Four Independent Studies of Voraxaze® Published at European Hematology Conference 2022

Vienna – 9 June 2022 – The European Hematology Association (EHA) Conference published four independent studies of Voraxaze® (glucarpidase) by various investigators for inclusion in its June 2022 EHA Hybrid Congress abstract book and for publication in HemaSphere, EHA’s peer-reviewed, open access journal.

All four abstracts are the result of independent studies in Europe and the United States; three explore whether routinely giving Voraxaze® in combination with high-dose methotrexate (“planned use”) might alleviate toxicity, manage the risk to patients and help them to complete therapy.

• PLANNED-USE GLUCARPIDASE FOR OUTPATIENT HIGH DOSE METHOTREXATE (HD-MTX) ADMINISTRATION IN PATIENTS WITH CNS LYMPHOMA (CNSL) DURING THE COVID-19 PANDEMIC, by Lauren Schaff, Mina Lobbous, Dean Carlow, Lisa DeAngelis, Louis Burt Nabors, and Christian Grommes, demonstrates the feasibility of planned-use Voraxaze to facilitate the outpatient administration of HD-MTX for patients with CNS lymphoma. Twenty outpatient treatments of HD-MTX with glucarpidase were administered to a total of 7 patients. Some grade 1 and grade 2 adverse events attributable or possible attributable to Voraxaze were noted. None of the patients required hospital admission during treatment. [Link to abstract]

• REDUCTION OF METHOTREXATE (MTX) LEVELS AFTER GLUCARPIDASE IN PATIENTS WITH DIFFUSE LARGE B-CELL LYMPHOMA (DLBCL) AT RISK OF CENTRAL NERVOUS SYSTEM (CNS) INVOLVEMENT WHO RECEIVE HD-MTX: PHASE 2 by Adolfo De La Fuente, Francisco Javier Peñalver, Pilar Herrera, Raul Cordoba, Carmen Martinez Chamorro, Maria Teresa Olave, Isidro Jarque, and Mónica Estévez, is an open-label, interventional, non-randomized, phase 2 pilot multicenter study in diffuse large B-cell lymphoma patients at high risk of CNS relapse to assess prophylactic effect of glucarpidase in MTX related-toxicity administered after 12 hours of HD-MTX. The study aims to describe the reduction of MTX levels after systematic administration of glucarpidase 12 hours following start of HDMTX infusion in DLBCL patients at high risk for CNS involvement. This trial has not been completed. [Link to abstract]

• INTENDED USE OF GLUCARPIDASE IN PATIENTS WITH CENTRAL NERVOUS SYSTEM LYMPHOMA AND IMPAIRED RENAL FUNCTION – THE VALIDATE STUDY by Sven Liebig, Susen Burock, Immanuel Merz, Franz Knörnschild, Peggy Kießling, Kai Kappert, Ulrich Keller, and Stefan Schwartz evaluates the safety as well as pharmacological efficacy of intended and repeated use of glucarpidase following HD-MTX in patients with CNSL and impaired renal function. Glucarpidase administration resulted in a rapid and pronounced reduction of MTX blood levels (> 97%) within 15 minutes and in all 12 treatment cycles for four patients, demonstrating that treatment with HD-MTX and glucarpidase is feasible in patients with CNSL and impaired renal function. [Link to abstract]

• A POPULATION PHARMACOKINETIC MODEL OF HIGH-DOSE METHOTREXATE IN ADULTS WITH CANCER IDENTIFIES BASELINE ALBUMIN, BODY WEIGHT, AND TIME-DEPENDENT CREATININE AS PREDICTORS OF ACUTE KIDNEY INJURY by Scott Howard, Manuel Ibarra, Ryan Combs, and Paula Schaiquevich, describes a population pharmacometric model of HD-MTX elimination developed by the authors that considers weight, albumin, and time-dependent creatinine to predict AKI and guide supportive care in patients with delayed HD-MTX elimination. Patients with AKI who did not receive glucarpidase experienced significantly longer hospital length of stay (p<0.01) and time to methotrexate elimination (p<0.01). [Link to abstract]

“We are excited to see top researchers working to explore how routinely giving Voraxaze® in combination with methotrexate might improve cancer care for appropriate patients,” said Christon Hill, Group Chief Innovation Officer for SERB and BTG Specialty Pharmaceuticals. “These encouraging early findings suggests that such regimens may help to manage the risk to patients and ensure more patients are able to complete their cancer therapy.”

About Voraxaze® (glucarpidase)

Voraxaze® (glucarpidase) is currently approved in all members states of the European Union as well as Iceland, Liechtenstein, and Norway, where it is designated an orphan medicine.

Voraxaze® is approved by the European Commission to reduce toxic plasma methotrexate concentration in adults and children (aged 28 days and older) with delayed methotrexate elimination or at risk of methotrexate toxicity.

Detailed recommendations for the use of Voraxaze ® are described in the Summary of Product Characteristics (SmPC) and in the U.S. prescribing information (USPI).

SERB Specialty Pharmaceuticals market Voraxaze® in the European Union. BTG Specialty Pharmaceuticals markets Voraxaze® in the United States. SERB acquired BTG Specialty Pharmaceuticals in March 2021. Glucarpidase is sold in Japan as Megludase® by BTG’s marketing partner, Ohara Pharmaceutical Co.

About SERB and BTG Specialty Pharmaceuticals

Together, SERB and BTG Specialty Pharmaceuticals are a growing specialty pharmaceutical company and a dedicated ally to healthcare providers treating patients with critical conditions, focusing on emergency care and rare diseases. For over 30 years we have made treating these complex and life-threatening conditions possible, supporting clinicians, healthcare systems and governments while offering hope to patients and their families. As a fully integrated company, we have the experience and capabilities to acquire, develop, and manufacture our medicines to the highest standards, and make them available worldwide through our secure supply chain.

Learn more about SERB: https://serb.eu/
Learn more about BTG Specialty Pharmaceuticals: btgsp.com.

For further information contact:

Chris Sampson, Corporate Communications Director
chris.sampson@btgsp.com; Mobile: +44 (0)7773 251 178