FDA Approval for Panobinostat in Multiple Myeloma Withdrawn in the United States
Panobinostat, which received accelerated approval by the FDA in February 2015 for patients with previously treated multiple myeloma, has had its indication withdrawn in the United States.
An accelerated FDA approval for panobinostat (Farydak) in combination with bortezomib (Velcade) and dexamethasone for the treatment of patients with multiple myeloma who have received at least 2 prior regimens including bortezomib and an immunomodulatory agent has been withdrawal in the United States, according to a press release from developer Secura Bio.1
The approval was based on progression-free survival (PFS) data from the phase 3 PANORAMA1 study (NCT01023308).2,3 Although the findings were consistent with FDA regulations, there was still a need for adequate and controlled clinical research to confirm the regimen’s clinical benefit. Secura Bio, following discussions with the FDA, came to the conclusion that it would not be possible to complete the necessary post-approval clinical research that was needed as part of the post–accelerated approval process. As the trials were a requirement, panobinostat’s clinical benefit has not yet been confirmed following the necessary constraints of the accelerated approval process.
In light of the withdrawn submission, there will be discussions centered around panobinostat at an upcoming meeting of the Oncologic Drugs Advisory Committee. The FDA is expected to publish a Federal Register notice detailing news of the withdrawal.
Data from the PANORAMA1 trial, which enrolled 768 patients with a median follow up of 6.47 months, demonstrated a median PFS of 11.99 months with the panobinostat regimen compared with 8.08 months in the placebo arm (HR, 0.63; 95% CI, 0.52-0.76; P <.0001). Additionally, immature overall survival (OS) data at the time the study was published indicated that patients in the panobinostat arm experienced a median OS of 33.64 months vs 30.39 months in the placebo group (HR, 0.87; 95% CI, 0.69-1.10; P = .26).
References
- Secura Bio announces U.S. withdrawal of FARYDAK ® (panobinostat) NDA. News release. Secura Bio. November 30, 2021. Accessed December 1, 2021. https://bit.ly/3okhY2R
- FDA approves panobinostat for some patients with multiple myeloma. News release. National Cancer Institute. March 19, 2015. Accessed December 1, 2021. https://bit.ly/31mQ2lP
- San-Miguel JF, Hungria VT, Yoon SS, et al. Panobinostat plus bortezomib and dexamethasone versus placebo plus bortezomib and dexamethasone in patients with relapsed or relapsed and refractory multiple myeloma: a multicentre, randomised, double-blind phase 3 trial. Lancet Oncol. 2014;15(11):P1195-1206. doi:10.1016/S1470-2045(14)70440-1
