samsung Bioepis in certain countries in
the E.U.
We and Eisai co-promote AVONEX, TYSABRI and
TECFIDERA in Japan in certain settings.
RITUXAN, GAZYVA and OCREVUS are marketed
by the Roche Group and its sublicensees.
We focus our sales and marketing efforts on
specialist physicians in private practice or at major
medical centers. We use customary pharmaceutical
company practices to market our products and to
educate
physicians, such as sales representatives
calling on individual physicians, advertisements,
professional symposia, direct mail, public relations
and other methods.
Distribution Arrangements
We distribute our products in the U.S. principally
through wholesale distributors of pharmaceutical
products, mail order specialty distributors or shipping
service providers. In other countries, the distribution
of our products varies from country to country,
including through wholesale distributors of
pharmaceutical products and third-party distribution
partners who are responsible for most marketing and
distribution activities.
AbbVie distributes ZINBRYTA in the U.S., and we
distribute ZINBRYTA in ex-U.S. markets.
We distribute BENEPALI and FLIXABI in certain
countries in the E.U.
Eisai distributes AVONEX, TYSABRI, TECFIDERA
and PLEGRIDY in India
and other Asia-Pacific markets,
excluding China.
RITUXAN, GAZYVA and OCREVUS are distributed
by the Roche Group and its sublicensees.
Our product sales to two wholesale distributors,
AmerisourceBergen and McKesson, each accounted
for more than 10% of our total revenues for the years
ended December 31, 2017, 2016 and 2015, and on
a combined basis, accounted for approximately 56%,
57% and 60% of our gross product revenues for the
years ended December 31, 2017, 2016 and 2015,
respectively. For additional information, please read
Note 25, Segment Information, to our consolidated
financial statements included in this report.
Patents and Other Proprietary Rights
Patents are important to obtaining and
protecting exclusive rights in our products and product
candidates. We regularly seek patent protection in the
U.S. and in selected countries outside the U.S. for
inventions originating
from our research and
development efforts. In addition, we license rights to
various patents and patent applications.
U.S. patents, as well as most foreign patents,
are generally effective for 20 years from the date the
earliest application was filed; however, U.S. patents
that issue on applications filed before June 8,
1995 may be effective until 17 years from the issue
date, if that is later than the 20-year date. In some
cases, the patent term may be extended to recapture
a portion of the term lost during regulatory review of
the claimed therapeutic or, in the case of the U.S.,
because of U.S. Patent and Trademark Office (USPTO)
delays in prosecuting the application. Specifically, in
the U.S., under the Drug Price Competition and Patent
Term Restoration Act of 1984, commonly known as
the
Hatch-Waxman Act, a patent that covers an FDA-
approved drug may be eligible for patent term