Multiple myeloma is a cancer of the bone marrow that results in the production of abnormal blood plasma cells.
Approximately three in every 10,000 people are diagnosed with multiple myeloma worldwide each year (1), making it a rare disease. The average age for diagnosis is 70 years of age and there is currently no cure, with most patients dying within five years of their diagnosis.
Whilst patients being treated for multiple myeloma will experience symptom free periods, they will inevitably experience relapses as the disease develops a resistance to the treatments being used. At this point the disease is classified as relapsed and refractory multiple myeloma. Once the disease returns, either during treatment or within two months of the patient’s last treatment, the disease is classified as late-stage relapsed and refractory multiple myeloma.
Once the disease has reached its late-stages, patients suffer from symptoms including skeletal fractures and infections as a result of a weakened immune system as well as from the side effects of the treatments available today. In this stage of the disease, the focus of patient care becomes extending and improving quality of life. The development program for melflufen (Ygalo®) initially intends to improve the care for this group of patients.
In recent years, improved treatments have increased the five-year median survival rate for multiple myeloma patients to by more than 50%. These developments, together with an ageing population, are forecast to increase the number of patients undergoing treatment for late-stage relapsed and refractory multiple myeloma by 168% in 2023 compared to the number of patients receiving treatment in 2013.
The market for the use of pomalidomide, the current standard of care in late-stage relapsed and refractory multiple myeloma patients and the target patient group for melflufen, is expected to grow to USD 2.3 billion by 2019.
Therapies that have been granted orphan drug status, such as melflufen, usually come at a higher price, driven by the low incidence of the disease.
 Global Data: includes US, Japan, China (urban), France, Germany, Italy and the UK