FDA action alert: Verrica, Citius and Daiichi Sankyo

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FDA action alert: Verrica, Citius and Daiichi Sankyo

Published: Jul 17, 2023

By Tristan Manalac

BioSpace

After making a splash with the full approval of Eisai and Biogen’s Leqembi (lecanemab) on July 6, the FDA has just three scheduled target action dates coming up in the next two weekstwo for cancer and one for a viral skin disease. 

Verrica Hopes Third Time’s the Charm for Viral Skin Disease Treatment

On July 23, the FDA is set to make a decision on Verrica Pharmaceuticals’ investigational topical treatment VP-102 (cantharidin 0.7% topical solution), which is being proposed to treat molluscum contagiosum.

If approved, VP-102 would become the first authorized treatment for this viral skin infection.

Affecting some six million patients in the U.S. —primarily children—molluscum contagiosum is a highly contagious viral skin disease characterized by distinctive, raised pinkish lesions that are itchy and sometimes painful. In some cases, these bumps can also lead to inflammation and increase the risk of infection.

Molluscum contagiosum is caused by a pox virus that is easily transmissible via fomites or direct skin-to-skin contact. Though usually mild or benign, the disease can persist for several months or years when left untreated.

VP-102 is a drug-device combination containing a 0.7% formulation of cantharidin delivered through a single-use applicator that ensures precise dosing. Verrica has prepared a U.S. brand name of Ycanth if the treatment is approved.

This is Verrica’s third attempt at securing an approval for VP-102. The first was dashed in September 2021, when the FDA rejected its New Drug Application due to deficiencies at a contract manufacturing organization (CMO) facility, according to the company’s news release at the time. These issues were not directly related to VP-102’s manufacturing but pertained to more general quality problems at the plant.

CMO problems also thwarted Verrica’s second filing, which the FDA rejected in May 2022.

FDA to Decide on Daiichi Sankyo’s Quizartinib for AML

Following a three-month delay to provide more time for review, the FDA is scheduled on July 24 to decide on Daiichi Sankyo’s proposal to administer quizartinib in combination with standard chemotherapy to treat patients with acute myeloid leukemia (AML).

The FDA first accepted Daiichi Sankyo’s NDA in October 2022 and granted it Priority Review, which is meant to accelerate the regulator’s decision. However, in April 2023, the FDA pushed the action date back by three months to accommodate updates to the proposed Risk Evaluation and Mitigation Strategies for quizartinib.

Daiichi Sankyo is backing its NDA with data from the Phase III QuANTUM-First trial, which demonstrated that quizartinib can cut the risk of death by 22.4% in AML patients compared to chemotherapy alone. This advantage persisted until 40 months of follow-up, at which point the quizartinib arm had more than double the median overall survival of placebo comparators.

Quizartinib also had a “generally manageable” toxicity profile and led to no new concerning safety signals, according to a June 2022 press release. Still, 11.3% of quizartinib-treated patients died from treatment-emergent adverse events, most of which were infections. This was compared to 9.7% of patients receiving chemotherapy alone.

Quizartinib is a small molecule drug designed to be orally administered. It targets the tyrosine kinase receptor FLT3, which is commonly found on hematopoietic stem cells. Specifically, quizartinib targets FLT3 bearing the ITD mutation, which in AML is linked to worse survival and higher rates of relapse.

The FDA previously rejected an application for quizartinib as a monotherapy in patients with relapsed/refractory AML with FLT3-ITD mutations.

Citius Seeks Approval for I/ONTAK Ahead of Spin-Off Plans

Citius Pharmaceuticals is planning to spin out its immune asset I/ONTAK into a separate publicly-traded entity, pending the FDA’s verdict on the candidate, due July 28.

I/ONTAK, which Citius is proposing as a treatment for T-cell lymphoma, is a reformulation of Eisai’s Ontak (denileukin diftitox), which won initial approval in 1999 for the same indication. Citius’ candidate is a recombinant fusion protein that combines a diphtheria toxin with the interleukin-2 protein. It works by specifically binding to IL-2 receptors to precisely deliver its toxic payload, thereby preventing protein synthesis in malignant T cells.

I/ONTAK also targets the immunosuppressive regulatory T cells, which in turn allows the body to produce a stronger immune response against the cancer.

The therapeutic fusion protein is approved in Japan, where it is marketed under the brand name Lymphir.

The FDA accepted Citius’ Biologics License Application for I/ONTAK in December 2022 and initially gave it a target action date of Sept. 28, 2023. The regulator later shortened its review period and provided the updated deadline of July 28.  

Citius is backing I/ONTAK’s regulatory bid with data from a pivotal Phase III study. The company released positive topline data from this trial in April 2022, demonstrating that the candidate can elicit an overall response rate of 42.3%, indicating that nearly half of patients show partial or complete response to treatment.

Source: BioSpace