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The FDA has granted orphan drug designation to the oral agent OPN-6602 for the treatment of patients with relapsed or refractory multiple myeloma.
The FDA granted ODD to OPN-6602 for relapsed or refractory multiple myeloma: © stock.adobe.com
The Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the oral small molecule inhibitor OPN-6602 for the treatment of patients with relapsed or refractory multiple myeloma, according to a news release from Opna Bio, a clinical-stage biopharmaceutical company. The news release also added that the agent is currently being evaluated in the phase 1 OPN6602-C01 trial.
Orphan drug designation (ODD): a program that gives drugs and biologics for rare diseases certain benefits, including tax credits and market exclusivity.
Synergistic: when the combined effect of two or more drugs is greater than the sum of the individual effects.
Pharmacokinetic: The activity of drugs in the body over a period of time.
“We are pleased to have received ODD for OPN-6602 for the treatment of multiple myeloma, a further validation of the drug’s therapeutic potential in patients with this disease who have limited treatment options once they have relapsed,” Dr. Gideon Bollag, chief scientific officer of Opna Bio, stated in the news release.
The FDA grants agents ODD in order to "encourage the development of therapies for rare diseases, which are defined as those affecting fewer than 200,000 people in the U.S. The designation provides several benefits, including tax credits for clinical trial costs, a waiver of certain FDA fees and eligibility for seven years of market exclusivity upon approval," according to the news release.
In line with this, multiple myeloma is a rare cancer of the plasma cells, which is aggressive; it forms in the marrow that and often leads to serious complications such as bone damage, kidney failure and immune suppression. Furthermore, myeloma typically affects older patients and has limited treatment options available for those with relapsed or refractory disease.
OPN-6602 effectively suppresses selective oncogenes, leading to antitumor activity in multiple myeloma and the halt of myeloma cell growth.
Recently, the company presented data on the clinical agent at the 2024 American Society of Hematology (ASH) Annual Meeting in December. According to information shared at the meeting, data that were presented showed that, in human-derived multiple myeloma models, OPN-6602 suppressed tumor growth, while downregulating key multiple myeloma driving genes. Furthermore, OPN-6602 in combination with dexamethasone, Pomalyst (pomalidomide) and mezigdomide demonstrated synergistic effects. The distinct pharmacokinetic profile of the agent also allowed for continuous daily dosing that potentially results in a lower incidence of toxicities and improved efficacy.
The OPN6602-C01 clinical study is a phase 1b, open-label study which is investigating the safety, tolerability, pharmacokinetics and early antitumor activity of OPN-6602, both alone and in combination for patients with relapsed or refractory multiple myeloma. OPN-6602 is taken orally once daily in 21-day cycles.
Moreover, the study has three parts: part 1a evaluates OPN-6602 alone, part 1b examines it with dexamethasone and part 2 expands the preferred regimen, comparing two dose levels. Additionally, up to 130 patients will be enrolled onto the clinical trial.
The primary goal of parts 1a and 1b is to determine the recommended phase 2 dose study does based on tolerability and safety, while part 2 focuses on early antitumor activity using response rates.
In order to be eligible for enrollment, participants must have confirmed multiple myeloma, relapsed or refractory disease after at least three prior treatments, measurable disease and adequate organ function.
The phase 1 clinical trial of patients with relapsed or refractory multiple myeloma is taking place at multiple cancer centers across the United States. The company expects to complete the single agent, dose-escalation phase of the study in 2026, and further development of OPN-6602, the agent under investigation, in combination with other standard-of-care agents in multiple myeloma is planned, the news release concluded.
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