ADMA Biologics' Novel IVIG ASCENIV Receives FDA Approval for Primary Immunodeficiency Treatment

VCBeat (WeChat ID: vcbeat) has learned that ASCENIV, a novel intravenous immunoglobulin produced by the biopharmaceutical company ADMA Biologics, has recently received FDA approval for the treatment of primary humoral immunodeficiency (PI) in adults and adolescents aged 12 to 17 years.

ADMA Biologics is a biopharmaceutical company headquartered in New Jersey, USA. Since its establishment in 2004, the company has focused on the research and development, manufacturing, and sales of plasma-derived biologics for the treatment of immune deficiency diseases and the prevention of specific infectious diseases. In addition, ADMA Biologics operates an FDA-licensed plasma collection division, which supplies a portion of the plasma required for the production of its flagship products, RI-002 and RS-002. To date, the company has raised $20 million to support the development of immunoglobulin preparations.

Primary immunodeficiency (PI) is an immune deficiency caused by more than 350 different genetic factors. It predominantly affects infants and young children, who, due to their weaker immune defenses, are prone to recurrent infections that can be life-threatening in severe cases. Based on the nature of the immune defect, PI can be classified into three major categories: humoral immune deficiency, cellular immune deficiency, and combined immune deficiency involving both. Among these, humoral immune (B-cell) deficiency is clinically characterized by reduced or absent immunoglobulin levels.

PI includes, but is not limited to, humoral immune deficiencies associated with primary agammaglobulinemia, common variable immunodeficiency (CVID), X-linked agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiency (SCID). Data indicate that 6 million children and adults worldwide are affected by PI, with 250,000 diagnosed cases in the United States alone; however, intravenous immunoglobulin (IVIG), an effective treatment for this condition, has remained in short supply.

It is understood that 53% of patients with primary immunodeficiency (PI) experience decreased serum immunoglobulin levels due to defects in the body’s antibody production mechanism. To prevent infections, PI patients typically require monthly injections of polyclonal antibody immunoglobulins—proteins within the human immune system that neutralize microorganisms and help prevent infection. ASCENIV (formerly known as RS-002), an intravenous immunoglobulin (IVIG) product manufactured by ADMA Biologics, contains naturally occurring polyclonal antibodies and neutralizing antibodies against respiratory syncytial virus (RSV), significantly boosting patients’ immunoglobulin levels.

It is reported that ADMA Biologics recently completed a pivotal Phase III clinical study. Researchers enrolled 59 patients with primary immunodeficiency (PI) at nine clinical centers in the United States and administered ASCENIV infusions for one year to evaluate whether ASCENIV could reduce the rate of serious bacterial infections (SBI). The final results showed that the SBI rate was zero in all patients. These findings have been published in the Journal of Clinical Immunology. However, researchers also observed adverse events in 5% of patients, including headache, sinusitis, diarrhea, viral gastroenteritis, nasopharyngitis, upper respiratory tract infection, bronchitis, and nausea.

Notably, the prescribing information for ASCENIV includes a boxed warning indicating that the drug may cause adverse effects such as thrombosis, renal impairment, and renal failure; therefore, it should be used under the supervision of a physician. ADMA Biologics anticipates that ASCENIV will be launched in the United States in the second half of 2019.

Adam Grossman, President and Chief Executive Officer of ADMA Biologics, stated, “ASCENIV is a novel IVIG product that provides PI patients with better treatment options. Furthermore, once successfully launched, ASCENIV will also alleviate the shortage of PI medications in the United States.”

(Compiled by: Feng Yutong)