FDA’s Complete Response Letter for droxidopa as a possible orthostatic hypotension treatment highlights importance of Shire’s commitment to complete additional trials to verify and describe the clinical benefit of midodrine
Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, reaffirms its commitment to patients as set forth in its recent agreement with the Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration (FDA) to conduct two additional clinical trials to verify and describe the clinical benefit of midodrine HCI.
Midodrine HCl, approved in 1996 under Subpart H (an accelerated approval process) for the treatment of symptomatic orthostatic hypotension (SOH), will remain available to patients who rely on this medicine while Shire’s trials are conducted. To read the Midodrine Update from FDA please see the FDA website. Currently there are no alternative FDA-approved treatments for SOH.
“Our agreement with the FDA on clinical trials protocols to confirm the clinical benefit of midodrine is a good outcome and in the best interest of patients who rely on this medicine to manage their SOH symptoms,” said Jeffrey Jonas, M.D., Senior Vice President of Research & Development for Shire. “Our agreement is especially important in light of the recent FDA Complete Response Letter for the New Drug Application (NDA) for droxidopa, a competitor investigational product, for the treatment of symptomatic neurogenic orthostatic hypotension in certain patient types. We appreciate the FDA’s agreement to keep midodrine HCI on the market while we conduct the agreed upon trials.”
Shire is the 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 the FDA to develop this now agreed path forward that may permit midodrine to maintain its marketing authorization thus allowing it to remain available for patients who critically need this medicine.
“Even though Shire no longer generates revenue from midodrine, we’ve agreed to invest more time and resources in additional clinical trials because we know it’s the right thing to do for patients,” added Jonas. “Preliminary work on these two midodrine trials is underway and we anticipate completion in the first half of 2014.”
With the 1996 Subpart H accelerated approval of midodrine for the treatment of SOH came a post-approval commitment by Shire to conduct two clinical trials to verify and describe the clinical benefit of midodrine HCI. The initial approval was based on midodrine’s demonstrated ability to significantly raise blood pressure in patients with SOH. In 2000, Shire acquired the medicine and completed two clinical post-marketing trials as required and submitted the results to FDA in 2005. FDA took the position that those two trials were inadequate and requested that additional trials be completed. The agreement of the two additional clinical trials announced here is the outcome of the FDA request following the Shire submission of the 2005 data.
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.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.FDA.gov/medwatch or call 1-800-FDA-1088
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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.
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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.