FDA Advisory Committee Recommends Use of Investigational Drug Maribavir (TAK-620) to Treat Post-Transplant Recipients with Cytomegalovirus (CMV) Infection and Disease Refractory to Treatment With or Without Resistance

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced the U.S. Food and Drug Administration (FDA) Antimicrobial Drugs Advisory Committee (AMDAC) voted unanimously to recommend use of maribavir (TAK-620) for the treatment of refractory cytomegalovirus (CMV) infection and disease with genotypic resistance to ganciclovir, valganciclovir, foscarnet or cidofovir in transplant recipients. The committee also voted unanimously to recommend use of maribavir for the treatment of refractory CMV infection and disease without genotypic resistance to ganciclovir, valganciclovir, foscarnet or cidofovir in transplant recipients. Both recommendations were based on the results of the Phase 2 and Phase 3 TAK-620-303 (SOLSTICE) trials.

“Today’s vote in favor of our investigational antiviral drug marks a significant step towards delivering the first approved treatment for adult transplant recipients with refractory CMV infection and disease, with or without resistance,” said Obi Umeh, MD, Vice President and Maribavir Global Program Leader at Takeda. “We look forward to working with the FDA as it completes its review of our application.”

AMDAC also heard from patients, advocates, and healthcare providers in the public forum discussion who underscored the need for new treatment options for this patient population.

The New Drug Application (NDA) for maribavir is currently under Priority Review by the FDA. The FDA will consider the vote as part of its review of the NDA and is not bound by the AMDAC’s recommendation. The NDA submission is based on the pivotal Phase 3 TAK-620-303 (SOLSTICE) trial.

“The treatment of CMV in patients who have undergone a solid organ or stem cell transplant is complicated, especially in patients who have failed standard treatment and who may be at risk for side effects from currently available medications,” said Dr. Emily Blumberg, Director, Transplant Infectious Diseases, Penn Medicine. "I am excited about the potential for an additional treatment option for post-transplant patients with CMV.”

About CMV

CMV is a beta herpesvirus that commonly infects humans; serologic evidence of prior infection can be found in 40%-100% of various adult populations.¹ CMV typically resides latent and asymptomatic in the body but may reactivate during periods of immunosuppression. Serious disease may occur in individuals with compromised immune systems, which includes patients who receive immunosuppressants associated with various types of transplants, including hematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT).^2,3 Out of the estimated 200,000 adult transplants per year globally, CMV is one of the most common viral infections experienced by transplant recipients, with an estimated incidence rate between 16-56% in SOT recipients and 30-70% in HSCT recipients.^3–10