HQ Team
November 9, 2024: Johnson & Johnson has submitted regulatory applications to the US drug regulator and European Medicines Agency (EMA) for an extended approval of a blood cancer therapy.
The applications for monotherapy treatment of adult patients with high-risk smouldering myeloma were supported by data from ongoing end-stage trials, according to a company statement.
Myeloma, also known as multiple myeloma or plasma cell myeloma, is a blood cancer that affects the plasma cells in the bone marrow.
Plasma cells are white blood cells that produce disease- and infection-fighting antibodies in your body. Myeloma cells prevent the normal production of antibodies, leaving your body’s immune system weakened and susceptible to infection.
Early precursor
Smouldering multiple myeloma is an early precursor of active multiple myeloma, where abnormal cells can be detected in the bone marrow, but patients are typically asymptomatic.
Fifteen per cent of all cases of newly diagnosed multiple myeloma are classified as smouldering multiple myeloma, and half of those diagnosed with high-risk disease will progress to active multiple myeloma within two years.
Smouldering multiple myeloma is not generally treated until active multiple myeloma develops. The standard approach is to observe or track the disease for signs of biochemical progression or end-organ damage when treatment tends to be initiated.
“There remains an unmet need for early interventions and treatments that are both effective and well tolerated in people living with smouldering multiple myeloma at high risk of progressing to active multiple myeloma,” said Yusri Elsayed, MD, MHSc, PhD. Global Therapeutic Area Head, Oncology, Innovative Medicine, Johnson & Johnson.
‘Shifting treatment paradigm’
The therapy using daratumumab and hyaluronidase-fihj or branded by the company as Darzalex Faspro in the US and Darzalex in the European Union could become the first approved treatment for patients with high-risk smouldering multiple myeloma, “potentially shifting the treatment paradigm,” he said.
The first data from the study, evaluating the safety and efficacy of the drug, will be presented at the 2024 American Society of Hematology Annual Meeting, taking place in San Diego from December 7-10.
The main goal of the trials is progression-free survival, and secondary endpoints include time to progression, overall response rate and overall survival, according to the statement.
Patients in the study were diagnosed with smouldering multiple myeloma in the last five years and were excluded if they had prior exposure to approved or investigational treatments for smouldering multiple myeloma or multiple myeloma.
5-year survival rate
Multiple myeloma is the second most common blood cancer worldwide and remains an incurable disease.
In 2024, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma in the US and more than 12,000 will die from the disease. People with multiple myeloma have a 5-year survival rate of 59.8%.
While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.
