Revolutionary Advances and Bright New Horizons in Multiple Myeloma Treatment

Multiple myeloma, a complex blood cancer rooted in the bone marrow, affects 160K people globally each year, with a mortality rate of 106K. According to DelveInsight, nearly 75K new cases were reported across seven major markets in 2023, a figure expected to climb in the coming years. Yet, amid these sobering statistics, a wave of progress is reshaping the multiple myeloma treatment landscape, offering new hope to patients facing this challenging disease.

Traditionally, the fight against multiple myeloma has leaned on chemotherapy and steroids—reliable, if broad, tools. But the story has shifted in recent years, with therapies like CAR-T cell therapy stepping in to deliver targeted immunotherapy for patients unresponsive to other treatments. Bispecific antibodies have also emerged, linking immune cells to cancer cells for precise attacks, while genomic studies are uncovering genetic markers to personalize care. These advancements signal a move toward smarter, more tailored solutions.

The approved multiple myeloma drug lineup reflects this evolution. Since 2015, therapies like NINLARO, the first oral proteasome inhibitor, and EMPLICITI, an immune enhancer, have broadened multiple myeloma treatment options. DARZALEX, targeting CD38, joined in 2015, followed by BLENREP in 2020 with its anti-BCMA approach. TECVAYLI and CARVYKTI arrived in 2022 and TALVEY in 2023, each adding depth to the arsenal. These multiple myeloma drugs have not only expanded choices but also improved outcomes for patients.

Discover more about the approved drugs shaping the multiple myeloma treatment landscape —Read Our Blog!

SARCLISA Takes Center Stage

As we discuss approved therapies, one making headlines is SARCLISA, first approved in 2020 for treating adult patients with multiple myeloma who have received at least two prior therapies. In September 2024, it earned approval from the FDA in combination with bortezomib, lenalidomide, and dexamethasone (VRd) as a first-line treatment for newly diagnosed multiple myeloma (NDMM) patients ineligible for autologous stem cell transplants. Data showed a 40% reduction in disease progression or death compared to VRd alone—a first for an anti-CD38 therapy in this setting.

The momentum continued into December 2024 at the 66th American Society of Hematology (ASH) Annual Meeting in San Diego. Three oral presentations, including results from the IMROZ and GMMG-HD7 phase III studies, highlighted SARCLISA’s ability to deliver deep, durable responses and better long-term outcomes when added to standard NDMM regimens. These findings solidified its growing role.

In January 2025, China’s National Medical Products Administration (NMPA) approved SARCLISA with pomalidomide and dexamethasone for patients with at least one prior therapy line, including lenalidomide and a proteasome inhibitor. A month later, in February 2025, Japan’s Ministry of Health, Labour and Welfare (MHLW) followed suit, approving SARCLISA with VRd for NDMM based on the IMROZ study. Each approval marks a step forward in global access.

SARCLISA is currently approved in over 50 countries, including the US, EU, Japan, and China, for multiple indications, and each approval marks a step forward in global access.

A New Era for Multiple Myeloma Patients

The fight against multiple myeloma is far from over, but with each breakthrough, the future becomes more hopeful. The focus is shifting toward personalized medicine, where treatments of multiple myeloma​ are tailored to individual disease characteristics, maximizing efficacy while minimizing side effects. As multiple myeloma therapies like SARCLISA redefine first-line treatment strategies and emerging options push the boundaries of what’s possible, long-term survival—and even the prospect of a cure—may one day be within reach.

The journey continues, but the path is increasingly well-lit. With new developments unfolding at a rapid pace, let’s explore the most recent breakthroughs shaping the future of the newest treatment for multiple myeloma and how they are transforming patient care.

April 2025

April 2025 has seen significant progress in multiple myeloma and hematologic cancer treatments, with key findings from the 51st Annual EBMT Meeting (March 30 – April 2, 2025) highlighting the potential of CAR-T therapy and novel targeted treatments.

New data from the phase III CARTITUDE-4 trial (NCT04181827) reinforced the efficacy of ciltacabtagene autoleucel (cilta-cel; Carvykti) in lenalidomide-refractory multiple myeloma. Among evaluable patients, 69% achieved MRD negativity at a 10–5 threshold by day 56, rising to 86% at six months. Even in the intention-to-treat (ITT) population, MRD-negativity rates reached 48% at day 56 and 60% at six months. With an odds ratio (OR) of 13.3 (P < .0001) in the MRD-evaluable group, cilta-cel continues to demonstrate deep and sustained responses, reinforcing its role as a transformative CAR-T therapy. Beyond multiple myeloma, the phase I AFM28-101 trial (NCT05817058) presented promising early data on AFM28, a novel therapy for CD123-positive relapsed/refractory AML. 

Also, new data from the phase III PERSEUS trial (NCT03710603), presented at the EBMT Meeting, confirmed that D-VRd (daratumumab, bortezomib, lenalidomide, and dexamethasone) followed by daratumumab/lenalidomide maintenance improved progression-free survival (PFS) and MRD negativity compared to VRd followed by lenalidomide maintenance in transplant-eligible newly diagnosed multiple myeloma. The study showed PFS benefits across all cytogenetic risk groups, including those with high-risk abnormalities, reinforcing D-VRd as a stronger frontline option for newly diagnosed patients.

With cilta-cel setting new benchmarks in multiple myeloma and AFM28 showing early promise in AML, April 2025 marked a significant step toward more effective, long-lasting treatments for patients battling hematologic cancers.

March 2025 

In March 2025, the FDA issued its first untitled letter of the year, addressing concerns over the promotional practices of Edenbridge Pharmaceuticals—now rebranded as Dexcel Pharma. The notice was sent after the agency reviewed an exhibit booth panel promoting Hemady, an oral formulation of the steroid dexamethasone used in multiple myeloma treatment.

Hemady was designed to reduce pill burden by delivering 20 mg of dexamethasone per tablet, compared to the 4 mg found in some existing formulations. While the exhibit highlighted the drug’s advantages, the FDA found deficiencies in the way data was presented. Following a review prompted by a complaint through the Bad Ad program, the agency took issue with missing information and questioned the robustness of the study supporting some of the claims.

This regulatory action underscores the FDA’s continued vigilance in ensuring accurate and balanced communication around multiple myeloma treatments, reinforcing the importance of comprehensive data disclosure in pharmaceutical promotions. 

Also in March, IASO Biotherapeutics announced that the Pharmaceutical Administration Bureau of Macau (ISAF) approved the New Drug Application (NDA) for Equecabtagene Autoleucel (FUCASO). This marks the therapy’s first approval outside Mainland China for relapsed/refractory multiple myeloma (R/RMM) in patients who have received at least three prior treatments, including a proteasome inhibitor and an immunomodulatory agent.

The approval is based on data from the FUMANBA-1 trial (NCT05066646), which demonstrated strong efficacy and a favorable safety profile. The NDA was also recently accepted in Singapore and Hong Kong, further advancing IASO Bio’s goal of expanding global access to its CAR-T therapies.

February 2025 

In February 2025, Opna Bio’s OPN-6602, an oral small molecule inhibitor of EP300 and CBP, received Orphan Drug designation (ODD) from the FDA for multiple myeloma. Currently in a phase I trial for relapsed/refractory multiple myeloma, OPN-6602 has shown promise in preclinical studies, achieving 71% tumor suppression as monotherapy and 100% tumor regression in combination with dexamethasone, pomalidomide, or mezigdomide. The company aims to explore its potential in combination therapies to enhance multiple myeloma treatment options​ for patients with resistant disease.

Also in February 2025, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved SARCLISA (isatuximab) in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for newly diagnosed multiple myeloma (NDMM). This decision, based on findings from the IMROZ phase III study, marks SARCLISA’s fourth approved regimen in Japan, further expanding its role in both relapsed/refractory and frontline treatment settings.

January 2025

In January 2025, China’s National Medical Products Administration (NMPA) approved SARCLISA (isatuximab) in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for newly diagnosed multiple myeloma (NDMM) patients ineligible for transplant. This makes SARCLISA the first anti-CD38 therapy approved with VRd in this patient population.

The approval is based on findings from the IMROZ phase III study, which demonstrated that Sarclisa-VRd significantly improved progression-free survival (PFS) compared to VRd alone. Notably, this is Sarclisa’s second approval in China within three weeks, following its R/R multiple myeloma indication on January 13, 2025.

A Robust Pipeline

Looking ahead, the multiple myeloma pipeline is bustling with potential. Arcellx’s Anito-Cel, Bristol Myers Squibb/Celgene’s Mezigdomide, Novartis’s PHE885, Regeneron Pharmaceuticals’s REGN5459, Cartesian Therapeutics’s Descartes-11, Heidelberg Pharma’s HDP-101, Beigene’s BGB-11417, Carsgen Therapeutics’s Zevorcabtagene Autoleucel, C4 Therapeutics’s CFT7455 are among the contenders, joined by efforts from Poseida Therapeutics, Ichnos Sciences, Nerviano Medical Sciences, Ascentage Pharma, Ionis Pharmaceuticals, Chongqing Precision Biotech, CRISPR Therapeutics, AstraZeneca, IGM Biosciences, and others. Spanning CAR-T therapies, novel inhibitors, and antibodies, these candidates promise to further transform care.

The multiple myeloma treatment landscape stands at a pivotal moment. With groundbreaking research, expanding access to innovative treatments, and a robust pipeline of novel therapies, patients have more options—and more reasons for optimism—than ever before.

Pioneering Progress: Transforming the Multiple Myeloma Treatment Frontier

The multiple myeloma treatment market is a testament to the power of innovation and perseverance. From the global reach of multiple myeloma therapies like SARCLISA, now approved in over 50 countries, to the promising data emerging from trials like CARTITUDE-4 and PERSEUS, the field is advancing at an inspiring pace. The journey that began with traditional tools like chemotherapy and steroids has evolved into a sophisticated narrative of precision medicine—CAR-T therapies, bispecific antibodies, and genomic insights are no longer just possibilities but realities, improving lives.

The pipeline, brimming with contenders from Arcellx, Bristol Myers Squibb, Novartis, and beyond, signals that this progress is far from slowing. Each approval, each trial result, and each regulatory milestone lights the way toward a future where multiple myeloma may not just be managed but potentially overcome. With the newest treatments for multiple myeloma, including cutting-edge CAR-T therapies, bispecific antibodies, and novel targeted agents, patients have more multiple myeloma treatment options​ than ever before. For patients, caregivers, and clinicians, this moment offers more than hope—it provides tangible solutions and a clearer path forward. The road ahead remains long, but with every step, the horizon grows brighter, promising a new era of care for those touched by this complex disease.