ADVENTRX Applauds Inclusion of Rare Diseases Provisions in the FDA Safety and Innovation Act

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

PR Newswire

SAN DIEGO, July 11, 2012

SAN DIEGO, July 11, 2012 /PRNewswire/ -- 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: 

  o 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. 
  o 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. 
  o 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
www.adventrx.com.

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.

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SOURCE ADVENTRX Pharmaceuticals

Website: http://www.adventrx.com
Contact: ADVENTRX Pharmaceuticals, Ioana C. Hone, ir@adventrx.com,
+1-858-552-0866, ext. 303