Switzerland’s Agency for Therapeutic Products (Swissmedic) has awarded PharmaMar’s Aplidin orphan drug status to treat patients with multiple myeloma, following a similar designation by the U.S. Food and Drug Administration (FDA).
This decision was also supported by data regarding the drug’s efficacy, safety and quality, and well as the prevalence of multiple myeloma.
“This authorization is an important milestone in the treatment of multiple myeloma, recognizing that Aplidin, with its novel mechanism of action, could become a therapeutic alternative,” Luis Mora, managing director of PharmaMar’s oncology business unit, said in a news release.
Aplidin is an experimental anticancer drug originally obtained from the marine invertebrate Aplidium albicans. By binding to the eEF1A2 protein, which is involved in protein synthesis, Aplidin inhibits this process in tumors, leading to cell death.
This treatment produced promising results in the ADMYRE Phase 3 trial (NCT01102426), which investigated the safety and efficacy of Aplidin in combination with dexamethasone, compared to dexamethasone alone. The randomized, multicenter, open-label study included 255 patients with relapsed or refractory multiple myeloma, recruited from more than 80 medical centers in the United States and 18 other countries. All patients had at least three, but no more than six, prior therapeutic regimens to treat their disease.
The study’s primary objective was to compare the efficacy of Aplidin plus dexamethsone versus dexamethasone alone by measuring progression-free survival. Secondary endpoints in the study included response rate, duration of response and overall survival. Results showed that the combo therapy induced a “statistically significant reduction” of 35 percent in the risk of progression or death compared to dexamethasone alone, meeting the study’s primary endpoint.
Aplidin is also under investigation as a potential treatment for blood cancers. The drug is being evaluated in a Phase 1b clinical trial (NCT02100657) testing its effect in combination with Velcade (bortezomib) and dexamethasone in relapsed or refractory multiple myeloma, and in a Phase 2 trial in patients with relapsed or refractory angioimmunoblastic T-cell lymphoma.
Multiple myeloma is a relatively uncommon cancer. The lifetime risk of developing this disease is one in 143 (0.7 percent), according to the American Cancer Society, with around 30,000 new cases likely to be diagnosed in the United States this year.
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