2 The technology

2 The technology

2.1 Panobinostat (Farydak, Novartis Pharmaceuticals) is an oral potent histone deacetylase inhibitor that disrupts a key mechanism in the transformation of normal cells to cancerous cells and selectively targets tumour cells for cell death. Panobinostat has received a marketing authorisation in combination with bortezomib and dexamethasone, for the treatment of 'adult patients with relapsed and/or refractory multiple myeloma who have received at least 2 prior regimens including bortezomib and an immunomodulatory agent'.

2.2 In the PANORAMA‑1 trial (comparing panobinostat plus bortezomib and dexamethasone with placebo plus bortezomib and dexamethasone), diarrhoea, thrombocytopenia, anaemia, fatigue and nausea occurred more often with panobinostat plus bortezomib and dexamethasone than with placebo plus bortezomib and dexamethasone.

2.3 Panobinostat costs £776 per 20 mg tablet. The recommended starting dose of panobinostat is 20 mg, taken orally once a day, on days 1, 3, 5, 8, 10 and 12 of a 21‑day cycle. Patients should have panobinostat for 8 cycles, after which it is recommended that patients showing clinical benefit continue the treatment for 4 additional cycles of 6 weeks each. The company has agreed a patient access scheme with the Department of Health. This scheme provides a simple discount to the list price of panobinostat, with the discount applied at the point of purchase or invoice. The level of the discount is commercial in confidence. The Department of Health considered that this patient access scheme does not constitute an excessive administrative burden on the NHS.

  • National Institute for Health and Care Excellence (NICE)