ADMA Biologics, Inc., a late-stage biopharmaceutical company that develops, manufactures and intends to commercialise specialty plasma-based biologics for the primary immunodeficiency (PI) population and the treatment and prevention of certain infectious diseases, has submitted its Biologics License Application (BLA) to the United States Food and Drug Administration (FDA), seeking marketing authorisation for RI-002.
RI-002 demonstrated positive results in a phase III study in patients with PI, meeting its primary endpoint of no serious bacterial infections (SBI). These results, included in the submission, more than meet the requirement specified by the FDA guidance of = 1 SBI per patient-year.
"The BLA submission for RI-002 is a significant milestone for our growing company," stated Adam Grossman, president and chief executive officer of ADMA Biologics.
"This submission, combined with our recent announcement of obtaining patent protection for RI-002, places ADMA in a rare position as a company poised to transition from development to commercial stage. We believe that RI-002, if approved by the FDA, has great potential to provide meaningful clinical improvement for patients living with PI, as well as offers clinicians a much needed option for their immune compromised patients. Under PDUFA V, the BLA filing fee is $2.3 million. The FDA reviewed ADMA's small business waiver application and it was approved."
PI is a class of inherited genetic disorders that causes an individual to have a deficient or absent immune system due to either a lack of necessary antibodies or a failure of these antibodies to function properly, affecting approximately 250,000 people in the United States.
The FDA has a 60-day review period to determine whether ADMA's BLA submission for RI-002 is complete and acceptable for filing.
ADMA's lead product candidate, RI-002, is a specialty plasma-derived, polyclonal, intravenous immune globulin (IGIV) derived from human plasma containing naturally occurring polyclonal antibodies (e.g., Streptococcus pneumoniae, H. influenza type B, cytomegalovirus (CMV), measles, tetanus, etc.) as well as standardized, high levels of antibodies to respiratory syncytial virus (RSV).
On June 30, 2015, ADMA announced that it had received a notice of allowance for a US patent pertaining to RI-002 entitled "Compositions and Methods for the Treatment of Immunodeficiency." ADMA is pursuing an indication for the use of this specialty IGIV product for treatment of patients diagnosed with primary immune deficiency disease (PIDD). Polyclonal antibodies are the primary active component of IGIV/immuneglobulin products. Polyclonal antibodies are proteins that are used by the body's immune system to neutralise microbes, such as bacteria and viruses. Data review indicates that the polyclonal antibodies present in RI-002 support its ability to prevent infections in immune-compromised patients. ADMA's analysis demonstrated that the phase III trial met the primary endpoint with no serious bacterial infections (SBI) reported. These results more than meet the requirement specified by the FDA guidance of = 1 SBI per patient-year. A BLA was submitted to the FDA on July 31, 2015.