Five New Drugs Headed Towards Early Approval Following FDA Priority Review Designations

Five New Drugs Headed Towards Early Approval Following FDA Priority Review Designations

The U.S. FDA has granted priority review designations to five new drugs in 2020, giving these treatments the potential for early approval. Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. This designation shortens the FDA review period following the acceptance of the NDA to six months compared to ten months under Standard Review. 

Novartis was granted priority review for capmatinib (INC280), a MET inhibitor for first line and previously treated patients with locally advanced or metastatic MET exon 14 skipping (METex14) mutated non-small cell lung cancer (NSCLC). If approved, capmatinib will be the first therapy to specifically target METex14 mutated advanced lung cancer, a type of lung cancer with a particularly poor prognosis. The FDA previously granted Breakthrough Therapy designation for capmatinib. The NDA submission for capmatinib is supported by results from the GEOMETRY mono-1 Phase II study, which demonstrated an overall response rate of 67.9% among treatment-naïve and previously treated patients, respectively, based on the Blinded Independent Review Committee (BIRC) assessment per RECIST v1.1. Capmatinib was licensed to Novartis by Incyte Corporation in 2009. Under the Agreement, Incyte granted Novartis worldwide exclusive development and commercialization rights to capmatinib and certain back-up compounds in all indications.

Kite Pharma’s CAR T cell therapy, KTE-X19, received priority review for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL). KTE-X19 was also designated a breakthrough drug by the FDA. The BLA is supported by data from the single arm, open-label, Phase 2 ZUMA-2 trial, which showed that 93% of patients responded to a single infusion of KTE-X19, including 67% of patients achieving a complete response, as assessed by an Independent Radiologic Review Committee (IRRC; median follow-up of 12.3 months). In the safety analysis, Grade 3 or higher cytokine release syndrome (CRS) and neurologic events were seen in 15% and 31% of patients, respectively. No Grade 5 CRS or neurologic events occurred. 

Eli Lilly was granted priority review for selpercatinib (LOXO-292), for the treatment of patients with advanced RET fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer. Selpercatinib was designed to inhibit native RET signaling as well as anticipated acquired resistance mechanisms. The NDA is based on data from the LIBRETTO-001 Phase 1/2 trial in RET-altered lung and thyroid cancers. Selpercatinib has received both Breakthrough Therapy designation, as well as Orphan Drug designation.

GlaxoSmithKline received priority review for belantamab mafodotin (GSK2857916) for the treatment of patients with relapsed or refractory multiple myeloma whose prior therapy included an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. Belantamab mafodotin is an immunoconjugate comprising a humanized anti-B cell maturation antigen (BCMA) monoclonal antibody conjugated to the cytotoxic agent auristatin F via non-cleavable linker. The BLA included data from the DREAMM-2 study that showed an overall response rate of more than 30% in each group. At follow-up, the median duration of response lasted for 6.3 months and 6.9 months in groups that received different dosing levels. The median progression-free survival among patients who were given 2.5 mg/kg of belantamab mafodotin was 2.9 months compared with 4.9 months in the 3.4mg/kg group, GSK said. Belantamab mafodotin was previously granted Breakthrough Therapy designation.

Astex Pharmaceuticals announced its NDA for oral C-DEC (cedazuridine and decitabine) has been accepted for Priority Review as a treatment for adults with previously untreated intermediate- and high-risk MDS including CMML. C-DEC is a novel, orally administered fixed dose combination of cedazuridine, an inhibitor of cytidine deaminase, with the anti-cancer DNA hypomethylating agent, decitabine. By inhibiting cytidine deaminase in the gut and the liver, C-DEC is designed to allow for oral delivery of the approved DNA hypomethylating agent, decitabine, at exposures which emulate exposures achieved with the approved intravenous form of decitabine administered over 5 days. The NDA submission is based on data from the ASCERTAIN phase 3 study which evaluated the 5-day decitabine exposure equivalence of oral C-DEC and IV decitabine.