Syndax Partners with WODA to Expand Global Access to Revuforj Through Multi-Regional Managed Access Program
核心洞察
Syndax Pharmaceuticals and the World Orphan Drug Alliance (搜索) announced a collaboration to launch a Managed Access Program for Revuforj (revumenib) in regions where the first-in-class menin (搜索) inhibitor is not commercially available.
The program will expand access to Revuforj in parts of Eurasia, Central and Southeast Europe, Israel, the Middle East and Turkey, Latin America, and Africa for patients with relapsed or refractory acute leukemia.
Revuforj is FDA-approved for treating relapsed or refractory acute leukemia with KMT2A (搜索) translocations and acute myeloid leukemia with NPM1 (搜索) mutations in adult and pediatric patients one year and older.
Syndax Pharmaceuticals and the World Orphan Drug Alliance (搜索) (WODA (搜索)) have announced a collaboration to expand global access to Revuforj® (revumenib), a first-in-class menin (搜索) inhibitor, through a multi-regional Managed Access Program. The initiative will provide access to the FDA-approved acute leukemia treatment in regions where it is not commercially available, targeting parts of Eurasia, Central and Southeast Europe, Israel, the Middle East and Turkey, Latin America, and Africa.
Program Structure and Implementation
The Managed Access Program enables physicians to prescribe Revuforj to appropriate patients outside the United States where the drug is not approved but access to novel medicines is permitted by local regulations and funding can be secured. WODA (搜索), which operates in over 150 countries across six continents, will administer the program and provide Revuforj to healthcare providers on a named patient basis through its network of members.
"We are thrilled to partner with WODA (搜索) to begin expanding access to Revuforj around the globe, further advancing our mission to transform care for cancer patients," said Anjali Ganguli, Ph.D., Chief Strategy Officer at Syndax Pharmaceuticals. "In addition to providing a pathway for patients to access Revuforj in regions where it would otherwise be inaccessible, this program will also allow more physicians to gain valuable firsthand experience with the medicine, supporting our long-term goal to establish Revuforj as a standard of care treatment globally."
Revuforj's Approved Indications and Clinical Profile
Revuforj is FDA-approved for treating relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A (搜索)) translocation as determined by an FDA-authorized test in adult and pediatric patients one year and older. The drug is also approved for R/R acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1 (搜索)) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options. These blood cancers are associated with limited treatment options and poor prognosis.
The oral menin (搜索) inhibitor has received multiple regulatory designations, including Orphan Drug Designation for AML, ALL, and acute leukemias of ambiguous lineage from the U.S. FDA and for AML from the European Commission. The FDA also granted Fast Track designation for adult and pediatric patients with R/R acute leukemias harboring a KMT2A (搜索) rearrangement or NPM1 (搜索) mutation, and Breakthrough Therapy Designation for patients with R/R acute leukemia harboring a KMT2A rearrangement.
Safety Profile and Clinical Trial Data
Clinical trials involving 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia revealed important safety considerations. Differentiation syndrome (DS) occurred in 60 patients (25%), with higher rates observed in patients with KMT2A (搜索) translocations: 33% in AML patients, 33% in mixed-phenotype acute leukemia patients, and 9% in acute lymphoblastic leukemia patients. DS occurred in 18% of patients with NPM1m AML. The condition was Grade 3 or 4 in 12% of patients and fatal in 2 patients, with a median time to onset of 9 days.
QTc interval prolongation was reported as an adverse reaction in 86 patients (36%), with Grade 3 prolongation in 15% and Grade 4 in 2%. The QTcF was greater than 500 msec in 10% of patients, and the increase from baseline QTcF was greater than 60 msec in 24%. One patient experienced a fatal cardiac arrest, and one patient had non-sustained Torsades de Pointes.
Addressing Global Access Challenges
Patrick Jordan, Chairman at WODA (搜索), emphasized the collaboration's significance in addressing healthcare disparities: "This collaboration underscores WODA's mission to bridge the access gap for patients with rare and life-threatening diseases. Our alliance model enables us to reach patients in regions where access to innovative therapies, including in oncology, remains a critical challenge."
The program will be conducted in accordance with local regulatory and ethical frameworks to ensure compliant, patient-focused distribution. Healthcare professionals may inquire about program details by contacting medinfo@syndax.com, while patients and caregivers should contact their physicians for information.
Multiple trials of revumenib are ongoing or planned across the treatment landscape, including combination studies with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML, potentially expanding the drug's therapeutic applications in the future.