Annals of Hematology: Expert consensus supports use of Pepaxti in myeloma
Oncopeptides today announced that a panel of leading multiple myeloma experts has published clinical recommendations for the optimal use of melflufen (branded in Europe as Pepaxti) in Annals of Hematology. The review article provides practical guidance on patient selection, dosing, and management, reinforcing Pepaxti’s role as an important treatment option for patients with relapsed, refractory multiple myeloma (RRMM).
The expert group, led by Prof. Dr. Heinz Ludwig (Wilhelminen Cancer Research Institute, Vienna), assessed available clinical trial data together with real-world experience from European practice. Their consensus highlights that melflufen is a valuable treatment alternative for heavily pretreated patients, including elderly patients and those with high-risk disease, with a well-characterized and manageable safety profile.
“Based on both trial data and our clinical experience, we conclude that melflufen offers meaningful benefit to a broad spectrum of patients with relapsed or refractory myeloma, especially those with few remaining therapeutic options,” says Prof. Dr. Heinz Ludwig, lead author of the article. “Our recommendations aim to assist treating physicians in optimizing patient outcomes with melflufen in everyday clinical practice.”
“These expert recommendations add further weight to Pepaxti’s positioning as an effective therapy in later lines of treatment,” says Stefan Norin, Chief Medical Officer at Oncopeptides. “Importantly, they confirm the drug’s utility across different patient subgroups and align with the evidence base from both the pivotal HORIZON and OCEAN studies as well as real-world use.”
The article, “Optimizing the use of melflufen in relapsed or refractory multiple myeloma: Recommendations for clinical practice”, is available online in Annals of Hematology.
Q&A for Investors
What is this?
An independent panel of leading European myeloma experts has published practical guidance on the clinical use of Pepaxti in Annals of Hematology. These recommendations are based on both clinical trial evidence and real-world practice.
Why is this important?
Consensus recommendations in a high-impact peer-reviewed journal reinforce Pepaxti’s clinical credibility and support broader physician adoption. They confirm the drug’s value in treating heavily pretreated RRMM patients, many of whom have limited alternatives.
What are the key takeaways from the article?
- Patient selection: Pepaxti is recommended for triple-class refractory RRMM patients, including elderly patients, those with renal impairment, and selected high-risk subgroups.
- Dosing: The recommended starting dose is 40 mg intravenously every 28 days in combination with dexamethasone, with a reduced starting dose of 30 mg for patients ≤60 kg or with moderate renal impairment.
- Safety: The safety profile is well-characterized, with primarily hematologic adverse events (thrombocytopenia, neutropenia, anemia) that can be managed with standard supportive care and dose adjustments.
- Clinical benefit: Around one-third of patients achieve meaningful clinical benefit, with durable responses seen in both trial and real-world settings.
How does this impact Oncopeptides?
The publication underscores Pepaxti’s role in the RRMM treatment landscape and strengthens the product’s positioning with healthcare professionals and payers. Such consensus papers are important in driving physician confidence, guideline inclusion, and market uptake.
How does this compare to clinical trial results?
The recommendations are consistent with the efficacy and safety outcomes observed in the pivotal HORIZON and OCEAN trials, as well as with accumulating real-world data. This alignment reinforces Pepaxti’s clinical value across both controlled and real-world practice.
Who authored the article?
The expert panel included Prof. Dr. Heinz Ludwig (Vienna, Austria), Prof. Dr. Elias K. Mai (Heidelberg, Germany), Dr. med. Marion Högner (Munich, Germany), Dr. med. Manfred Welslau (Aschaffenburg, Germany), and Dr. med. Johannes M. Waldschmidt (Würzburg, Germany). Axel Glasmacher, MD, and Christoph Weiligmann, PhD, provided assistance in the preparation of the manuscript. Their editorial support was funded by Oncopeptides.
Does this change the approved label?
No. The consensus provides recommendations for clinical practice within the scope of Pepaxti’s existing approved label in the EU and UK.