MedicalResearch.com Interview with:
Dr. Charles T. Nakar, MD
Indiana Hemophilia and Thrombosis Center Pediatrics
MedicalResearch.com: What is the background for this study?
Response: Congenital plasminogen deficiency is a rare genetic disorder that is caused by mutations in the PLG gene. Mutations in this gene lead to clinical manifestations such as fibrinous deposits on mucous membranes leading to disruption of tissue or organ function. These symptoms, when untreated, lead to significant morbidity and decreased quality of life. Life-threatening episodes may occur especially when the respiratory system is affected. There is currently no established approach to treatment of type 1 plasminogen deficiency and the available topical and systemic therapies (e.g. FFP, corticosteroids, immunomodulatory drugs, anticoagulants, amongst others) lack consistent efficacy. Patients may undergo multiple surgeries to remove lesions, but this approach typically leads to regrowth of lesions. Prometic’s intravenous plasminogen replacement therapy represents the first direct treatment for this serious disorder.
MedicalResearch.com: What are the main findings?
Response: In the largest therapeutic trial on plasminogen deficiency to date, Prometic’s intravenous plasminogen replacement therapy demonstrated excellent efficacy including complete resolution of visible lesions without recurrence over the 48-week study period. The treatment also increased plasminogen activity in all reported subjects. The treatment was easily administered at-home via intravenous injection and was well-tolerated over 48 weeks without safety concerns.
MedicalResearch.com: What should readers take away from your report?
Response: Prometic’s intravenous plasminogen replacement therapy represents a critical breakthrough in the treatment of congenital plasminogen deficiency. Due to the lack of effective existing treatments for this disorder, affected patients have suffered from symptoms. However, this may change in the near future, as a Biologics License Application (BLA) for Prometic’s intravenous plasminogen replacement therapy is currently being assessed by the U.S. Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA) date of April 14, 2018.
MedicalResearch.com: Is there anything else you would like to add?
Response: This rare disabling disorder has lacked a consistently effective treatment; it is our hope that both patients and clinicians will have access to this therapy that clearly demonstrated a positive impact on disease manifestations.
The FDA has recognized the seriousness and unmet need of available effective therapeutic solutions for patients with plasminogen deficiency and has thus granted orphan, fast-track and rare pediatric disease designations for Prometic’s intravenous plasminogen replacement therapy.
The Indiana Hemophilia and Thrombosis Center received funding from Prometic for conduct of the clinical research of this agent.
Dr. Charles Nakar is an investigator for the Phase II/III open-label clinical trial involving plasminogen (human) IV. His clinical practice is based in Indianapolis, IN.
Please note: Plasminogen (human) IV is under review by the FDA; not an approved therapy.
MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.
Pivotal Trial with Intravenous Plasminogen Replacement in Patients with Plasminogen Deficiency Demonstrates Long-Term Efficacy for Treatment and Prevention of Ligneous Lesions
Charles T Nakar, Joseph M Parker, Neelam Thakral, Brandon M. Hardesty, Gary Albert, Pierre Laurin, John Moran, Per Morten Sandset and Amy D. Shapiro
Blood 2017 130:84;
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