FDA Action Alert: Takeda, ChemoCentryx, Genmab and Seagen, and Avadel


The U.S. Food and Drug Administration (FDA) is kicking off the month of October with a number of PDUFA dates and an advisory committee meeting. Read on for more details.

Takeda Pharmaceuticals’ Maribavir for Cytomegalovirus Infection

The agency has scheduled a meeting for October 7, 2021, of the Antimicrobial Drugs Advisory Committee for Takeda Pharmaceuticals’ New Drug Application (NDA) for maribavir oral tablets. The NDA is for the drug for treatment of adults with post-transplant cytomegalovirus infection and/or disease, including infections resistant and/or refractory to ganciclovir, valganciclovir, cidofovir, or foscarnet. 

The application is built on data from the pivotal Phase III TAK-620-303 SOLSTICE trial. The drug was granted Orphan Drug Designation by the FDA for treatment of clinically significant CMV viremia and disease in at-risk patients, as well as Breakthrough Therapy Designation for CMV infection and disease in transplant patients resistant or refractory to previous therapies.

Maribavir is an antiviral that targets and inhibits the UL97 protein kinase and its natural substrates.

ChemoCentryx’ Avacopan for Autoantibody-Associated Vasculitis

ChemoCentryx has a target action date of October 7 for its NDA for avacopan for Antineutrophil Cytoplasmic Autoantibody (ANCA)-associated vasculitis (AAV). It originally had a PDUFA date three months earlier, but the agency requested additional information, which the company supplied. The FDA then declared the filing an amendment and extended the goal date.

The drug was approved in Japan on September 27 for treatment of microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA), the two main types of ANCA-associated vasculitis or ANCA vasculitis. It was approved under the brand name Tavneos. Tavneos is a first-in-class, oral small molecule that blocks the C5aR receptor for the pro-inflammatory complement system fragment known as C5a on destructive inflammatory cells such as blood neutrophils.

Genmab and Seagen’s Tisotumab Vedotin for Cervical Cancer

Genmab and Seagen had a target action date of October 10 for their Biologics License Application (BLA) for tisotumab vedotin for recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. The drug is an antibody-drug conjugate (ADC) directed to tissue factor (TF), a cell-surface protein found on multiple solid tumors. It is linked to tumor growth, angiogenesis, metastasis and poor prognosis.

On September 20, the FDA granted the drug accelerated approval under the brand name Tivdak. The approval was based on tumor response and the durability of the response. Continued approval is contingent on verification of clinical benefit in confirmatory trials.

In the innovaTV 204 trial, the drug was tested in 101 patients with recurrent or metastatic cervical cancer who had no more than two previous systemic treatments in the recurrent or metastatic setting, including at least one previous platinum-based chemotherapy treatment. The data showed 24% confirmed objective response rate (ORR), with a median duration of response (DOR) of 8.3 months. The drug comes with a Boxed Warning for ocular toxicity and warnings for peripheral neuropathy, hemorrhage, pneumonitis, and embryo-fetal toxicity.

“Once recurrent or metastatic cervical cancer progresses, there is a need for more options for these patients,” said Robert L. Coleman, Chief Scientific Officer, US Oncology Research and lead investigator of the innovaTV 204 trial. “This is an important development for patients with recurrent or metastatic cervical cancer.”

Avadel’s Sodium Oxybate for Narcolepsy

Avadel Pharmaceuticals has a target action date of October 15 for its NDA for its once-nightly formulation of sodium oxybate for daytime sleepiness and cataplexy in adults with narcolepsy. FT218 also includes the company’s MicroPump controlled-release (CR) technology.

At the company’s second-quarter financial report on August 9, Greg Divis, Chief Executive Officer of Avadel, said, “This quarter, we made significant progress advancing FT218, which we believe holds tremendous potential to transform the treatment landscape for patients as a once-at-bedtime option for managing EDS and cataplexy in adult patients with narcolepsy. The data presented at SLEEP 2021 demonstrates the additional benefit of consolidating sleep, which we believe holds great promise as many people with narcolepsy also suffer from fragmented sleep. As we enter the final stages of NDA review and approach our October PDUFA date, we remain confident in the strength of our regulatory filing strategy. In parallel, our commercial and launch preparations are on track, including the addition of key hires to our leadership team, and we look forward to providing more detail on our commercial strategy following potential approval of FT218.”

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