Global
Rare Disease Drugs Market is impacted by various key trends which are
driven by supply as well as demand side. Wide gap between market needs and
potential market offerings is the main driving force behind an increasing
interest of the manufacturers towards providing therapies in this segment of
the healthcare industry. According to the U.S. Food & Drug Administration
(FDA), in 2016, 582 requests for orphan drug designation were filed by the
biopharma companies. Furthermore, the sales of orphan drug diseases has been on
a rise due to availability of highly effective drug therapies. Moreover,
potential drug therapies are present in pipeline for various cancers and rare
diseases such as Niraparib by Tesaro, Inc. in phase I for the treatment of
ovarian cancer, Emicizumab by Roche in phase III for the treatment of
hemophilia, and LentiGlobin by Bluebird Bio in different phases for the
treatment of sickle cell anemia and beta-thalassemia major. Positive results
from these clinical trials are expected to change the outlook of the global
rare disease drugs market over the foreseeable future.
Rare diseases or orphan diseases
are conditions that affect a small percentage of population at any given time.
Orphan drug designation is given to the drugs indicated for the treatment of
rare diseases. According to the U.S. Food & Drug Administration (FDA), an
orphan disease is defined as a disease or condition affecting less than 200,000
people in the country. Orphan Drugs Act under the U.S. legislation allows
manufacturer to request FDA to grant an orphan drug status to drugs intended to
treat a rare disease or condition. According to the FDA, 2017, number of orphan
drug designation requests have steadily increased over the past few years.
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The global rare disease drugs
market was not considered lucrative until recently, owing to various
uncertainties associated with these diseases. Small percentage of affected
population provided limited opportunity for study. Moreover, diseases were
poorly understood, owing to huge differentiation and heterogeneity among the
group of diseases which discouraged manufacturers to address unmet needs in
this space. However, introduction of some highly priced orphan drugs e.g.
spinal muscular atrophy drug Spinraza priced at US$ 125,000 per vial, and high
unmet needs has drawn attention of manufacturers towards rare disease drugs
market.
Novartis AG is a global
pharmaceutical organization with a diverse product folio ranging from drug
therapies for cancer treatment to ophthalmic products. The company operates
business through Innovative Medicines Division, which consists of oncology
business unit and Novartis pharmaceutical business units. Innovative medicines
Oncology division offers products for oncology diseases and rare diseases, and
pharmaceutical business consists of ophthalmology, neuroscience, immunology and
dermatology, respiratory, cardio-metabolic, and established medicines. Novartis
lost patent protection for its orphan drug Gleevec for the treatment of chronic
myeloid leukemia (CML) in 2015 and Sun Pharma brought out generic Gleevec in
2016 hampering the sales of Novartis for Gleevec.
Celgene Corporation specializes
in therapies for cancer treatment and inflammatory diseases which includes
drugs such as Revlimid, Pomalyst, and Otezla. The company currently is
concentrated on expanding the indication range of the present therapies and has
a robust pipeline for oncology conditions. For instance, Revlimid
(lenalidomide), in phase III clinical trials for diffuse large B-cell lymphoma,
indolent lymphoma, and follicular lymphoma.
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Furthermore, the company has
developed its own target-identification and drug-discovery technology platforms
which enable the company to proceed rapidly from target identification and
validation to lead identification and optimization.
Shire Plc. became global leader
in orphan drug products category (excluding oncology products) in year 2016 and
grossed almost US$ 11 billion. The company acquired Baxalta in 2016, and its
three therapeutic segments namely hematology, immunology, and oncology. Shire
Plc. is also focused on providing therapies for rare diseases as it invested
US$ 1.4 billion in research and development. The company had 37 therapies in
research and the pipeline has further been strengthened by acquisition of Dyax
and Baxalta in 2016.
This study provides a detailed
competitive landscape and competitive analysis for leading players in this
market. This will the aid one in understanding the operations of successful
companies in this market and their long term goals.
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