The drug regulator designated isatuximab, a treatment for relapsed and refractory multiple myeloma, as an orphan drug, granting additional indications for rufinamide and elotuzumab.
The Ministry of Food and Drug Safety named isatuximab as a new orphan drug on Monday, under regulations for the designation of orphan drugs.
Isatuximab received the orphan drug designation as “combination therapy with pomalidomide and dexamethasone in patients with multiple myeloma who previously received two or more treatments, including lenalidomide and proteasome inhibitors.”
Developed by Sanofi, isatuximab is a CD38-targeting monoclonal antibody designed for the treatment of recurrent and refractory multiple myeloma.
According to the ICARIA-MM phase-3 trial announced at the annual meeting of the American Society of Clinical Oncology (ASCO) in the U.S. in May, the combo of isatuximab, pomalidomide, and dexamethasone reduced disease progression and mortality risk by about 40 percent, compared to pomalidomide plus dexamethasone.
The median of the progression-free survival (PFS) was extended by five months, and overall response (OS) recorded 60 percent, much higher than 35 percent OS in the control group.
Further analysis showed that the combo of the three agents improved therapeutic effects in patients aged 75 or more, those with renal failure, and those with lenalidomide resistance.
Sanofi sought approval for isatuximab in Europe in the first half of the year. In the U.S., it also applied for the FDA’s nod in July.
The food and drug safety ministry also expanded the user age of the orphan drug rufinamide from four to one. Rufinamide is used as an adjunct therapy in the treatment of seizures associated with Lennox-Gastaut syndrome (LGS).
Elotuzumab, a multiple myeloma treatment, also won additional indication. The drug is newly indicated as “combination therapy with pomalidomide and dexamethasone in the treatment of multiple myeloma patients who have previously received two or more treatments, including lenalidomide and proteasome inhibitors.”