{"entity":{"id":"panobinostat","kind":"drug","name":"Panobinostat","aka":[],"tldr":"An HDAC inhibitor for relapsed myeloma approved in 2015 and withdrawn in 2021 after its confirmatory trial was never completed.","summary":"Panobinostat is a pan-HDAC inhibitor that blocks class I, II and IV histone deacetylases including HDAC6. Inhibiting HDAC6 disrupts aggresome-mediated disposal of misfolded proteins, which synergises with proteasome inhibition in myeloma. PANORAMA-1 showed median progression-free survival of 12 versus 8.1 months when panobinostat was added to bortezomib-dexamethasone, at the cost of severe diarrhoea and cardiac events. The FDA granted accelerated approval in 2015 restricted to patients after at least two regimens including bortezomib and an immunomodulatory drug, and the EU approved it the same year. The confirmatory trial was never completed, and Secura Bio withdrew the US approval in 2021. Panobinostat showed that HDAC inhibition adds real activity in myeloma, but its toxicity meant the trade-off never earned a permanent place.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Panobinostat","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Panobinostat"}],"tags":["gap-fill"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":[],"companies":["novartis","securabio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00742027","nct02506959","nct00880269"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Farydak","modality":"Pan-HDAC inhibitor","mechanism":"Inhibits class I, II and IV HDACs including HDAC6, disrupting aggresome-mediated protein disposal and synergising with proteasome inhibition in myeloma.","approvals":[{"region":"US","year":2015,"indication":"Multiple myeloma after ≥2 regimens including bortezomib and an IMiD","note":"Withdrawn 2021"},{"region":"EU","year":2015,"indication":"Relapsed/refractory multiple myeloma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2015-02-23","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with bortezomib (BTZ) and dexamethasone (DEX) for the treatment of patients with multiple myeloma (MM) who have received at least 2 prior regimens, including BTZ and an immunomodulatory agent."},{"date":"2022-03-24","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 7.1 years after its accelerated approval.","indication":"In combination with bortezomib (BTZ) and dexamethasone (DEX) for the treatment of patients with multiple myeloma (MM) who have received at least 2 prior regimens, including BTZ and an immunomodulatory agent."}]},"route":"/drugs/panobinostat/","neighbours":{"cancer":[{"id":"multiple-myeloma","kind":"cancer","name":"Multiple myeloma","route":"/cancers/multiple-myeloma/"}],"technology":[{"id":"epigenetic-drugs","kind":"technology","name":"Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)","route":"/technologies/epigenetic-drugs/"}],"company":[{"id":"novartis","kind":"company","name":"Novartis","route":"/companies/novartis/"},{"id":"securabio","kind":"company","name":"SecuraBio","route":"/companies/securabio/"}],"trial":[{"id":"nct00880269","kind":"trial","name":"Efficacy and Safety of Panobinostat (LBH589) in Patients With Refractory de Novo or Secondary Acute Myelogenous Leukemia (AML)","route":"/trials/nct00880269/"},{"id":"nct02506959","kind":"trial","name":"Panobinostat, Gemcitabine Hydrochloride, Busulfan, and Melphalan Before Stem Cell Transplant in Treating Patients With Refractory or Relapsed Multiple Myeloma","route":"/trials/nct02506959/"},{"id":"nct00742027","kind":"trial","name":"Phase II Study of Oral Panobinostat in Adult Participants With Relapsed/Refractory Classical Hodgkin's Lymphoma","route":"/trials/nct00742027/"},{"id":"nct05725200","kind":"trial","name":"Study to Investigate Outcome of Individualized Treatment in Patients With Metastatic Colorectal Cancer","route":"/trials/nct05725200/"}],"roadmap":[{"id":"epigenetics-roadmap","kind":"roadmap","name":"Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome","route":"/roadmaps/epigenetics-roadmap/"}]}}