Roche's Glofitamab and Pralsetinib Granted Orphan Drug Designation by U.S. FDA for Rare Cancers

News on February 23, 2022 /BioValleyBIOON/ -- According to the U.S. Food and Drug Administration (FDA) According to the latest data from the Orphan Drug Database, 2 anticancer drugs from Roche have received orphan drug designation (ODD): (1) Glofitamab (formerly known as CD20-TCB) for the treatment of mantle cell lymphoma (MCL); (2) Pralsetinib for the treatment of metastatic RET fusion-positive solid tumors.

Glofitamab is aNovel CD20xCD3 T-cell Engaging Bispecific Antibody, and is currently being developed for the treatment of various types of lymphoma.Glofitamab can simultaneously bind to CD20 on the surface of malignant B cells and CD3 on the surface of T cells, bringing T cells close to tumor cells and eliminating them.TumorCell.Glofitamab has a novel structural pattern of "2:1", containing two Fab regions targeting CD20 and one Fab region targeting CD3.

Glofitamab has a dual-targeting action,Can activate and redirect patients' existing endogenous T cells, bind to target B cells, and eliminate these malignant B cells by releasing toxic proteins into them., providing an innovative approach for lymphoma treatment. Previously published data shows that glofitamab has demonstrated positive therapeutic responses across various types of non-Hodgkin's lymphoma (NHL).

Pralsetinib (Pralsetinib) is a precisionTumorStudy drugs, this drug is aAn orally administered, once-daily, potent and highly selective RET inhibitor designed to inhibit RET alterations (fusions and mutations, including predicted resistance mutations) that drive various cancers.Pralsetinib has demonstrated efficacy in treating various types of solid tumors, reflectingTumour-agnosticThe potential.

Pralsetinib (Pralsetinib) was designed by the research team at Blueprint Medicines based on their proprietary compound library. In preclinical studies, pralsetinib consistently demonstrated subnanomolar potency against the most common RET gene fusions, activating mutations, and resistance mutations. Furthermore, pralsetinib showed significantly enhanced selectivity for RET compared to approved multi-kinase inhibitors, with over 90-fold greater potency for RET than for VEGFR2. By inhibiting primary and secondary mutations, pralsetinib has the potential to overcome and prevent the occurrence of clinical resistance. This treatment is expected to achieve durable clinical responses in patients with various RET alterations while maintaining a favorable safety profile.

In July 2020, Roche entered into a $1.7 billion licensing and collaboration agreement with Blueprint Medicines, obtaining exclusive rights to Gavreto outside the United States (excluding Greater China) and co-commercialization rights in the U.S. market. According to the previous agreement signed with Blueprint,CStone Pharmaceuticals has exclusive rights to Gavreto in Greater China.。

In the United States, pralsetinib (brand name: Gavreto) has been approved for three indications: (1) for the treatment of...FDA(1) Approved testing methods confirm adult patients with metastatic RET fusion-positive NSCLC; (2) For the treatment of advanced or metastatic RET-mutant medullary thyroid cancer (MTC) in adults and pediatric patients aged 12 years and older requiring systemic therapy; (3) For the treatment of advanced or metastatic RET fusion-positive thyroid cancer in adults and pediatric patients aged 12 years and older requiring systemic therapy and who are radioactive iodine-refractory (if applicable).

In China, Gavreto (Pralsetinib) has been approved by the National Medical Products Administration (NMPA)., for the treatment of adult patients with locally advanced or metastatic NSCLC who are positive for rearranged during transfection (RET) gene fusion and have previously received platinum-based chemotherapy. The new indication application for Gavreto (Pralsetinib), used in advanced or metastatic RET-mutant medullary thyroid carcinoma (MTC) requiring systemic therapy, as well as advanced or metastatic RET fusion-positive thyroid cancer that requires systemic therapy and is radioactive iodine-refractory (if radioactive iodine is applicable), was accepted by the NMPA in April 2021 and included in the priority review. (Bioon.com)

Source of the original text:Orphan Drug Designations and Approvals