The U.S. Food and Drug Administration (FDA) approved ADMA Biologics’ Asceniv to treat Primary Humoral Immunodeficiency Disease (PIDD or PI) in adults and adolescents.
The U.S. Food and Drug Administration (FDA) approved ADMA Biologics’ Asceniv to treat primary humoral immunodeficiency disease (PIDD or PI) in adults and adolescents.
Asceniv, Immune Globulin Intravenous, Human, is a polyclonal, intravenous immune globulin, essentially a shot of naturally occurring antibodies. PIDD is a class of inherited genetic diseases that result in a deficient or absent immune system. Asceniv, basically, replaces the missing immune cells, helping to fend off infections.
The approval was based on a pivotal Phase III trial that enrolled 59 patients with PIDD at nine sites in the U.S. Over the course of a full year, patients received regular infusions of Asceniv. The primary endpoint was the rate of Serious Bacterial Infections (SBI) in patients receiving Asceniv. Secondary endpoints included time to first SBI and to first serious infection, days on antibiotics, days off school or work due to infections, all confirmed infections of any kind, and hospitalizations due to infections.
There were no SBIs during the 12-month trial period. The results have been published in the Journal of Clinical Immunology (2016;36:590-599) by Richard Wasserman, et al.
The FDA slapped a boxed warning on the therapy about potential thrombosis and renal dysfunction or failure, in addition to the most common side effects observed in the Phase III trial, headache, sinusitis, diarrhea, gastroenteritis viral, nasopharyngitis, upper respiratory tract infection, bronchitis, and nausea.
“We are excited about this significant achievement in receiving FDA approval for Asceniv, a novel, patented IVIG product that we feel is a necessary addition to existing available therapies for patients who suffer from PI,” stated Adam Grossman, president and chief executive officer of ADMA Biologics. “We hope availability of Asceniv will help ameliorate a portion of the current shortages facing U.S. IVIG supply.”
There are about 250,000 PI patients diagnosed and living in the U.S.
Grossman went on to say, “As previously disclosed, Asceniv is manufactured using our unique, patented plasma donor screening methodology and tailored plasma pooling design, which blends normal source plasma and plasma from donors tested using our proprietary microneutralization assay. Going forward, we believe this FDA approval better positions ADMA to further its mission to evaluate Asceniv in immune-compromised patients infected with or at-risk for Respiratory Syncytial Virus infection. We look forward to working with the FDA and the immunology and infectious disease community on developing a clinical investigation to evaluate use of Asceniv in this patient population in the near future.”
ADMA Biologics indicates it expects to have Asceniv on the market in the second half of this year.
Grossman also said, “With the receipt of Asceniv’s FDA approval, ADMA, at its sole option, can elect to access up to an additional $27.5 million of available funding from perceptive Advisors under ADMA’s existing credit facility. This option remains available to the company through June 2020, and such funds could be used to support the launch of Asceniv, procure plasma raw material inventory, and begin construction on potential new plasma centers, as well as for general corporate activities.”
