FDA approves new drug to prevent a common virus infection in certain cell transplant patients based on research at Dana-Farber/Brigham and Women's Cancer Center

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Following FDA approval of letermovir last month, the New England Journal of Medicine published the main results from the pivotal Phase 3 clinical study of the new drug today. Letermovir is a new medicine for prevention of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients [R+] of allogeneic hematopoietic stem cell transplants (HSCT) and is manufactured by Merck & Co under the brand name PREVYMIS™. The study results were published online and will appear in a forthcoming print issue of the journal. The results were initially reported in February and represent a breakthrough in a decade-long effort to identify an effective drug for the prevention of cytomegalovirus (CMV) infection in transplant patients that doesn't produce side effects that negate the benefit of the drug itself, the study authors said. This multi-year major clinical trial was led by researchers at Dana-Farber/Brigham and Women's Cancer Center and has shown that a novel agent can protect against the most common viral infection that patients face after transplantation.

CMV-seropositive patients who undergo allogeneic hematopoietic cell transplantation are at high risk for CMV reactivation. CMV infection is a common clinically significant complication in these patients and early CMV reactivation after transplant is associated with increased mortality. Letermovir is a first-in-class antiviral drug that inhibits CMV replication by targeting the virus terminase complex.

"This study culminates more than a decade of efforts to identify new, highly effective antiviral medicines for patients that can be prescribed prophylactically after hematopoietic cell transplantation," said Francisco M. Marty, MD, attending physician in transplant and oncology infectious diseases at Dana-Farber/Brigham and Women's Cancer Center in Boston. "Patients receiving cell transplantation are already facing tremendous adversities. It is very rewarding to be able to help avoid one of the most common and potentially dangerous complications associated with these transplants."

In the study, significantly fewer patients in the letermovir arm (37.5%, n=122/325) compared to the placebo arm (60.6%, n=103/170) developed clinically significant CMV infection, discontinued treatment or had missing data through Week 24 post-transplant (p<0.001), the primary efficacy endpoint. The treatment effect for letermovir in preventing clinically significant CMV infection was consistent across pre-specified high- and low-risk strata for CMV reactivation both at Week 14 (end of treatment) and at Week 24 post-transplant. All-cause mortality in patients receiving PREVYMIS was lower compared to placebo at Week 24 post-transplant and at Week 48 post-transplant.

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