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FDA Grants Orphan Drug Designation to Sonus Pharmaceuticals' TOCOSOL Paclitaxel in Bladder Cancer Indication

Posted on: Thursday, 27 January 2005, 09:00 CST

Company conducting Phase 2b bladder cancer clinical trials in U.S. and Europe

Sonus Pharmaceuticals, Inc. (Nasdaq:SNUS) today announced that the U.S. Food and Drug Administration (FDA) has granted an Orphan Drug designation to its lead oncology candidate, TOCOSOL(R) Paclitaxel, for the treatment of non-superficial urothelial cancer. The most common form of urothelial cancer is bladder cancer. The Orphan Drug designation adds to the Fast Track designation awarded in 2003 by the FDA Division of Oncology Drug Products for the development of TOCOSOL Paclitaxel for the treatment of metastatic or locally advanced, inoperable transitional cell carcinoma (TCC) of the urothelium. TOCOSOL Paclitaxel is a novel formulation of paclitaxel, a widely prescribed anti-cancer drug for the treatment of solid tumors.

In parallel with developing TOCOSOL Paclitaxel for the treatment of bladder cancer, Sonus is pursuing initial market entry through a 505(b)(2) New Drug Application in an indication for which paclitaxel-based products have already been approved, e.g. breast, ovarian or lung cancers. The Company is currently in discussions with the FDA to finalize the pivotal Phase 3 protocol for TOCOSOL Paclitaxel, which Sonus believes will be initiated in 2005.

"We have been pursuing the Phase 2b program since the end of 2003 to demonstrate the value of TOCOSOL Paclitaxel for treating bladder cancer," said Michael B. Stewart, M.D., Chief Medical Officer of Sonus Pharmaceuticals. "By granting both Orphan Drug and Fast Track status to TOCOSOL Paclitaxel, the FDA recognizes the potential of our drug to treat advanced cancers of the bladder and urinary system -- an uncommon, but important medical need. Currently available treatments are not particularly effective and/or have serious side effects. Despite the challenge of performing rigorously controlled clinical trials in a disease with a small patient population, we believe that addressing this need is an important component of the overall development objective for TOCOSOL Paclitaxel, and we are pleased that the FDA has agreed with us."

The Company is conducting Phase 2b bladder cancer studies in Cleveland, Philadelphia, Baltimore and Seattle, and will soon be opening sites in Spain and the U.K. In addition to the bladder cancer program, Sonus is also conducting a Phase 2b study of TOCOSOL Paclitaxel in patients with metastatic breast cancer.

Sonus has completed patient enrollment in Phase 2a studies of TOCOSOL Paclitaxel in bladder, ovarian and lung cancer patients, and these trials have generated encouraging data on safety and anti-tumor activity. The Company reported results of the Phase 2a studies in three presentations at the annual meeting of the American Society of Clinical Oncology (ASCO) in 2004. TOCOSOL Paclitaxel offers the convenience of a ready-to-use formulation, which does not require reconstitution, dilution or pharmacy preparation as is required with the marketed paclitaxel products. Additionally, TOCOSOL Paclitaxel is administered to patients in a short 15-minute infusion, compared to the three-hour infusion required with Taxol(R), the innovator paclitaxel product.

About FDA Orphan Drug and Fast Track Designations

Products designated as orphan drugs are those that are being developed to treat diseases affecting fewer than 200,000 people in the United States. The Orphan Drug Act guarantees seven years of market exclusivity to the first sponsor that obtains market approval for an orphan-designated product. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a waiver from the FDA user fee for review of an application for the indication designated.

The FDA's Fast Track program is intended to facilitate the development and review of new drugs intended to treat serious or life-threatening conditions, and for which the product has the potential to address an unmet medical need.

About Sonus Pharmaceuticals, Inc.

Headquartered near Seattle, Sonus Pharmaceuticals, Inc. is focused on the development of novel drugs for the treatment of cancer that may offer improved administration, tolerability, safety and effectiveness. The Company's lead product is TOCOSOL Paclitaxel, a novel formulation of the leading anti-cancer drug paclitaxel. TOCOSOL Paclitaxel has been designed to overcome the limitations associated with Taxol and generic paclitaxel-based chemotherapy, including long infusion times, undesirable or treatment-limiting side effects as well as time consuming and expensive preparation prior to administration. With patient enrollment complete in Phase 2a studies of TOCOSOL Paclitaxel in non-small cell lung, bladder and ovarian cancers, and Phase 2b studies ongoing in bladder and breast cancers, Sonus continues to advance the product toward Phase 3 testing. In addition to executing its plans for TOCOSOL Paclitaxel, the Company remains focused on driving product and corporate development activities towards building and expanding its pipeline of oncology product candidates. For additional information, including news releases, please visit Sonus' web site at www.sonuspharma.com.

Safe Harbor

Certain statements made in this press release are forward-looking such as those, among others, relating to the development, safety and efficacy of drug delivery products and potential applications for these products. As discussed in Sonus Pharmaceuticals' filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K filed on March 12, 2004 and Quarterly Report on Form 10-Q filed November 15, 2004, actual results could differ materially from those projected in the forward-looking statements as a result of the following factors, among others: the Company's products will require extensive clinical testing and approval by regulatory authorities; such approvals are lengthy and expensive and may never occur; risks that clinical studies with TOCOSOL Paclitaxel will not be successful; risks that the FDA may not approve the Company's proposed New Drug Application; and risks of successful development of additional drug delivery products. Sonus undertakes no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof.

Taxol(R) is a registered trademark of Bristol-Myers Squibb Company.

Source: Business Wire

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