JAPAN – Takeda has received the first FDA approval for a treatment for refractory cytomegalovirus (CMV) infections in organ transplant patients, which can lead to complications and even death.
Livtencity (maribavir) has been approved for use in transplant patients aged 12 and up who have CMV infections that do not respond to first-line CMV antiviral treatment with ganciclovir, valganciclovir, foscarnet, or cidofovir.
The FDA followed the advice of its advisory committee and approved Livtencity for use regardless of whether patients are infected with CMV strains with resistance mutations, as testing for these could cause treatment to be delayed.
CMV is one of the most common and serious post-transplant infections, with an estimated incidence rate ranging from 16% to 56% in solid organ transplants and 30% to 70% in hematopoietic stem-cell transplantation (HSCT) procedures.
An orally bioavailable compound, Livtencity hiders the pUL97 enzyme, a protein kinase and its natural substrates to prevent replication of the virus.
It also avoids some of the serious hematological side effects associated with older antivirals. When Takeda merged with Shire in 2019, it acquired one of the drugs.
The approval is the culmination of a lengthy development process for a drug that failed a phase 3 trial for CMV prevention in HSCT recipients more than a decade ago.
It was originally developed by GlaxoSmithKline and licensed to ViroPharma, which was later purchased by Shire.
The approval is based on the results of the phase 3 SOLSTICE trial, which demonstrated that Livtencity outperformed conventional antiviral therapies in refractory CMV.
Takeda’s drug resulted in confirmed viral clearance in more than 55 percent of transplant recipients with refractory infections, compared to around 24 percent of those on older drugs.
In other latest developments, the FDA recently approved BioMarin Pharmaceutical Inc’s once-daily injection, Voxzogo, for children with the most common type of dwarfism making it the country’s first approved therapy for achondroplasia.
BioMarin Pharmaceutical developed the treatment for children with achondroplasia, a rare genetic disorder that causes dwarfism and can lead to serious medical complications.
Patients who received the drug grew 1.6 centimeters more over the course of a year than those who received a placebo in a pivotal clinical trial.
According to BioMarin, patients who take Voxzogo throughout childhood are more likely to reach heights comparable to their peers who do not have achondroplasia.
The approval of Voxzogo, which is intended for children aged five and up, comes months after it became the first therapy approved by European regulators for the treatment of the genetic condition.