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BTG Announces FDA Approval of VISTOGARD® (Uridine Triacetate) as Antidote to Overdose and Early Onset, Severe, or Life-Threatening Toxicities from Chemotherapy Drugs 5-Fluorouracil (5-FU) or Capecitabine

VISTOGARD is First and Only Treatment Available in US to Reverse Effects of and Prevent Death from Severe 5-FU Toxicity

London, UK, December 11, 2015: BTG plc (LSE: BTG) announced today that the United States Food and Drug Administration (FDA) has approved Wellstat Therapeutics’ VISTOGARD® (uridine triacetate) as the first and only drug to treat patients following an overdose of chemotherapy drugs 5-fluorouracil (5-FU) or capecitabine, or in patients exhibiting early-onset, severe or life-threatening toxicity affecting the cardiac or central nervous system, and/or early-onset, unusually severe adverse reactions (e.g., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of 5-FU or capecitabine administration. VISTOGARD received approval following a priority review by the FDA.

Potentially life-threating or lethal toxicity from 5-FU can occur if the drug has been administered at a dose or rate greater than intended, or when a patient has genetic variations, impaired clearance or other factors that increase susceptibility to the toxicities of the drug. In combination with other drugs or radiation, 5-FU is a mainstay of chemotherapy across a variety of solid tumors, including those of the colon, pancreas, stomach, esophagus, breast, and head and neck. Capecitabine is an orally administered chemotherapy prodrug of 5-FU that is transformed into 5-FU within the body.

Wellstat Therapeutics developed VISTOGARD and, following today’s approval of VISTOGARD, BTG will market, sell and distribute the drug for this indication in the US.

Principal investigator of the VISTOGARD clinical development program, Wen Wee Ma, MD, Associate Professor of Oncology, GI Cancers & Drug Development Program, Roswell Park Cancer Institute, commented: “Severe 5-FU toxicity has, historically, been difficult to treat and sometimes resulted in death for those affected. It is important to recognize the signs of severe 5-FU and capecitabine toxicity early, which often include unexpected side effects on the first cycle – including gastrointestinal toxicities such as mucositis, central nervous system toxicities such as altered mental state, hematologic toxicities, and even cardiotoxicity. The approval of VISTOGARD is important because it represents the first treatment with a demonstrated track record of efficacy and, just as important, it allows some patients to resume chemotherapy sooner following the resolution of the toxicity. VISTOGARD also has a very good safety profile.”

BTG’s Chief Executive Officer, Louise Makin, commented: “Severe 5-FU toxicity is an ongoing concern for the oncology community and, in some patients, it may go unrecognized. VISTOGARD not only saves lives but also helps some patients get back to fighting their cancer with chemotherapy. We’re proud to add VISTOGARD to our portfolio of innovative products and potentially life-saving antidotes.”

VISTOGARD Clinical Development Program

The FDA approval of VISTOGARD is based on data from a development program in 135 patients designed to demonstrate the efficacy and safety of a single course of 10 grams given orally every six hours for a total of 20 doses (patients in the studies had either received an overdose of 5-FU or capecitabine, or presented with severe or life-threatening toxicities within 96 hours following the end of 5-FU or capecitabine administration). In clinical studies, overall survival of patients with 5-FU toxicity receiving VISTOGARD was 96 percent, compared with 16 percent in historical cases employing standard supportive care measures. VISTOGARD also helped patients resume chemotherapy sooner, with 33 percent resuming their cancer treatment within 30 days.

Adverse events that occurred in greater than 2 percent of patients were vomiting (10%), nausea (5%), and diarrhea (3%).

Following today’s approval, VISTOGARD will be made available to the US market as soon as possible. In the interim, physicians treating patients in need of VISTOGARD treatment are encouraged to follow the current emergency access protocol and contact Wellstat Therapeutics.

Selected Important Safety Information for VISTOGARD (Uridine Triacetate) oral granules

INDICATION
VISTOGARD is indicated for the emergency treatment of adult and pediatric patients:
  • following a fluorouracil or capecitabine overdose regardless of the presence of symptoms, or
  • who exhibit early-onset, severe or life-threatening toxicity affecting the cardiac or central nervous system, and/or early-onset, unusually severe adverse reactions (e.g., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of fluorouracil or capecitabine administration.
Limitations of use:
  • VISTOGARD is not recommended for the non-emergent treatment of adverse reactions associated with fluorouracil or capecitabine because it may diminish the efficacy of these drugs.
  • The safety and efficacy of VISTOGARD initiated more than 96 hours following the end of fluorouracil or capecitabine administration have not been established.
IMPORTANT SAFETY INFORMATION
  • In clinical studies, adverse reactions occurring in > 2% patients receiving VISTOGARD were vomiting (10%), nausea (5%) and diarrhea (3%).
  • One patient receiving uridine triacetate experienced grade 3 nausea and vomiting.
Please see full Prescribing Information.

About VISTOGARD (uridine triacetate) oral granules

VISTOGARD (uridine triacetate) is an orally administered drug approved by the FDA to treat patients following an overdose of 5-fluorouracil (5-FU) or capecitabine or in patients exhibiting early-onset, severe or life-threatening toxicity affecting the cardiac or central nervous system, and/or early-onset, unusually severe adverse reactions (e.g., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of 5-FU or capecitabine administration. VISTOGARD received orphan drug designation from the FDA as an antidote in the treatment of 5-FU poisoning and from the European Medicines Agency (EMA) as a treatment for 5-FU overdose. In Europe, under a named patient program, VISTOGARD is currently provided to patients at risk of excess 5-FU toxicity due to overdose and patients exhibiting severe toxicities to 5-FU within 96 hours of 5-FU administration.

For more information please visit www.vistogard.com.

About 5-Fluorouracil (5-FU)

5-FU is on the World Health Organization’s List of Essential Medicines, a compilation of the most important medications needed in a basic health system. Because 5-FU is administered in different doses and schedules as a frequent component of standard chemotherapy regimens for a variety of cancers, patients can experience dramatically different patterns of toxicity.

Used in combination with other chemotherapy agents and/or radiation, 5-FU has been for decades a mainstay of various treatment regimens for solid tumors, including those of the colon, pancreas, stomach, esophagus, breast, and head and neck. The drug is most commonly administered by infusion pump at or near what is considered the maximum tolerated dose. Expected side effects of 5-FU include myelosuppression (a reduction in white-blood-cell counts and thus increased risk of infection), diarrhea, nausea, vomiting, and mucositis (a painful inflammation and ulceration of the mucous membranes lining the digestive tract). Overexposure to 5-FU can lead to severe myelosuppression, gastrointestinal hemorrhage, septic shock, multiple organ failure, cardiac and neurological complications, and death.

About Capecitabine

Capecitabine is an orally administered chemotherapy prodrug 5-FU that is enzymatically activated within the body and transformed into 5-FU. When capecitabine comes into contact with a naturally occurring protein called thymidine phosphorylase, capecitabine is transformed into 5-FU. Because many cancers have higher levels of thymidine phosphorylase than do normal tissues, more 5-FU is delivered to the tumor than to other tissue.

About Wellstat Therapeutics

Wellstat Therapeutics Corporation is a privately-held biopharmaceutical company located in Gaithersburg, Maryland. Wellstat Therapeutics is committed to discovering, developing and commercializing products that will provide new and improved treatments for patients in the fields of oncology and metabolic, neurometabolic and neurodegenerative diseases. For more information, please visit the website at http://www.wellstattherapeutics.com. Wellstat Therapeutics is part of the Wellstat group of companies (http://www.wellstat.com).

About BTG

BTG is a growing international specialist healthcare company bringing to market innovative products in specialist areas of medicine to better serve doctors and their patients. We have a portfolio of Interventional Medicine products to advance the treatment of liver tumors, advanced emphysema, severe blood clots and varicose veins, and Specialty Pharmaceuticals that help patients overexposed to certain medications or toxins. Inspired by patient and physician needs, BTG is investing to expand its portfolio to address some of today’s most complex healthcare challenges. To learn more about BTG, please visit: www.btgplc.com.

For further information contact:

BTG

Andy Burrows, VP Corporate & Investor Relations
+44 (0)20 7575 1741; Mobile: +44 (0)7990 530 605
Stuart Hunt, Investor Relations Manager
+44 (0)20 7575 1582; Mobile: +44 (0)7815 778 536
Chris Sampson, Corporate Communications Director +44 20 7575 1595; Mobile: +44 7773 251 178