Syndax Prescription drugs has gained FDA approval for a drug addressing superior instances of acute leukemia carrying a specific genetic signature that results in an aggressive type of the illness. The regulatory resolution makes the Syndax drug the primary remedy in a brand new class of medicines for blood cancers.
The FDA approval particularly covers the therapy of adults and youngsters age 1 and older. The Syndax drug, a twice-daily tablet identified in improvement as revumenib, might be marketed beneath the model identify Revuforj. The product’s late Friday approval got here six weeks forward of the goal date for a regulatory resolution.
In leukemia, the proliferation of irregular white blood cells prevents bone marrow from producing pink blood cells and platelets. Chemotherapy is a regular therapy. Blood transfusions don’t treatment the illness, however they will increase a affected person’s ranges of pink blood cells and platelets.
Waltham, Massachusetts-based Syndax designed Revuforj to inhibit a protein known as menin. In sufferers who’ve a rearrangement of the KMT2A gene, menin prompts pathways that drive most cancers development. This specific genetic rearrangement drives an estimated 10% of acute leukemias, Syndax mentioned in an investor presentation. Sufferers whose most cancers carries this genetic signature have poor prognoses and excessive charges of drug resistance and relapse. Revuforj is a small molecule that blocks the interplay of menin with KMT2A fusion proteins.
Syndax evaluated Revuforj in a single-arm, open-label Section 1/2 research that enrolled 104 members, each adults and pediatric sufferers. Outcomes confirmed that therapy led to finish remission or full remission with partial hematologic restoration in 21.2% of research members. The median period of the response was 6.4 months. Syndax mentioned 23% (24 out of 104) of sufferers within the research underwent stem cell transplants after therapy with Revuforj. This process can restore a affected person’s capability to provide blood cells. Outcomes from the research have been revealed in August within the Journal of Medical Oncology. The corporate mentioned extra information will introduced subsequent month in the course of the annual assembly of the American Society of Hematology.
Revuforj’s label carries a black field warning for the danger of differentiation syndrome, a complication wherein a most cancers drug triggers an extreme immune response from the affected leukemia cells. Differentiation syndrome can develop into deadly if it results in failure of key organs, comparable to the center. Based on the Revuforj’s label, if differentiation syndrome is suspected, clinicians ought to begin corticosteroid remedy and monitor the affected person’s blood circulation and coronary heart till signs resolve.
“The FDA approval of the primary menin inhibitor is a serious breakthrough for sufferers with [relapsed/refractory] acute leukemia with a KMT2A translocation, a genetic alteration related to a really poor prognosis,” Dr. Ghayas Issa, affiliate professor of leukemia at The College of Texas MD Anderson Most cancers Middle,” mentioned in Syndax’s announcement of the approval. “The numerous medical profit and strong efficacy seen with Revuforj represents a considerable enchancment over what has been traditionally noticed in these sufferers with beforehand out there therapies and has the potential to be an necessary new therapy possibility for sufferers.”
Whereas Revuforj is now the primary FDA-approved menin inhibitor, potential opponents are on its heels. Kura Oncology has reached the Section 2 portion of a Section 1/2 take a look at of ziftomenib in superior acute myeloid leukemia. Icovamenib, a menin inhibitor from Biomea Fusion, is in early medical improvement in each liquid and stable tumors. Different corporations creating small molecule menin inhibitors embrace Johnson & Johnson, Sumitomo Dainippon, and Daiichi Sankyo.
In the meantime, Syndax is operating extra research that would help increasing Revuforj to make use of as an earlier line of therapy for leukemia characterised by a KMT2A translocation in addition to for leukemias pushed by a genetic mutation to mNPM1 gene, which is present in about 30% of acute myeloid leukemia instances. Final week, Syndax reported constructive preliminary Section 2 information in acute myeloid leukemia pushed by mNPM1. Within the first half of 2025, the corporate it plans to publish and current these outcomes at a medical convention and search FDA approval on this indication.
Syndax has priced Revuforj at $39,500 a month, which works out to $474,000 yearly earlier than any rebates or reductions. Dosing of Revuforj is based on a affected person’s weight. The corporate mentioned it expects the 110 mg and 160 mg tablets of the drug will develop into out there later this month by way of specialty distributors and specialty pharmacies. The bottom dose, 25 mg, is for sufferers who weigh lower than 40 kg (about 88 kilos). Syndax expects this dose will develop into commercially out there subsequent 12 months, both late within the first quarter or early within the second quarter. Till then, Syndax will provide an oral resolution of this dose through an expanded entry program.
Picture: Getty Pictures, Sarah Silbiger
