""Orphan Product Designation
The Orphan Drug Act (ODA) provides for granting special status to a product to treat a rare disease or condition upon request of a sponsor. The combination of the product to treat the rare disease or condition must meet certain criteria. This status is referred to as orphan designation. Orphan designation qualifies the sponsor of the product for the tax credit and marketing incentives of the ODA. A marketing application for a prescription drug product that has been designated as a drug for a rare disease or condition is not subject to a prescription drug user fee unless the application includes an indication for other than a rare disease or condition.""
This milestone from the firm was a herculean task achieved by the brilliant team lead by Dr. Peters and Dr. Norris.
The news is so fresh firm this morning, that when I called their offices to congratulate the team, the receptionist was unaware that the FDA orphan drug status had been granted, nor did she understand the significance.
For a refresher of some things Adherex, google > "wendman adherex genesis" and read the post describing early days of the firm after renewal of management by the then fresh merger / takeover by the Oxiquant team.
This recent FDA orphan status approval is likely the mere beginning of realization of the promise of the firm's technology, a significant milestone in the quest by Prof. Orest Blaschuk of McGill and others to apply Cadherin technology to cancer treatments. It has been a long and arduous journey to this amazing first substantive FDA milestone by Adherex.
|Press Release||Source: Adherex Technologies Inc.|
Adherex Receives Orphan Drug Designation for ADH-1 in Melanoma Additional centers to be added to ongoing Phase I/IIb trial
Friday February 22, 8:30 am ET
Additional centers to be added to ongoing Phase I/IIb trial
"FDA orphan drug designation for ADH-1 is an important asset in Adherex's development of this drug," said Dr. William P. Peters, CEO and Chairman of Adherex. "Orphan drug designation provides multiple incentives for Adherex to continue its accelerated development of ADH-1 for this significant clinical problem. Melanoma is a disease with an extremely poor prognosis and one in which the molecular target for ADH-1, N-cadherin, is frequently and often intensively expressed. N-cadherin is also intimately involved in the invasion and metastasis of melanoma. Our experience to date combining ADH-1 and melphalan for the treatment of in-transit melanoma has been very encouraging. To continue with the rapid development of this combination, two additional centers, Lehigh Valley and H. Lee Moffitt, have joined our Phase IIb trial which is ongoing at Duke and the MD Anderson. All four participating institutions are first-class medical centers with experienced investigators, and we are very pleased to have them involved in this development program."
The FDA orphan drug designation, administered by the Office of Orphan Products Development, provides potential incentives such as funding for clinical studies, study design assistance, waiver of FDA user fees, tax credits and, importantly, up to seven years of market exclusivity upon marketing approval.
Adherex is currently evaluating the synergy of systemic ADH-1 in combination with regionally-infused melphalan for the treatment of melanoma in a Phase I/IIb trial. The Lehigh Valley Hospital in Pennsylvania and the H. Lee Moffitt Cancer Center in Florida are being added as additional clinical trial sites to provide further multi-institutional experience. The Phase IIb portion of this trial is anticipated to complete patient accrual by approximately mid-2008, with data to be released at an appropriate scientific venue.
Another Phase I trial is also nearing completion at US Oncology in which systemic ADH-1 is being evaluated in combination with three different systemic chemotherapies: ADH-1 + docetaxel (Taxotere®), ADH-1 + carboplatin, and ADH-1 + capecitabine (Xeloda®). Subsequent Phase II and potential randomized, prospective trials of ADH-1 in combination with chemotherapy will be planned and based upon the results of the combination studies currently ongoing.
About Adherex Technologies
Adherex Technologies Inc. is a biopharmaceutical company dedicated to the discovery and development of novel cancer therapeutics. We aim to be a leader in developing innovative treatments that address important unmet medical needs in cancer. We currently have multiple products in the clinical stage of development, including eniluracil, ADH-1 and sodium thiosulfate (STS). Eniluracil, an oral dihydropyrimidine dehydrogenase (DPD) inhibitor, was previously under development by GlaxoSmithKline for oncology indications. ADH-1, our lead biotechnology compound, selectively targets N-cadherin, a protein present on certain tumor cells and established blood vessels that feed solid tumors. STS, a drug from our specialty pharmaceuticals pipeline, protects against the disabling hearing loss that can often result from treatment with platinum-based chemotherapy drugs. With a diversified portfolio of unique preclinical and clinical-stage cancer compounds and a management team with expertise in identifying, developing and commercializing novel cancer therapeutics, Adherex is emerging as a pioneering oncology company. For more information, please visit our website at www.adherex.com.
This press release contains forward-looking statements that involve significant risks and uncertainties. The actual results, performance or achievements of the Company might differ materially from the results, performance or achievements of the Company expressed or implied by such forward-looking statements. Such forward-looking statements include, without limitation, those regarding the development plans of the Company and the expected results of our development. We can provide no assurance that such development will proceed as currently anticipated or that the expected results of such development will be realized. We are subject to various risks, including the uncertainties of clinical trials, drug development and regulatory review, the early stage of our product candidates, our reliance on collaborative partners, our need for additional capital to fund our operations, our history of losses, and other risks inherent to the biopharmaceutical industry. For a more detailed discussion of related risk factors, please refer to our public filings available at www.sedar.com and www.sec.gov.
Adherex Technologies Inc.
D. Scott Murray
Senior Vice President, Corporate Development
Source: Adherex Technologies Inc.
here are links to prior Adherex posts of mine