BTG Specialty Pharmaceuticals announces the European Medicines Agency has accepted for review a marketing authorisation application for Voraxaze®

Rhea-AI Summary

BTG Specialty Pharmaceuticals, a division of Boston Scientific (NYSE:BSX), announced that the European Medicines Agency (EMA) has accepted its Marketing Authorisation Application (MAA) for Voraxaze® (glucarpidase). This application aims to authorize Voraxaze® for treating adults and children at risk of methotrexate toxicity due to delayed elimination. The MAA is underpinned by clinical data and real-world experience, having previously treated nearly 3,000 patients in the US since FDA approval in 2012. A decision from the EMA could lead to marketing authorization across the EU and additional countries.

Positive

• EMA acceptance of Voraxaze® MAA could expand market reach in Europe.
• Voraxaze® has a proven track record, treating approximately 2,867 patients in the US since 2012.
• The application is based on substantial clinical data showing a median reduction of >98% in methotrexate concentration within 15 minutes.

Negative

• Voraxaze® has only been available in Europe on a named patient basis for eight years, indicating limited previous access.
• Approval is not guaranteed and is contingent on the EMA's review process.

LONDON, Aug. 17, 2020 /PRNewswire/ -- BTG Specialty Pharmaceuticals, a division of Boston Scientific (NYSE:BSX), today announced the European Medicines Agency (EMA) has accepted for review a Marketing Authorisation Application (MAA) for Voraxaze^® (glucarpidase). BTG Specialty Pharmaceuticals is seeking marketing authorization of Voraxaze^® for the treatment of adults and children (from 28 days of age) at risk of methotrexate toxicity due to delayed methotrexate elimination.

The MAA for Voraxaze^® is based on clinical data as well as real world experience in the US, where glucarpidase was approved by the FDA in 2012. Since its launch in the US, an estimated 2,867 patients have been treated with Voraxaze^®. The efficacy of Voraxaze^® has been evaluated in four open-label multi-center studies in patients with delayed methotrexate elimination due to renal dysfunction. In 169 patients with methotrexate concentrations measured by chromatographic methods, a median reduction of > 98% in methotrexate concentration occurred within 15 minutes following Voraxaze^® administration.

The EMA will review the application under the centralized marketing authorization procedure. If approved Voraxaze^® would receive marketing authorization in all member states of the European Union (EU), as well as in Iceland, Liechtenstein and Norway.

"While Voraxaze^® has been available in Europe on a named patient basis for eight years, we're eager to do more to educate physicians about the potential for Voraxaze^® to help cancer patients around the world suffering the toxic effects of methotrexate," said Anthony Higham, President of BTG Specialty Pharmaceuticals. "This filing is a first step toward a marketing authorisation that would allow us to speak with European clinicians about treating this condition."

Voraxaze^® is also being independently studied to explore whether routinely giving Voraxaze^® in combination with high-dose methotrexate might alleviate toxicity, manage the risk to patients and help them to complete therapy. Enrollment in these studies is ongoing. For more information about these studies, or to contact an investigator about participation, please visit https://clinicaltrials.gov/ct2/show/NCT03684980 or https://clinicaltrials.gov/ct2/show/NCT03960177.

About BTG Specialty Pharmaceuticals
BTG Specialty Pharmaceuticals, a division of Boston Scientific, provides rescue medicines that counteract the potentially life-threatening effects associated with exposure or overexposure to certain toxins. These acute care products are typically used in emergency rooms and intensive care units to treat patients for whom there are limited or no existing treatment options. We are dedicated to delivering quality medicines that make a real difference to patients and their families through the development, manufacture, and commercialization of pharmaceutical products. To learn more about BTG Pharmaceuticals, please visit: btgsp.com.

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

Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934.  Forward-looking statements may be identified by words like "anticipate," "expect," "project," "believe," "plan," "estimate," "intend" and similar words.  These forward-looking statements are based on our beliefs, assumptions and estimates using information available to us at the time and are not intended to be guarantees of future events or performance.  These forward-looking statements include, among other things, statements regarding our business plans and product performance and impact.  If our underlying assumptions turn out to be incorrect, or if certain risks or uncertainties materialize, actual results could vary materially from the expectations and projections expressed or implied by our forward-looking statements.  These factors, in some cases, have affected and in the future (together with other factors) could affect our ability to implement our business strategy and may cause actual results to differ materially from those contemplated by the statements expressed in this press release.  As a result, readers are cautioned not to place undue reliance on any of our forward-looking statements. 

Factors that may cause such differences include, among other things: future economic, competitive, reimbursement and regulatory conditions; new product introductions; demographic trends; intellectual property; litigation; financial market conditions; and future business decisions made by us and our competitors.  All of these factors are difficult or impossible to predict accurately and many of them are beyond our control.  For a further list and description of these and other important risks and uncertainties that may affect our future operations, see Part I, Item 1A – Risk Factors in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, which we may update in Part II, Item 1A – Risk Factors in Quarterly Reports on Form 10-Q we have filed or will file hereafter.  We disclaim any intention or obligation to publicly update or revise any forward-looking statements to reflect any change in our expectations or in events, conditions or circumstances on which those expectations may be based, or that may affect the likelihood that actual results will differ from those contained in the forward-looking statements.  This cautionary statement is applicable to all forward-looking statements contained in this document.

US INDICATION AND LIMITATIONS OF USE

• Voraxaze^® is a carboxypeptidase indicated to reduce toxic plasma methotrexate concentration (greater than 1 micromole per liter) in adult and pediatric patients with delayed methotrexate clearance (plasma methotrexate concentrations greater than 2 standard deviations of the mean methotrexate excretion curve specific for the dose of methotrexate administered) due to impaired renal function
• Limitations of Use: Voraxaze^® is not recommended for use in patients who exhibit the expected clearance and expected plasma methotrexate concentration. Reducing plasma methotrexate concentration in these patients may result in subtherapeutic exposure to methotrexate

IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS

Serious Hypersensitivity Reactions

• Serious hypersensitivity reactions, including anaphylactic reactions, may occur. Serious hypersensitivity reactions occurred in less than 1% of patients

Monitoring Methotrexate Concentration/Interference with Assay

• Methotrexate concentrations within 48 hours following Voraxaze® administration can only be reliably measured by a chromatographic method due to interference from metabolites. Measurement of methotrexate concentrations within 48 hours of Voraxaze® administration using immunoassays results in an overestimation of the methotrexate concentration

ADVERSE REACTIONS

• In clinical trials, the most common related adverse events (occurring in >1% of patients) were paresthesia, flushing, nausea and/or vomiting, hypotension and headache

DRUG INTERACTIONS

• Voraxaze^® can decrease leucovorin concentration, which may decrease the effect of leucovorin rescue unless leucovorin is dosed as recommended, and may also reduce the concentrations other folate analogs or folate analog metabolic inhibitors

Please see full Prescribing Information.

 

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SOURCE BTG Specialty Pharmaceuticals

FAQ

What is the significance of the EMA accepting the MAA for Voraxaze® (BSX)?

The EMA's acceptance of the MAA for Voraxaze® is crucial as it may lead to wider availability and marketing authorization across the EU, enhancing patient access to this important treatment.

How many patients have been treated with Voraxaze® since its US approval?

Since its FDA approval in 2012, approximately 2,867 patients have been treated with Voraxaze® in the US.

What are the expected benefits of Voraxaze® for patients?

Voraxaze® aims to significantly reduce methotrexate toxicity, as shown by clinical data indicating a median reduction of over 98% in methotrexate concentration shortly after administration.

What is the next step after the EMA's acceptance of the MAA for BSX?

The EMA will conduct a review of the MAA, and if approved, Voraxaze® will be available for marketing in all EU member states, as well as in Iceland, Liechtenstein, and Norway.

What is the primary use of Voraxaze® as indicated in the press release?

Voraxaze® is primarily indicated to reduce toxic plasma methotrexate concentrations in adults and children experiencing delayed methotrexate clearance due to renal dysfunction.