Date: 2014-10-15
Type of information: Granting of the orphan status in the EU
Product name: raxibacumab
Compound: raxibacumab
Therapeutic area: Infectious diseases
Action mechanism: Raxibacumab is a monoclonal antibody that neutralizes toxins produced by B. anthracis that can cause massive and irreversible tissue injury and death. Raxibacumab was developed by Human Genome Sciences (HGS) in conjunction with the Biomedical Advanced Research and Development Authority (BARDA) of the Office of the Assistant Secretary for Preparedness and Response (ASPR), U.S. Department of Health and Human Services (HHS) under contract number HHS010020050006C. Human Genome Sciences has since been acquired by GSK in July 2012. The development of raxibacumab is the result of a coordinated response to a recognised public health threat and the US government’s request for new medical countermeasures in the event of an anthrax attack against the civilian population.
Company: GSK (UK)
Disease: inhalational anthrax
Latest news:
- • On 2-4 September 2014, the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending raxibacumab for designation as an orphan medical product for the treatment of inhalation anthrax disease.
- • On December 14, 2012, the FDA approved raxibacumab injection to treat inhalational anthrax, a form of the infectious disease caused by breathing in the spores of the bacterium Bacillus anthracis. Raxibacumab also is approved to prevent inhalational anthrax when alternative therapies are not available or not appropriate. The FDA granted raxibacumab fast track designation, priority review, and orphan product designation. The drug demonstrated the potential to fill an unmet medical need, has the potential to provide safe and effective treatment where no satisfactory alternative therapy exists, and is intended to treat a rare disease, respectively.
Raxibacumab is the first monoclonal antibody approved under the FDA’s Animal Efficacy Rule, which allows efficacy findings from adequate and well-controlled animal studies to support FDA approval when it is not feasible or ethical to conduct trials in humans. In this case, because inhalational anthrax is a rare and lethal disease, it is not possible to conduct adequate efficacy trials in humans. Raxibacumab’s effectiveness for inhalational anthrax was demonstrated in one study in monkeys and three studies in rabbits. All animals were administered aerosolized B. anthracis spores, and efficacy was determined by survival at the end of the studies. Animals received varying doses of raxibacumab, placebo or antibiotics normally used to treat anthrax.
More animals treated with raxibacumab lived compared to animals treated with placebo. Sixty-four percent of animals in the monkey study and 44 percent of animals in one rabbit study receiving the 40 milligrams per kilogram dose of raxibacumab survived exposure to anthrax, compared with none in the placebo groups. All surviving animals developed toxin-neutralizing antibodies. Another study in rabbits showed that 82 percent of animals treated with antibiotics and raxibacumab survived exposure to anthrax compared with 65 percent of animals receiving antibiotic treatment alone.
The safety of raxibacumab was evaluated in 326 healthy human volunteers. Common side effects included rash, extremity pain, itching and drowsiness.
• On November 2, 2012, GSK has announced that the Anti-Infective Drugs Advisory Committee to the FDA voted 16 to 1 in support of the clinical benefit of raxibacumab for the treatment of inhalational anthrax, with one abstention. In addition, the committee voted 18 – 0 in favour of the risk-benefit profile of raxibacumab. The Committee provides non-binding recommendations for consideration by FDA, with the final decision on approval made by FDA. The Prescription Drug User Fee Act (PDUFA) goal date for raxibacumab is 15th December 2012.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization: 2012-12-14
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2003-11-12
Orphan status UE: 2014-10-15
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
