CHMP Recommends EU Conditional Marketing Authorisation for Natpar® (Parathyroid Hormone) for Patients with Chronic Hypoparathyroidism

Zug, Switzerland (ots/PRNewswire) -

This Press Release is intended for Global Use

If approved, Natpar would be the first licensed recombinant
parathyroid hormone in Europe for the management of chronic
hypoparathyroidism, the only endocrine-deficiency disorder with no
hormone treatment[1]

Shire plc (LSE: SHP, NASDAQ: SHPG) announces that the Committee
for Medicinal Products for Human Use (CHMP) of the European Medicines
Agency (EMA) has adopted a positive opinion recommending the granting
of Conditional Marketing Authorisation for Natpar (rhPTH[1-84]), a
recombinant human protein with the full length 84-amino-acid sequence
of endogenous parathyroid hormone (PTH), as an adjunctive treatment
for adult patients with chronic hypoparathyroidism who cannot be
adequately controlled with standard therapy alone.

Hypoparathyroidism, a designated orphan disease by the European
Commission (EC), is a rare disease that occurs when inadequate levels
of PTH are secreted by the parathyroid glands, resulting in a mineral
imbalance in the body expressed by a low concentration of calcium
(hypocalcemia) and high concentration of phosphate
(hyperphosphatemia) in the blood.

"This endorsement by the EMA is a critical step towards addressing
an unmet need for patients with chronic hypoparathyroidism," said
Philip J. Vickers, Ph.D., Head of R&D, Shire. "As a leader in rare
diseases, we are committed to advancing therapeutic options for
patients with hypoparathyroidism, and we believe that by offering the
first recombinant PTH in hypoparathyroidism, we can help patients who
are inadequately controlled on standard therapy alone to manage their
condition."

The EC will now consider the CHMP positive opinion in its final
decision on whether to provide Conditional Marketing Authorisation
for Natpar; a final decision from the EC is expected later in 2017.

"Hypoparathyroidism is the last endocrine disorder for which there
is no hormone treatment" said Liz Glenister, Chief Executive of a
hypoparathyroid patient organization. "Currently, there is a high
unmet medical need in patients in the EU who may not be adequately
controlled in managing their hypoparathyroidism; those patients are
looking forward to this new potential option in the management of
their disease."

Supportive Data

The CHMP submission was based on outcomes from the Phase III
efficacy and safety of rhPTH(1-84) in hypoparathyroidism clinical
trial (REPLACE) in patients aged 18-85 years with chronic
hypoparathyroidism. In the double-blind, placebo-controlled,
randomized Phase III study 134 patients with hypoparathyroidism were
randomized in a ratio of 2:1 to either placebo or 50 micrograms once
daily of rhPTH(1-84) for 24 weeks. The primary endpoint was a 50
percent or greater reduction from baseline in their daily dose of
oral calcium and active vitamin D while maintaining a stable albumin
corrected serum calcium concentration greater than or equal to
baseline concentration (baseline was 2.12 mmol/L for the rhPTH(1-84)
group and 2.15 mmol/L for the placebo group) and less or equal to the
upper limit of normal (normal range 2.1-2.6 mmol/L) but ideally
within the target range of 2.0-2.25 mmol/L. At the end of the
follow-up period, 53 percent of the patients on rhPTH(1-84) achieved
the primary endpoint compared with 2 percent of patients in the
placebo group (p<0.0001).[2]

The overall incidences of adverse events were similar in both
groups. By week 24, 93 percent of patients in the rhPTH(1-84) and 100
percent of patients in the placebo group had at least one adverse
event, with hypocalcemia, muscle spasm, paraesthesias, headache, and
nausea being the most common adverse events. Ten (11 percent)
patients in the rhPTH(1-84) group and four (9 percent) in the placebo
group had serious adverse events; only one serious adverse event
(hypercalcemia requiring a brief hospital stay) in the rhPTH(1-84)
group was regarded as treatment related.[2]

About Natpar

Natpar is a recombinant human parathyroid hormone (PTH), which if
approved will be available as a 25, 50, 75 and 100 micrograms
once-daily injection as adjunctive treatment of adult patients with
chronic hypoparathyroidism who cannot be adequately controlled with
standard therapy alone.

Natpar is approved in the United States under the trade name
Natpara® (parathyroid hormone).

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result of governmental regulations and market developments may
affect Shire's future revenues, financial condition and results of
operations;
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products and is reliant on third party contract manufacturers to
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things, significant delays, an increase in operating costs, lost
product sales, an interruption of research activities or the delay
of new product launches;
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which may prevent Shire from timely responding to market forces and
effectively managing its production capacity;
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development. The successful development of these products is highly
uncertain and requires significant expenditures and time, and there
is no guarantee that these products will receive regulatory
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Corp. or Baxalta Incorporated may adversely affect Shire's
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to Shire's reputation, the withdrawal of the product and legal
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condition or results of operations;
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Baxalta acquisition, which may decrease its business flexibility
and increase borrowing costs; and

1. Bilezikian JP, et al. J Bone Miner Res. 2011;26:2317-2337
2. Mannstadt M, et al. Lancet Diabetes Endocrinol. 2013;1:275-283

ots Originaltext: Shire Pharmaceuticals Group Plc
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NOTES TO EDITORS
About Shire
Shire is the leading global biotechnology company focused on serving
people with rare diseases and other highly specialized conditions. We
strive to develop best-in-class products, many of which are available
in more than 100 countries, across core therapeutic areas including
Hematology, Immunology, Neuroscience, Ophthalmics, Lysosomal Storage
Disorders, Gastrointestinal / Internal Medicine / Endocrine and
Hereditary Angioedema; and a growing franchise in Oncology.
Our employees come to work every day with a shared mission: to develop
and deliver breakthrough therapies for the hundreds of millions of
people in the world affected by rare diseases and other high-need
conditions, and who lack effective therapies to live their lives to
the fullest.
http://www.shire.com
Forward-Looking Statements
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without limitation statements concerning future strategy, plans,
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a further list and description of risks, uncertainties and other
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References
Investor Relations
Ian Karp
ikarp@shire.com
+1-781-482-9018

Robert Coates
rcoates@shire.com
+44-1256-894874

Media
Annabel Cowper
annabel.cowper@shire.com
+41-44-878-6638

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