As 2022 comes to its conclusion, the FDA has a handful of Prescription Drug User Fee Amendments still to wrap up. Here’s a look at that and more.
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As 2022 comes to its inevitable conclusion, the FDA has a handful of Prescription Drug User Fee Amendments (PDUFA) still to wrap up. Here’s a look at the key decisions.
Mirati’s Adagrasib for KRASG12C-Mutated NSCLC
Mirati Therapeutics has a target action date of Dec. 14 for its New Drug Application (NDA) for adagrasib in second-line non-small cell lung cancer with a KRASG12C mutation. It is being evaluated under an Accelerated Approval pathway and the FDA’s Real-Time Oncology Review (RTOR) pilot program.
The NDA is built on data from the Phase II registration-enabling cohort of the KRYSTAL-1 trial. The company announced positive topline data from the cohort in September 2021.
Adagrasib is a highly selective and potent small molecule designed to inhibit KRASG12C.
On Dec. 6, the company reported positive data for the drug with Merck’s checkpoint inhibitor Keytruda (pembrolizumab) as first-line treatment for NSCLC. That was preliminary data from the KRYSTAL-7 Phase II trial and KRYSTAL-1 Phase Ib cohort.
Coherus and Shanghai Junshi’s Toripalimab for Nasopharyngeal Carcinoma
Coherus Biosciences and Shanghai Junshi Biosciences Co. have a target action date of Dec. 23 for the Biologics License Application (BLA) resubmission for toripalimab in combination with gemcitabine and cisplatin in first-line advanced, recurrent or metastatic nasopharyngeal carcinoma (NPC).
The BLA is also for toripalimab monotherapy for second-line or later recurrent or metastatic NPC after platinum-containing chemotherapy.
The COVID-19 pandemic delayed the FDA’s ability to complete required inspections, necessitating a resubmission schedule.
There are no immunotherapies authorized in the U.S. for the treatment of NPC.
The BLA is based on data from the Phase III JUPITER-02 trial and the POLARIS-02 open-label Phase II study.
In China, regulators approved the drug in 2021 for two NPC indications. It is marketed in China as Ruoyi and has been approved for five indications.
Toripalimab is an anti-PD-1 monoclonal checkpoint inhibitor.
Gilead’s Lenacapavir for HIV Infection
Gilead Sciences has a target action date of Dec. 27 for its NDA resubmission for lenacapavir for heavily treatment-experienced people with multi-drug resistant HIV-1 infection.
The NDA was resubmitted on Jun. 27, 2022, and contained comprehensive Chemistry Manufacturing and Controls (CMC) data regarding an alternative vial type. The NDA was based on Phase II/III CAPELLA trial results.
Lenacapavir is a first-in-class, long-acting HIV capsid inhibitor. It was approved in Europe under the brand name Sunleca in August in combination with other antiretrovirals in adults with multi-drug resist HIV infection. It is dosed twice a year.
TG’s Ublituximab for Multiple Sclerosis
TG Therapeutics has a target action date of Dec. 28 for its ublituximab to treat relapsing forms of multiple sclerosis.
The drug is an investigational glycoengineered monoclonal antibody targeting a unique epitope on CD20-express B-cells. The BLA had an earlier PDUFA goal date, but after the FDA requested additional data, the agency determined it was a major amendment and pushed it back three months.
The BLA was built on data from the ULTIMATE I and II Phase III trials, which compared ublituximab to Sanofi’s Aubagio (teriflunomide) in patients with relapsing MS.
