PharmAthene and Medarex Receive Orphan Drug Designation for Valortim(TM) to Treat Anthrax Infection
PR Newswire
Medarex, Inc. (Nasdaq: MEDX) and PharmAthene, Inc., a privately held biotechnology company specializing in the development of biodefense therapeutics, today announced that the United States Food and Drug Administration (FDA) has granted orphan drug designation to Valortim(TM)(MDX-1303) for the treatment of anthrax infection.
Valortim is an investigational fully human antibody created using Medarex's UltiMAb Human Antibody Development System(R) that targets the Bacillus anthracis protective
antigen.
The U.S. Orphan Drug Act is intended to encourage companies to develop safe and effective therapies for the treatment of rare diseases and conditions, specifically those expected to affect fewer than 200,000 people in the U.S. Orphan drug designation provides important benefits to companies such as eligibility for a special seven-year period of market exclusivity upon approval for the compound and indication with orphan designation, potential tax credits for research, potential grant funding for research and development, reduced filing fees for marketing applications, and assistance with clinical trial protocol review.
"Despite aggressive antibiotic therapy and supportive care, the anthrax attacks of 2001 resulted in a mortality rate of nearly 50%, illustrating the urgent need for more effective anthrax therapeutics," remarked David P. Wright, President and Chief Executive Officer of PharmAthene. "Our receipt of orphan drug designation for Valortim and, earlier this year, fast track designation, should facilitate a more streamlined development process through more frequent dialogue with the FDA."
Mr. Wright continued, "We believe Valortim may be the superior choice for procurement in the Strategic National Stockpile under Project BioShield. Its
mechanism of action appears to work under the same mechanism as the natural protective response to anthrax vaccine yet Valortim has the potential to provide immediate immunity compared to vaccines, which may take months to confer immunity. Animal studies have demonstrated efficacy in both pre- and post-exposure prophylaxis and treatment for anthrax infection, potentially enabling therapeutic intervention after the appearance of disease symptoms, when antibiotic therapy is progressively less effective."
Valortim is currently being evaluated in a Phase I open-label, dose-escalation clinical trial to evaluate the safety, tolerability, immunogenicity, and pharmacokinetics of a single dose of Valortim administered intravenously or intramuscularly in healthy volunteers. Results from this trial are anticipated later this year.
Preclinical studies suggest that Valortim has the potential to provide significant protection against anthrax infection when administered prophylactically, or prior to the emergence of symptoms of anthrax infection, and also may increase survival when administered therapeutically, or once symptoms become evident. In these studies, Valortim has been shown to protect both rabbits and monkeys against the lethal effects of anthrax infection when administered at the time of exposure at doses as low as 1 mg/kg. When administered to rabbits after the development of symptoms, Valortim also improved survival as late as 48 hours post-exposure as compared to controls.
Medarex, Inc. (Nasdaq: MEDX) and PharmAthene, Inc., a privately held biotechnology company specializing in the development of biodefense therapeutics, today announced that the United States Food and Drug Administration (FDA) has granted orphan drug designation to Valortim(TM)(MDX-1303) for the treatment of anthrax infection.
Valortim is an investigational fully human antibody created using Medarex's UltiMAb Human Antibody Development System(R) that targets the Bacillus anthracis protective
antigen.
The U.S. Orphan Drug Act is intended to encourage companies to develop safe and effective therapies for the treatment of rare diseases and conditions, specifically those expected to affect fewer than 200,000 people in the U.S. Orphan drug designation provides important benefits to companies such as eligibility for a special seven-year period of market exclusivity upon approval for the compound and indication with orphan designation, potential tax credits for research, potential grant funding for research and development, reduced filing fees for marketing applications, and assistance with clinical trial protocol review.
"Despite aggressive antibiotic therapy and supportive care, the anthrax attacks of 2001 resulted in a mortality rate of nearly 50%, illustrating the urgent need for more effective anthrax therapeutics," remarked David P. Wright, President and Chief Executive Officer of PharmAthene. "Our receipt of orphan drug designation for Valortim and, earlier this year, fast track designation, should facilitate a more streamlined development process through more frequent dialogue with the FDA."
Mr. Wright continued, "We believe Valortim may be the superior choice for procurement in the Strategic National Stockpile under Project BioShield. Its
mechanism of action appears to work under the same mechanism as the natural protective response to anthrax vaccine yet Valortim has the potential to provide immediate immunity compared to vaccines, which may take months to confer immunity. Animal studies have demonstrated efficacy in both pre- and post-exposure prophylaxis and treatment for anthrax infection, potentially enabling therapeutic intervention after the appearance of disease symptoms, when antibiotic therapy is progressively less effective."
Valortim is currently being evaluated in a Phase I open-label, dose-escalation clinical trial to evaluate the safety, tolerability, immunogenicity, and pharmacokinetics of a single dose of Valortim administered intravenously or intramuscularly in healthy volunteers. Results from this trial are anticipated later this year.
Preclinical studies suggest that Valortim has the potential to provide significant protection against anthrax infection when administered prophylactically, or prior to the emergence of symptoms of anthrax infection, and also may increase survival when administered therapeutically, or once symptoms become evident. In these studies, Valortim has been shown to protect both rabbits and monkeys against the lethal effects of anthrax infection when administered at the time of exposure at doses as low as 1 mg/kg. When administered to rabbits after the development of symptoms, Valortim also improved survival as late as 48 hours post-exposure as compared to controls.
