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

Friday, February 24, 2017 General News
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ZUG, Switzerland, February 24, 2017 /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).

For further information please contact: 

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 

Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products, are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire's results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:

  • Shire's products may not be a commercial success;
  • increased pricing pressures and limits on patient access as a result of governmental regulations and market developments may affect Shire's future revenues, financial condition and results of operations;
  • Shire conducts its own manufacturing operations for certain of its products and is reliant on third party contract manufacturers to manufacture other products and to provide goods and services. Some of Shire's products or ingredients are only available from a single approved source for manufacture. Any disruption to the supply chain for any of Shire's products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time;
  • the manufacture of Shire's products is subject to extensive oversight by various regulatory agencies. Regulatory approvals or interventions associated with changes to manufacturing sites, ingredients or manufacturing processes could lead to, among other things, significant delays, an increase in operating costs, lost product sales, an interruption of research activities or the delay of new product launches;
  • certain of Shire's therapies involve lengthy and complex processes, which may prevent Shire from timely responding to market forces and effectively managing its production capacity;
  • Shire has a portfolio of products in various stages of research and 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 approval;
  • the actions of certain customers could affect Shire's ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely affect Shire's revenues, financial conditions or results of operations;
  • Shire's products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
  • adverse outcomes in legal matters, tax audits and other disputes, including Shire's ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the combined company's revenues, financial condition or results of operations;
  • inability to successfully compete for highly qualified personnel from other companies and organizations;
  • failure to achieve the strategic objectives, including expected operating efficiencies, cost savings, revenue enhancements, synergies or other benefits at the time anticipated or at all with respect to Shire's acquisition of NPS Pharmaceuticals Inc., Dyax Corp. or Baxalta Incorporated may adversely affect Shire's financial condition and results of operations;
  • Shire's growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
  • a slowdown of global economic growth, or economic instability of countries in which Shire does business, as well as changes in foreign currency exchange rates and interest rates, that adversely impact the availability and cost of credit and customer purchasing and payment patterns, including the collectability of customer accounts receivable;
  • failure of a marketed product to work effectively or if such a product is the cause of adverse side effects could result in damage to Shire's reputation, the withdrawal of the product and legal action against Shire;
  • investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire's activities in the highly regulated markets in which it operates may result in significant legal costs and the payment of substantial compensation or fines;
  • Shire is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on Shire's revenues, financial condition or results of operations;
  • Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which may decrease its business flexibility and increase borrowing costs; and

a further list and description of risks, uncertainties and other matters can be found in Shire's most recent Annual Report on Form 10-K and in Shire's subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in "ITEM 1A: Risk Factors", and in subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire's website.

All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to update or revise forward-looking statements, whether as a result of new information, future events or otherwise.

References 

  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

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

 

SOURCE Shire plc



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