On July 2, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Lynozyfic (linvoseltamab-gcpt) to treat adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti‑CD38 monoclonal antibody (Press release, Regeneron, JUL 2, 2025, View Source [SID1234654222]). Lynozyfic was granted accelerated approval based on response rate and durability of response in the LINKER-MM1 trial. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
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Lynozyfic is the first FDA-approved BCMAxCD3 bispecific antibody that can be dosed every two weeks starting at week 14, and every four weeks if a very good partial response (VGPR) or better is achieved following completion of at least 24 weeks of therapy. The regimen includes hospitalization for safety during the step-up dosing period (one 24-hour period after the first step-up dose, and another 24-hour period after the second step-up dose).
"The FDA approval of Lynozyfic represents meaningful progress for the multiple myeloma community. Lynozyfic demonstrated early, deep and durable responses in heavily pre-treated patients, which I saw firsthand in clinical trials," said Sundar Jagannath, M.D., Network Director of the Center of Excellence for Multiple Myeloma at Mount Sinai in New York City and a trial investigator. "Lynozyfic has a convenient response-adapted dosing regimen, which provides the potential to extend time between doses. This is a significant patient-centric advancement that could help reduce treatment burden."
The FDA approval is based on results from the pivotal Phase 1/2 LINKER-MM1 trial investigating linvoseltamab in R/R MM in which patients (n=80) experienced a:
70% objective response rate (ORR), with 45% achieving a complete response (CR) or better, as determined by an independent review committee.
0.95 month median time to first response (range: 0.5 to 6 months).
Median duration of response (DoR) that was not reached (95% Confidence Interval [CI]: 12 months to not estimable). The estimated DoR was 89% at 9 months (95% CI: 77 to 95 months) and 72% at 12 months (95% CI: 54 to 84 months) among responders who had a median follow-up of 13 months.
The prescribing information for Lynozyfic has a Boxed Warning for cytokine release syndrome (CRS) and neurologic toxicity – including immune effector cell-associated neurotoxicity syndrome – in addition to warnings and precautions for infections, neutropenia, hepatotoxicity and embryo-fetal toxicity. The most common adverse reactions (≥20%) in the safety population of LINKER-MM1 (n=117) were musculoskeletal pain, CRS, cough, upper respiratory tract infection, diarrhea, fatigue, pneumonia, nausea, headache and dyspnea. The most common Grade 3 or 4 laboratory abnormalities (≥30%) were decreased lymphocyte count, decreased neutrophil count, decreased hemoglobin and decreased white blood cell count. Lynozyfic is available only through a restricted program called the Lynozyfic Risk Evaluation and Mitigation Strategy (REMS). Details of the Important Safety Information are included below.
"The FDA approval of Lynozyfic reinforces the strength of our bispecific antibody program as well as our commitment to delivering critical medicines to the cancer community," said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer of Regeneron. "With a 70% overall response rate in heavily pre-treated patients, we believe Lynozyfic is poised to potentially become a new standard of care for multiple myeloma. Furthermore, given the strength of the data, we are rapidly advancing our broad clinical development program for Lynozyfic – exploring its use in earlier lines of therapy as monotherapy and in novel combinations – as we aim to meaningfully advance care for patients."
Regeneron is committed to helping patients who have been prescribed Lynozyfic access their medication. The company has launched Lynozyfic Surround, which offers financial and educational resources to help support patients throughout their treatment journey. For more information, patients can call 1-844-RGN-HEME (1-844-746-4363).
"Even though the number of treatment options for multiple myeloma has expanded in recent years, it remains an incurable disease with considerable unmet need, especially among patients who have undergone multiple lines of treatment," said Diane Moran, R.N., M.A., Ed.M., Chief Executive Officer (Interim) and Senior Vice President of Strategic Planning at the International Myeloma Foundation. "The FDA approval of Lynozyfic is a welcome milestone. It provides appropriate multiple myeloma patients and their care teams with a novel patient-centric treatment option that includes a dosing schedule that can be adapted based on patient response. We appreciate Regeneron’s continued research to further advance treatment for this community."
About Multiple Myeloma
As the second most common blood cancer, there are over 187,000 new cases of MM diagnosed globally every year, with more than 36,000 diagnosed and 12,000 deaths anticipated in the U.S. in 2025. In the U.S., there are approximately 8,000 people who have MM that has progressed after three lines of therapy, and 4,000 whose disease has progressed after four or more therapies.
The disease is characterized by the proliferation of cancerous plasma cells (MM cells) that crowd out healthy blood cells in the bone marrow, infiltrate other tissues and cause potentially life-threatening organ injury. Despite treatment advances, MM is not curable and while current treatments are able to slow progression of the cancer, most patients will ultimately experience cancer progression and require additional therapies.
About Lynozyfic (linvoseltamab-gcpt)
Lynozyfic was invented using Regeneron’s VelocImmune technology and is a fully human BCMAxCD3 bispecific antibody designed to bridge B-cell maturation antigen (BCMA) on MM cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
Linvoseltamab is administered with an initial step-up dosing regimen followed by the full 200 mg dose administered weekly. At week 14, patients transition to every two-week dosing. A response-adapted regimen further enables patients to shift to every four-week dosing if they achieve and maintain a VGPR or better after having completed at least 24 weeks of therapy. Patients should be hospitalized for 24 hours after administration of the first step-up dose and for 24 hours after administration of the second step-up dose, with the potential for additional hospitalization if patients experience certain adverse events.
The generic name for Lynozyfic in its approved U.S. indication is linvoseltamab-gcpt with gcpt as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the FDA. Outside of the U.S., the generic name of Lynozyfic in its approved indications is linvoseltamab. Lynozyfic is also approved in the European Union to treat adults with R/R MM after at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy. For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu in due course.
About the Linvoseltamab Clinical Development Program
The ongoing, open-label, multicenter Phase 1/2 dose-escalation and dose-expansion LINKER-MM1 trial is investigating linvoseltamab in more than 300 enrolled patients with R/R MM. The Phase 1 intravenous dose-escalation portion of the trial – which is now complete – primarily assessed safety, tolerability and dose-limiting toxicities across nine dose levels of linvoseltamab and explored different administration regimens. A subcutaneous Phase 1 portion is ongoing. The Phase 2 intravenous dose expansion portion is ongoing and assessing the safety and anti-tumor activity of linvoseltamab, with the primary endpoint of ORR. Key secondary endpoints include DoR, progression-free survival, rate of minimum residual disease negative status and overall survival.
Linvoseltamab is being investigated in a broad clinical development program exploring its use as a monotherapy as well as in combination regimens across different lines of therapy in MM, including earlier lines of treatment, as well as MM precursor and other plasma cell disorders. This includes evaluating linvoseltamab in a Phase 1b trial (LINKER-MM2) in combination with other cancer treatments in R/R MM as well as a Phase 3 confirmatory trial (LINKER-MM3) as a monotherapy in R/R MM. For more information on Regeneron’s clinical trials in blood cancer, visit the clinical trials website, or contact via [email protected] or 1-844-734-6643.