On 25 June 2015, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product panobinostat (Farydak), intended for the treatment of relapsed and/or refractory multiple myeloma.
Farydak will be available as capsules of 10, 15 and 20 mg. The active substance of Farydak is panobinostat, a histone deacetylase (HDAC) inhibitor (ATC code: L01XX42) that inhibits the enzymatic activity of HDACs resulting in increased acetylation of histone proteins, leading to transcriptional activation.
The benefits with Farydak, in combination with bortezomib and dexamethasone, are its ability to delay the progression of disease.
The most common side effects are diarrhoea, fatigue, nausea, vomiting, thrombocytopenia, anaemia, neutropenia and lymphopenia.
The full indication is:
‘‘Farydak, in combination with bortezomib and dexamethasone, is indicated for the treatment of adult patients with relapsed and/or refractory multiple myeloma who have received at least two prior regimens including bortezomib and an immunomodulatory agent".
It is proposed that treatment with Farydak should be initiated by a physician experienced in the use of anti-cancer therapies.
Detailed recommendations for the use of this product will be described in the summary of product characteristics, which will be published in the European public assessment report and made available in all official European Union (EU) languages after the marketing authorisation has been granted by the European Commission.
Panobinostat in multiple myeloma
Multiple myeloma is generally an incurable disease that leads to bone destruction and kidney failure. In 2012, around 39,000 people had multiple myeloma in the EU. Around 50% of patients diagnosed with multiple myeloma under current treatment are still alive after five years.
The recommendation from CHMP is based on a study of Farydak in combination with bortezomib and dexamethasone in 768 patients with multiple myeloma, 193 of whom had received at least two prior treatments that included bortezomib and an immunomodulatory agent. Participants were randomly assigned to receive a combination of Farydak, bortezomib and dexamethasone, or bortezomib and dexamethasone alone.
The study found that among patients who had received prior treatment with bortezomib and an immunomodulatory agent, the disease progressed more slowly (by a median of 4.8 months) with Farydak treatment.
The most common side effects of Farydak were blood disorders, haemorrhage, diarrhoea, nausea, vomiting and fatigue.
In the context of these sometimes serious side effects, the CHMP considered that the overall benefit-risk balance for Farydak is only positive in patients with relapsed and/or refractory multiple myeloma who have received at least two prior regimens including bortezomib and an immunomodulatory agent.
A follow-up plan to monitor the safety of Farydak and lower its risks was agreed by the CHMP.
Because multiple myeloma is rare, Farydak was designated as an orphan medicine by the Committee for Orphan Medicinal Products (COMP) in 2012. Orphan designation gives medicine developers access to incentives such as fee reductions for scientific advice and is the key instrument available in the EU to encourage the development of medicines for patients with rare diseases.
The opinion adopted by the CHMP at its June 2015 meeting is an intermediary step on Farydak’s path to patient access. The CHMP opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.