IVIG (Primary Immunodeficiency Diseases):
Intravenous immunoglobulin is a preparation of antibodies purified from plasma donations from healthy individuals. It is indicated for the maintenance treatment of patients with primary immunodeficiencies (PID), including common variable immunodeficiency, X-linked agammaglobulinemia and severe combined immunodeficiency. It is also indicated for the treatment of immune thrombocytopenic purpura (ITP) and for many other autoimmune diseases, including Guillain-Barré syndrome, Kawasaki disease.
According to recent market data, Canada is ranked as the second country in the world after the United States for the average consumption of IVIG (measured in kilograms per million people), with sales exceeding $600 million in 2016. The global IVIG market CAGR is expected to reach 5-6% between 2016 and 2025 with Canada having one of the lowest IVIG self-sufficiency ratios (i.e. less than 20%).
Primary immunodeficiencies are disorders in which part of the body’s immune system is missing or does not function normally. To be considered a primary immunodeficiency, the cause of the immune deficiency must not be secondary in nature (i.e., caused by other disease, drug treatment, or environmental exposure to toxins). Most primary immunodeficiencies are genetic disorders; the majority are diagnosed in children under the age of one, although milder forms may not be recognized until adulthood. While there are over 100 recognized PIDDs, most are very rare. About 1 in 500 people in the United States are born with a primary immunodeficiency. Immune deficiencies can result in persistent or recurring infections, autoinflammatory disorders, tumors, and disorders of various organs. There are currently no cures for these conditions; treatment is palliative and consists of managing infections and boosting the immune system
IVIG is the second biopharmaceutical arising from the plasma-derived therapeutics platform that is expected to be launched commercially, if approved. Currently being studied in a non-inferiority pivotal phase 3 open label, single arm, two-cohort multicenter clinical trial that is investigating the safety, tolerability, efficacy and pharmacokinetics of our plasma purified IVIG in a total of 75 patients suffering from PIDD, including 50 adults (cohort 1) and 25 children (cohort 2). The non-inferiority phase 3 clinical trial for IVIG in adults suffering from PIDD was completed in Q1 of 2018, meeting both the clinical primary and secondary endpoints, Prometic’s IVIG demonstrating comparable safety and efficacy data to existing commercial IVIG products without any significant drug related safety issues. The trial involving the pediatric cohort is expected to be completed in Q1 2019.
The primary end point is the rate of clinically documented serious bacterial infections (SBIs), defined as bacterial pneumonia, bacteremia and septicemia, osteomyelitis/septic arthritis, bacterial meningitis or visceral abscess. The FDA Guidance for Industry on studies required to support marketing of IGIV states: “…a statistical demonstration of a serious infection rate per person-year less than 1.0 is adequate to provide substantial evidence of efficacy”. Since there were no SBIs observed during the study, Prometic IGIV 10% met this requirement.
Secondary endpoints including episodes of fever (≥100.4°F), number of missed days, number of days of hospitalization due to infection, number of days on antibiotics, number of infections other than SBI, and trough IgG level were comparable between Prometic’s IGIV and commercial drugs. Only 4.94 days/subject/year were lost from work with Prometic IGIV 10%, which was significantly less than the rate observed while on commercial product.