The upcoming week is set to be a quieter one for the FDA, with only two key events scheduled. However, the agency has recently experienced a flurry of verdicts and meetings, making the upcoming week a much-needed break.
Roche Pushes Polivy into the Frontline
The Oncologic Drugs Advisory Committee of the FDA is set to meet on March 9th to discuss Genentech’s supplemental Biologics License Application for Polivy (polatuzumab vedotin-piiq). This revolutionary anti-CD79b antibody-drug conjugate has the potential to revolutionize cancer treatment, as it is developed by Genentech, a Roche company.
Genentech is exploring a revolutionary new treatment for patients with diffuse large B-cell lymphoma (DLBCL) who haven’t received prior lines of treatment: a combination of Polivy and the R-CHP regimen. This innovative approach has the potential to make a huge difference in the lives of those affected by this serious condition.
Genentech made a bold move in August 2022 when they submitted their sBLA alongside data from the Phase III POLARIX trial, showing that Polivy plus R-CHP had a remarkable 27% reduction in the risk of disease progression, relapse, or death compared to the current standard of care in DLBCL: R-CHOP.
The safety profiles of Polivy plus R-CHP and R-CHOP were remarkably similar, suggesting that the former may be a viable alternative to the latter.
After two decades of research, POLARIX has finally delivered a monumental breakthrough in the medical field. The study demonstrated a clinically significant improvement in progression-free survival over the standard of care. Genentech has submitted a supplemental Biologics License Application (sBLA) to the US Food and Drug Administration (FDA), which is due to reach a formal decision by April 2, 2023. POLARIX’s success is a major step forward in the fight against cancer, offering hope to those suffering from this devastating disease.
In 2019, the FDA gave the go-ahead to Polivy, a revolutionary new treatment for Diffuse Large B-Cell Lymphoma (DLBCL) that had proved resistant to two lines of therapy or more. This groundbreaking approval marks a major milestone in the fight against this debilitating form of cancer.
Acadia Readies for Landmark Rett Ruling
Acadia Pharmaceuticals is on the brink of a major milestone in the treatment of Rett syndrome as its candidate drug, trofinetide, awaits an FDA decision on March 12. Developed in collaboration with Neuren Pharmaceuticals, the successful approval of trofinetide could be a major breakthrough in the fight against this debilitating disorder.
If approved, trofinetide would be a revolutionary breakthrough in the treatment of Rett syndrome, a devastating neurodevelopmental disorder that primarily affects young girls. Characterized by a regression of developmental skills, loss of language and hand function, and impaired gait, this ultra-rare condition has long eluded medical treatment – until now. With the potential approval of trofinetide, a light of hope has been cast for those suffering from this affliction.
Investigating a revolutionary new solution to the struggle against neurological disorders, scientists have developed a synthetic analog of the amino-terminal tripeptide domain of the IGF-1 protein: Trofinetide. Unlike typical treatments that aim to reduce symptoms, this novel compound works by preventing the overactivation of microglia and astrocytes, promoting synaptic function, and reducing neuroinflammation – a pioneering approach that could revolutionize the way we treat neurological disorders.
In September 2022, Acadia submitted a New Drug Application for trofinetide, supported by data from the Phase III Lavender study. This study compared the candidate to placebo in 187 girls and young women, providing invaluable insight into the potential of this promising new drug.
Trofinetide treatment significantly improved co-primary endpoints, as indicated by changes in Rett Syndrome Behavior Questionnaire scores from baseline and Clinical Global Impression-Improvement scale scores, compared to placebo. Further, Lavender demonstrated impressive results in its key secondary endpoints.
The FDA has granted the groundbreaking drug trofinetide the highest level of priority review, Fast Track Status, Orphan Drug and Rare Pediatric Disease designations, signaling a major step forward in the fight against rare and debilitating pediatric diseases.
The FDA’s Office of Neuroscience is facing its second decision since the sudden departure of its former leader, Billy Dunn. With the pressure on, the team must grapple with the task of finding the right path forward. Will they be able to make the right decisions and move forward with confidence? Only time will tell.