FDA Action Alert: Immunocore, Reata, CTI, Gilead and Legend/Janssen

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

The end of February picked up significantly from early this year for the U.S. Food and Drug Administration and its PDUFA dates. Read on for more details.

Immunocore’s Tebentafusp for Metastatic Uveal Melanoma

Immunocore had a target action date of February 23, 2022 for its Biologics License Application (BLA) regarding tebentafusp for treatment of HLA-A*02:01-positive adults with metastatic uveal melanoma (mUM). It was under Priority Review. The FDA approved the drug on January 26 under the brand name Kimmtrak. It was also being reviewed under the Real-Time Oncology Review (RTOR) pilot program, an initiative of the FDA’s Oncology Center of Excellence. RTOR is designed to speed the delivery of safe and effective cancer therapies. It is also under review via the FDA’s Project Orbis initiative, which allows it to be reviewed at the same time by partner countries that have requested participation.

Tebentafusp is a novel bispecific protein made up of a soluble T cell receptor fused to an anti-CD3 immune-effector function. It specifically targets gp100, a lineage antigen expressed in melanocytes and melanoma.

Reata’s Bardoxolone Methyl for Chronic Kidney Disease Caused by Alport Syndrome

Reata Pharmaceuticals has a target action date of February 25 for its New Drug Application (NDA) for bardoxolone methyl for patients with chronic kidney disease (CKD) caused by Alport syndrome. It was based on the data from the CARDINAL Phase III clinical trial. Alport syndrome is a rare, genetic type of CKD caused by mutations in the genes that code for type IV collagen, a major structural component of the glomerular basement membrane in the kidney. The drug is a once-daily, oral Nrf2 activator. Nrf2 is a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress and inhibiting pro-inflammatory signaling.

CTI BioPharma’s Pacritinib for Myelofibrosis

CTI BioPharma has a target action date of February 28 for its NDA for pacritinib for adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF) with a baseline platelet count of <50 x 109/L. Originally, the company had a PDUFA date of November 30, 2021, but during product labeling discussions, the FDA requested additional clinical data, which the company supplied. The FDA considered it a major amendment to the NDA and pushed the date back by three months.

The drug is a novel oral kinase inhibitor with specificity for JAK2, IRAK1 and CSF1R, without inhibiting JAK1. The NDA was based on data from the Phase III PERSIST-1 and 2 trials and the Phase II PAC203 trials.

Gilead’s Lenacapavir for Multi-Drug Resistant HIV

Gilead Sciences has a target action date of February 28 for its NDA for lenacapavir for treatment of multi-drug resistant HIV-1 infection in heavily treatment-experienced patients in combination with other antiretroviral therapies. It is under Priority Review.

On February 16, the company announced new one-year data from the ongoing Phase II/III CAPELLA trial of the drug. The data showed that lenacapavir, dosed subcutaneously every six months with other antiretrovirals, hit high rates of virologic suppression and clinically meaningful increases in CD4 counts.

Legend Biotech’s Ciltacabtagene Autoleucel for Multiple Myeloma

Legend Biotech has a target action date of February 28 for its BLA for ciltacabtagene autoleucel (cilta-cel) for adults with relapsed and/or refractory multiple myeloma. The therapy is a BCMA-directed chimeric antigen receptor T cell (CAR-T) therapy. The BLA was submitted by Legend’s collaboration partner, Janssen Biotech Inc., a Johnson & Johnson company. The original PDUFA date was October 28, 2021, but the FDA requested updated analytical method data, which Janssen supplied. As a result, the FDA extended the PDUFA date by three months.

Legend and Janssen inked their worldwide license and collaboration deal in December 2017. In December 2020, Legend launched a rolling submission of the BLA for cilta-cel, which was accepted under Priority Review in May 2021.

Back to news