FDA finalizes guidance on drugs for cytomegalovirus in transplant patients
The US Food and Drug Administration (FDA) on Thursday finalized guidance on developing drugs to treat or prevent cytomegalovirus, a common virus in the beta-herpes virus group, in patients who have undergone solid organ (SOT) or hematopoietic stem cell transplantation (HSCT).Cytomegalovirus, which is through to be present in 40-80% of the US population, is typically benign and self-limiting in patients with a normal immune system. However, the virus can cause complications and increased morbidity in patients with compromised immune systems, such as transplant recipients.
The 31-page final guidance lays out considerations for drugmakers looking to develop drugs to treat or prevent cytomegalovirus in transplant patients, from early-phase development through Phase 3 efficacy trials.
The guidance comes two years after FDA released the draft version for comment and includes clarification on the use of a surrogate endpoint, nonclinical combination studies and updated background information on preventing cytomegalovirus in transplant patients.
In the final guidance, FDA clarifies that it considers cytomegalovirus viremia (DNAemia) to be a validated surrogate endpoint for certain clinical trials as part of a composite endpoint to support traditional approval. As such, FDA notes that “accelerated approval regulations are generally not applicable for [cytomegalovirus] treatment and prevention indications.”
FDA also explains that nonclinical combination studies for drug combinations are generally not needed, referring sponsors to the International Council for Harmonisation’s ICH M3(R2) for more guidance. “Unless data from nonclinical studies of an early-stage entity suggest a potential for serious synergistic toxicity with an approved therapeutic drug, combination toxicology studies are not recommended,” FDA writes.
Additionally, the guidance includes more up-to-date information about preventing cytomegalovirus in transplant patients in order to better reflect the current literature on the subject.
FDA