Oncopeptides has formally opened sEAPort, its open-label expanded access program allowing adults with triple-refractory multiple myeloma to access the investigational medicine melflufen (melphalan flufenamide) before its approval.
sEAPort (NCT04534322) is available to patients in the U.S. who tried at least two prior lines of therapy and failed to respond to at least one proteasome inhibitor, one immunomodulatory agent (IMiD), and one anti-CD38 monoclonal antibody.
The program is expected to enroll 100 to 200 adults at up to 50 clinical sites across the U.S. More information on the sEAPort program can be found here.
“Despite therapeutic advances, multiple myeloma remains incurable. There is an urgent need for more therapies as patients become multi-resistant earlier in their treatment journey,” Paula O’Connor, MD, U.S. head of Medical Affairs at Oncopeptides, said in a press release.
“Our Expanded Access Program enables us to provide access to melflufen as a potential treatment for eligible patients while our application is under review by the U.S. Food and Drug Administration,” O’Connor said.
sEAPort was launched following the company’s recent submission of a new drug application (NDA) to the U.S. Food and Drug Administration (FDA), requesting accelerated approval of melflufen plus dexamethasone for those with hard-to-treat multiple myeloma. The agency granted priority review to Oncopeptides’ application in August.
The sEAPort program and the company’s NDA were both supported by findings from the pivotal and open-label Phase 2 HORIZON study (NCT02963493), whose 157 patients were treated with melflufen plus dexamethasone for up to two years.
Top-line data from the study showed that 26% of patients with triple-refractory disease responded to treatment, which lasted a median of 4.4 months. Those who responded to treatment also lived longer without showing signs of disease worsening (median of 8.5 vs. 3.9 months), and had an overall survival of 11.2 months.
Most patients (94%) reported severe or life-threatening side effects, caused mostly by low levels of certain types of immune cells. Although not linked to treatment, 10 patients died during the trial.
Melflufen is a peptide-drug conjugate that specifically targets cancer cells. The medication is designed to be rapidly taken up by myeloma cells in which enzymes called aminopeptidases trigger the release of a cancer-killing agent (alkylating agent) that ends up destroying them.
Expanded access, or compassionate use, programs are designed to help people with serious or life-threatening conditions gain access to experimental medicines that are under investigation when other therapies have failed, no suitable treatments exist, or patients do not qualify to participate in clinical trials
The FDA signed a prescription drug user fee act (PDUFA) with an action date of Feb. 28, 2021. By then, the U.S. agency must decide if the combination therapy of melflufen and dexamethasone should be approved for those with hard-to-treat multiple myeloma.
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