FDA Action Alert: Roche and Acadia
Pictured: FDA Sign/courtesy of Sarah Silbiger/Getty Images
After the flurry of verdicts and meetings at the end of February, the FDA has a relatively quiet week ahead with only two stand-out events.
Roche Pushes Polivy into the Frontline
On March 9, the FDA’s Oncologic Drugs Advisory Committee will convene to discuss Genentech’s supplemental Biologics License Application for Polivy (polatuzumab vedotin-piiq), the biotech’s anti-CD79b antibody-drug conjugate. Genentech is a Roche company.
Genentech is proposing to use Polivy, in combination with the R-CHP regimen, to treat patients with diffuse large B-cell lymphoma (DLBCL) who have not received prior lines of treatment.
To support its first-line bid, Genentech offered data from the Phase III POLARIX trial, which it submitted along with the sBLA in August 2022. The study showed Polivy plus plus R-CHP cut the risk of disease progression, relapse or death by 27% as compared with the R-CHOP regimen, the current standard of care in DLBCL.
Polivy plus R-CHP had a similar safety profile as R-CHOP.
With these results, POLARIX became the first study in 20 years to demonstrate a clinically meaningful improvement in progression-free survival over standard of care, Genentech reported. The FDA is expected to reach a formal verdict on Polivy’s sBLA by April 2, 2023.
In 2019, The FDA greenlit Polivy for the treatment of DLBCL that had progressed or returned after at least two prior lines of therapy.
Acadia Readies for Landmark Rett Ruling
Acadia Pharmaceuticals is hoping to notch an approval for its Rett syndrome candidate trofinetide, which has an FDA action date of March 12. Acadia is developing trofinetide in partnership with Neuren Pharmaceuticals.
If approved, trofinetide would be the first drug for Rett syndrome, an ultra-rare, multisystem neurodevelopmental disorder characterized by developmental regression, loss of language and hand function, and impaired gait. It primarily affects female children.
Trofinetide is an investigational and novel synthetic analog of the amino‐terminal tripeptide domain of the IGF-1 protein. It works by preventing the overactivation of microglia and astrocytes, promoting synaptic function and reducing neuroinflammation.
Acadia submitted the New Drug Application for trofinetide in September 2022 and backed it up with data from the Phase III Lavender study, which compared the candidate against placebo in 187 girls and young women.
The co-primary endpoints – change in Rett Syndrome Behavior Questionnaire scores from baseline and Clinical Global Impression-Improvement scale score – were significantly improved after trofinetide treatment, as compared with placebo. Lavender also hit key secondary endpoints.
The FDA granted trofinetide priority review, Fast Track Status, Orphan Drug and Rare Pediatric Disease designations.
This will be the second decision for the FDA’s Office of Neuroscience following the abrupt departure of its former head, Billy Dunn.