What is Ninlaro and what is it used for?
Ninlaro is an anticancer medicine used to treat adult patients with multiple myeloma (a cancer of the bone marrow). It is given in combination with two other medicines, lenalidomide and dexamethasone, to patients who have received at least one previous cancer treatment.
Because the number of patients with multiple myeloma is low, the disease is considered 'rare' and Ninlaro was designated an 'orphan medicine' (a medicine used in rare diseases) on 27 September 2011.
Ninlaro contains the active ingredient ixazomib.
How is Ninlaro used - Ixazomib?
Ninlaro can only be obtained with a prescription. Treatment should be initiated and monitored by a physician experienced in the management of multiple myeloma.
Ninlaro is available in capsules (2.3, 3 and 4 mg) to be taken at least one hour before or two hours after meals. The recommended dose is 4 mg taken once a week (on the same day of the week) for 3 consecutive weeks, followed by a week without treatment with Ninlaro. This 4-week course of treatment should be continued until the disease worsens or the side effects become unacceptable. If the patient complains of some side effects, it may be necessary to temporarily stop the treatment or reduce the dose. The dose may also be reduced in patients with moderately or severely reduced liver function and in patients with severely reduced kidney function.
For more information, see the package leaflet.
How does Ninlaro - Ixazomib work?
The active substance in Ninlaro, ixazomib, is a proteasome inhibitor. This means that it blocks the proteasome, a complex inside the cells that breaks down proteins that are no longer needed. When proteins in cancer cells are not broken down, including proteins that control cell growth, cancer cells are damaged and eventually die.
What benefit has Ninlaro - Ixazomib shown during the studies?
Ninlaro was studied in one main study involving 722 adults with multiple myeloma whose disease did not respond to treatment or had recurred after previous therapy. The study compared Ninlaro with placebo (a dummy treatment), both taken in combination with lenalidomide and dexamethasone. An initial analysis of the data showed that Ninlaro is effective in prolonging the survival period of patients without worsening the disease (progression-free survival): the average period without worsening the disease was 21 months in patients treated with Ninlaro compared to 15 months of patients who received placebo. However, there is uncertainty regarding the extent of the improvement since a subsequent analysis of the data showed a reduced effect.
What are the risks associated with Ninlaro - Ixazomib?
The most common side effects with Ninlaro taken in combination with lenalidomide and dexamethasone (which may affect more than 1 in 5 people) were diarrhea, constipation, thrombocytopenia (low platelet count), peripheral neuropathy (damage to the nerves in the hands and feet which cause tingling or numbness), nausea, peripheral edema (swelling, especially of the ankles and feet), vomiting and back pain. Similar undesirable effects were observed when lenalidomide and dexamethasone were used without Ninlaro.
For the full list of restrictions and side effects reported with Ninlaro, see the package leaflet.
Why has Ninlaro - Ixazomib been approved?
Data from the main study indicate that Ninlaro improves progression-free survival of patients. However, due to the uncertainty regarding the extent of the improvement following a subsequent analysis, further confirmation data must be provided by the company that markets the medicine. Furthermore, Ninlaro does not appear to significantly increase the frequency of serious side effects when added to lenalidomide and dexamethasone, and offers the benefit to patients of being able to take the capsules at home.
The Agency's Committee for Medicinal Products for Human Use (CHMP) therefore decided that Ninlaro's benefits are greater than its risks and recommended that it be approved for use in the EU.
Ninlaro has obtained a "conditional approval". This means that further information on the medicine, which the company is required to provide, will be available in the future. Every year the European Medicines Agency will review the new information available and this summary will be updated accordingly.
What information is still awaited for Ninlaro?
Since a conditional approval was issued for Ninlaro, the company that markets Ninlaro will provide additional data on the benefits of this medicine from other studies, including a study of patients who have not been treated previously.
What measures are being taken to ensure the safe and effective use of Ninlaro - Ixazomib?
The company that markets Ninlaro will provide the final data of the main study related to the effects of the medicine on overall survival.
Recommendations and precautions to be followed by healthcare professionals and patients for Ninlaro to be used safely and effectively have also been included in the summary of product characteristics and the package leaflet.
More information on Ninlaro - Ixazomib
For the full version of EPAR and the summary of the Ninlaro risk management plan, consult the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information on Ninlaro therapy, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
The summary of the opinion of the Committee for Orphan Medicinal Products related to Ninlaro is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
