ADVENTRX APPLAUDS INCLUSION OF RARE DISEASES PROVISIONS IN THE FDA SAFETY AND INNOVATION ACT
· Creates new priority review voucher program for rare pediatric diseases
· Encourages flexible approaches to assessment of products under accelerated approval
SAN DIEGO, CALIFORNIA– July 11, 2012 – This week the Food and Drug Administration Safety and Innovation Act (Act) was signed into law. The Act amends the Federal Food, Drug, and Cosmetic Act in a variety of ways that encourage or facilitate the development of drugs for patients with rare diseases, including:
- Establishment of a Rare Pediatric Disease Priority Review Voucher Incentive Program. Entitles the sponsor of a “rare pediatric disease application” to a “priority review voucher” upon approval of the application. The voucher, which can be transferred (including by sale), entitles the holder to priority (6-month) review of a new drug application. Among other requirements, the rare pediatric disease application must target a rare pediatric disease, meaning a disease that primarily affects individuals from birth to 18 years and that is a rare disease or condition.
- Expansion of the Accelerated Approval framework. Encourages FDA to implement more broadly effective processes for expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions, including those for rare diseases or conditions, using a broad range of surrogate endpoints, which may result in fewer, smaller or shorter clinical trials. Enhances the statutory authority of FDA to consider appropriate scientific data, methods and tools, and to expedite development and access to novel treatments, for patients with a broad range of serious or life-threatening diseases or conditions. Specifically authorizes approval decisions to take into account the severity and rarity of the condition and the lack of alternative treatments.
- Increased Consultation between FDA and External Experts on Rare Diseases. For the purpose of promoting the efficiency of and informing the review by FDA of new drugs for rare diseases, (1) mandates that opportunities exist for consultation with stakeholders on a variety of issues related to rare diseases, including an assessment of the benefits and risks of therapies and the general design of clinical trials; and (2) requires development of a list of external experts who are qualified to provide advice on these issues.
“These changes are a tremendous benefit to patients with rare diseases and conditions, such as sickle cell disease, and should help expedite the development of new drugs, including by encouraging FDA to use surrogate endpoints earlier in the drug development cycle,” said Brian M. Culley, Chief Executive Officer of ADVENTRX.
About Accelerated Approval
FDA may approve a drug for a serious or life-threatening disease or condition upon a determination that the drug has an effect on a “surrogate endpoint” that is reasonably likely to predict clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments. This type of approval is referred to as “accelerated approval.”
A surrogate endpoint is a marker, such as a laboratory measurement or a physical sign, that is used in clinical trials as an indirect or substitute measurement that represents a clinically meaningful outcome, such as survival or symptom improvement. The use of a surrogate endpoint can shorten considerably the time required prior to receiving FDA approval. The evidence to support that an endpoint is reasonably likely to predict clinical benefit may include epidemiological, pathophysiological, therapeutic, pharmacologic, or other evidence developed using biomarkers, for example, or other scientific methods or tools. The studies that demonstrate the effect of the drug on the surrogate endpoint must be “adequate and well controlled” studies, the only basis under law for a finding that a drug is effective.
Accelerated approval of a drug may be subject to a requirement that the sponsor conduct appropriate post-approval studies to verify and describe the predicted clinical benefit. Accelerated approval of a drug may be withdrawn using expedited procedures if, among other reasons, the sponsor fails to conduct any required post-approval study of the drug with due diligence; a study required to verify and describe the predicted clinical benefit of the drug fails to verify and describe such benefit; or other evidence demonstrates that the drug is not safe or effective under the conditions of use.
About ADVENTRX Pharmaceuticals
ADVENTRX Pharmaceuticals is a biopharmaceutical company focused on developing proprietary product candidates. The Company’s lead product candidate is ANX-188, a rheologic, antithrombotic and cytoprotective agent that improves microvascular blood flow and has potential application in treating a wide range of diseases and conditions, such as complications arising from sickle cell disease. More information can be found on the Company’s web site at .
Forward Looking Statements
ADVENTRX cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements that are based on ADVENTRX's current expectations and assumptions. Such forward-looking statements include, but are not limited to, statements regarding whether the FDA Safety and Innovation Act will expedite the development of new drugs, including ANX-188. Among the factors that could cause or contribute to material differences between ADVENTRX’s actual results and those indicated from the forward-looking statements are risks and uncertainties inherent in ADVENTRX’s business, including, but are not limited to: the risk that FDA does not increase the use of surrogate endpoints or does not otherwise implement more broadly effective processes for the assessment of product candidates under the accelerated approval framework; the risk that sickle cell disease is determined not to be a rare pediatric disease; and other risks and uncertainties more fully described in ADVENTRX's news releases and periodic filings with the Securities and Exchange Commission. ADVENTRX's public filings with the Securities and Exchange Commission are available at www.sec.gov.
You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date when made. ADVENTRX does not intend to revise or update any forward-looking statement set forth in this press release to reflect events or circumstances arising after the date hereof, except as may be required by law.
Ioana C. Hone (firstname.lastname@example.org)
858-552-0866 Ext. 303