Shire and FDA agree on a joint clinical trials proposal to confirm the clinical benefit of midodrine; proposal submitted to FDA Commissioner for approval
PHILADELPHIA, Dec. 2, 2011 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces that it has reached an agreement with the Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration (FDA) to conduct additional clinical trials to confirm the clinical benefit of ProAmatine® (midodrine HCl), a medicine approved in 1996 under Subpart H (an accelerated approval process) for the treatment of symptomatic orthostatic hypotension (SOH).
This joint clinical trials proposal from CDER and Shire is being reviewed by the FDA Commissioner and, if accepted, will negate the need for a public hearing requested by Shire on this topic. Under the agreement, Shire will conduct two additional clinical trials and expects to begin the trials as soon as possible with an anticipated completion by the end of 2014. Midodrine HCl in its generic formulations will remain available to patients who rely on this medicine while the trials are conducted.
"The timeframe outlined in the proposal for additional clinical trials is consistent with Shire's original request and we are appreciative that we have come to agreement with CDER on this path forward," said Jeffrey Jonas, M.D., Senior Vice President of Research & Development for Shire. "We've been committed to completing these additional clinical trials to confirm the efficacy of midodrine as a treatment for SOH and keep this important medication available for patients."
Shire is the New Drug Application (NDA) holder for midodrine HCl, which had been marketed by Shire until 2010 under the brand name ProAmatine. Shire has no financial interest in midodrine, and no longer manufactures, distributes or markets the brand name version of midodrine HCI, ProAmatine. Beginning in 2003, midodrine has been manufactured and distributed by generic pharmaceutical companies. As the NDA holder, Shire has continued to invest in the needed regulatory processes and has worked diligently with FDA to develop a path forward that would allow the NDA to maintain its marketing authorization thus allowing the generic versions of ProAmatine to remain available for patients who critically need this medicine and who have no alternative treatments to manage their SOH symptoms.
ProAmatine was approved in 1996 under Subpart H (an accelerated approval process) for the treatment of SOH, with a post-approval commitment to conduct two clinical trials to confirm the clinical benefit of midodrine. The initial approval was based on ProAmatine's demonstrated ability to significantly raise blood pressure in patients with SOH. In 2000, Shire acquired ProAmatine and completed two clinical post-marketing trials as required and submitted the results to FDA in 2005. FDA took the position that these trials were inadequate and requested that additional trials be completed. Shire disagrees with the FDA and believes that these trials, together with 15 years of clinical experience, have established the clinical efficacy of ProAmatine and provide the necessary data to support the full approval of ProAmatine.
"This Shire agreement with CDER to conduct additional clinical studies that further demonstrate the clinical efficacy of midodrine as a treatment for SOH is in the best interest of patients and we are very pleased for those patients and their families that we have reached this outcome and are hopeful this proposal will be approved," added Jonas.
Important Safety Information
Warning: Because ProAmatine® can cause marked elevation of supine blood pressure, it should be used in patients whose lives are considerably impaired despite standard clinical care. The indication for use of ProAmatine® in the treatment of symptomatic orthostatic hypotension is based primarily on a change in a surrogate marker of effectiveness, an increase in systolic blood pressure measured one minute after standing, a surrogate marker considered likely to correspond to a clinical benefit. At present, however, clinical benefits of ProAmatine®, principally improved ability to carry out activities of daily living, have not been verified.
ProAmatine® is contraindicated in patients with severe organic heart disease, acute renal disease, urinary retention, pheochromocytoma or thyrotoxicosis. ProAmatine® should not be used in patients with persistent and excessive supine hypertension.
Please see Full Prescribing Information.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.FDA.gov/medwatchor call 1-800-FDA-1088
Notes to editors
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