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Shire plc Release: One Year Data Provide Evidence of Effectiveness and Tolerability of REPLAGAL(R) (agalsidase alfa) in Fabry Patients


3/30/2012 8:44:18 AM

NYON, Switzerland, March 29, 2012 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, presented data that show favorable treatment effect and tolerability of REPLAGAL® (agalsidase alfa) in Fabry patients who switched from Fabrazyme® (agalsidase beta) or were naive to enzyme replacement therapy (ERT) after one year of treatment with REPLAGAL at the America College of Medical Genetics (ACMG) annual meeting in Charlotte, NC.

Fabry disease is a rare, X-linked, lysosomal storage disorder caused by insufficient activity of the alpha-galactosidase enzyme. As a result of this deficiency, patients experience a spectrum of signs and symptoms including impairment in renal and cardiac functions. The natural course of Fabry disease includes decrease of renal function and increase in left ventricular mass index (LVMI).

"It's common for Fabry patients to suffer from chronic kidney and cardiovascular disease; these are signs of disease progression," said Dr. Ozlem Goker-Alpan, Director at Lysosomal Disorders Research and Treatment Unit, Center for Clinical Trials, Fairfax, VA. "These interim results show the effectiveness and tolerability of REPLAGAL in Fabry patients after one year of treatment."

Data Suggest Favorable Treatment Effect and Tolerability with REPLAGAL at the Recommended Dose of 0.2 mg/kg Body Weight after One Year of Treatment

One year data from Shire's HGT-REP-059 multicenter, open-label treatment protocol, suggests that cardiac structure, as measured by LVMI, remained stable in treatment-naive and switch patients after one year of treatment with REPLAGAL. In treatment naive patients (n=22), mean LVMI was 48.5 g/m(2.7) at baseline and 50.7 g/m(2.7) at 12 months, an increase of 2.20 g/m(2.7) +/- 1.63; p=0.187. In switch patients (n=39), mean LVMI was 60.4 g/m(2.7) at baseline and remained virtually unchanged at 12 months at 60.3 g/m(2.7) (0.00 +/- 2.32; p=0.306). The small increases of LVMI observed in the study were not statistically significant and were below the progression expected from natural history data.

The data show no significant decline in renal function as measured by estimated glomerular filtration rate (eGFR) in treatment-naive patients (n=29); the mean change from baseline, 83.85 +/- 7.12 mL/min/1.73m(2), to 12 months was -1.29 +/- 4.34 mL/min/1.73 m(2), p=0.175. Results were similar in switch patients (n=62), the mean change from baseline, 81.96 +/- 4.57 mL/min/1.73m(2), to 12 months was -3.17 +/- 1.69 mL/min/1.73m(2), p=0.009.

Biologic activity by reduction of pathological substrate globotriaosylceramide (Gb(3)) was also demonstrated by decline in both plasma Gb(3) (-2.60 +/- 0.52 nmol/mL; p<0.001 and -6.70 +/- 1.18 nmol/mL; p<0.001 for switch [n=56] and treatment naive patients [n=25] respectively) and creatinine normalized urine Gb(3) (-0.87 +/- 0.37 nmol/mg; p=0.009 and -2.11 nmol/mg; p=0.021) for switch and treatment naive patients respectively.

The data suggest that REPLAGAL was generally well tolerated. Safety and tolerability was consistent with the known safety profile of REPLAGAL and no new safety concerns emerged in naive or switch patients over one year of treatment. The safety of REPLAGAL has been well established in over 15 years of clinical and commercial experience.

This study was established to provide US Fabry patients with access to REPLAGAL during the Fabrazyme supply shortage. More than 2,800 Fabry patients are being treated with REPLAGAL globally.

About REPLAGAL (agalsidase alfa)
REPLAGAL is a human form of enzyme alpha-galactosidase A (alpha-Gal A) manufactured in a human cell line by gene activation. 2012 marks over 15 years of clinical experience with REPLAGAL, which is now approved in 46 countries worldwide. REPLAGAL is not approved for commercial sale in the US.

REPLAGAL is the only human-cell-line-derived form of enzyme replacement therapy that is indicated for the long-term treatment of patients with a confirmed diagnosis of Fabry disease (alpha-Gal A deficiency).

REPLAGAL Important Safety Information
The most serious adverse reactions seen with REPLAGAL were hypersensitivity reactions. Infusion-related reactions were the most commonly observed adverse reactions in patients treated with REPLAGAL in clinical studies. Most side effects are mild to moderate and include headache, tingling, numbness, tremors, fatigue, change in temperature sensation, increased blood pressure, upset stomach, diarrhea, coughing, sore throat, difficulty sleeping, change in the taste of food, change in smell, difficulty speaking, acne, dry skin and eye problems. About 1 out of 10 patients may have a reaction during or shortly after infusion of REPLAGAL. These effects include chills and facial flushing (warmth and redness).

As with all therapeutic proteins, there is a potential for immunogenicity. IgG antibodies appeared to develop following approximately 3 to 12 months of treatment. After 12 to 54 months of therapy, 17% of REPLAGAL treated patients were antibody positive whereas 7% showed evidence for the development of immunologic tolerance, based on the disappearance of IgG antibodies over time. No IgE antibodies have been detected in any patient receiving REPLAGAL.

REPLAGAL is not available in all countries and prescribing information may differ between countries. Please consult your local prescribing information.

SHIRE PLC
Shire's strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder, human genetic therapies, gastrointestinal diseases and regenerative medicine as well as opportunities in other therapeutic areas to the extent they arise through acquisitions. Shire's in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights. Shire believes that a carefully selected and balanced portfolio of products with strategically aligned and relatively small-scale sales forces will deliver strong results.

For further information on Shire, please visit the Company's website: www.shire.com.

"SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995

Statements included herein that are not historical facts 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, the Company's results could be materially adversely affected. The risks and uncertainties include, but are not limited to, risks associated with: the inherent uncertainty of research, development, approval, reimbursement, manufacturing and commercialization of the Company's Specialty Pharmaceuticals, Human Genetic Therapies and Regenerative Medicine products, as well as the ability to secure new products for commercialization and/or development; government regulation of the Company's products; the Company's ability to manufacture its products in sufficient quantities to meet demand; the impact of competitive therapies on the Company's products; the Company's ability to register, maintain and enforce patents and other intellectual property rights relating to its products; the Company's ability to obtain and maintain government and other third-party reimbursement for its products; and other risks and uncertainties detailed from time to time in the Company's filings with the Securities and Exchange Commission.

SOURCE Shire plc



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