Efficacy

Inqovi (decitabine and cedazuridine) for Leukemia

Inqovi, a combination of decitabine and cedazuridine, is an oral hypomethylating agent approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British (FAB) subtypes: refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia (CMML). MDS are a group of related blood disorders that can lead to leukemia. In clinical studies, Inqovi has demonstrated efficacy in improving hematologic parameters and reducing the need for red blood cell and platelet transfusions.

The efficacy of Inqovi for the treatment of these conditions was primarily based on results from two open-label, randomized, crossover trials. These trials compared the pharmacokinetics and pharmacodynamics of oral Inqovi to intravenous decitabine. Response rates, including complete remission (CR) and hematologic improvement, were similar between the oral and intravenous administration, indicating that Inqovi is an effective oral alternative to intravenous decitabine for patients with MDS and CMML.

Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) for Leukemia

Rylaze, also known as asparaginase erwinia chrysanthemi (recombinant)-rywn, is an asparagine-specific enzyme approved by the FDA as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in pediatric and adult patients who have developed hypersensitivity to E. coli-derived asparaginase. Rylaze works by depleting the amino acid asparagine, which is necessary for the growth of leukemia cells, thereby inhibiting their proliferation.

The efficacy of Rylaze was established in a clinical trial that included patients with ALL or LBL who were hypersensitive to E. coli-derived asparaginase. The trial measured the ability of Rylaze to achieve and maintain nadir serum asparaginase activity (NSAA) above the threshold believed to be therapeutically effective. Rylaze met the predefined efficacy criteria, with a high proportion of patients maintaining the therapeutic threshold of asparaginase activity, indicating its effectiveness in this patient population. The use of Rylaze allows for continued asparaginase treatment in patients who have developed hypersensitivity to other asparaginase preparations, which is critical for the successful management of ALL and LBL.