FDA Updates for Week of Oct. 24, 2022

FDA approves first bispecific antibody for multiple myeloma.

The FDA has granted accelerated approval for Johnson & Johnson’s Tecvayli (teclistamab-cqyv) to treat adult patients with relapsed or refractory multiple myeloma. Tecvayli is a first-in-class, bispecific T-cell engager antibody to treat patients who have previously received four or more prior lines of therapy. A bispecific antibody can bind to two different targets on two different cells at the same time. Tecvayli is an off-the-shelf therapy that targets the CD3 receptor expressed on the surface of T-cells and the B-cell maturation antigen (BCMA) expressed on the surface of multiple myeloma cells.

Tecvayli will be available around Nov. 4, 2022, and will be available through a risk management program. The list price is about $39,500 per month, according to a J&J spokesperson. In the clinical trial, the average patient was treated for 9 to 10 months. Based on this, the total cost of therapy ranges between a list price of $355,000-$395,000, the spokesperson said.

Multiple myeloma is an incurable blood cancer that affects plasma cells, which are found in the bone marrow. In 2022, it is estimated that more than 34,000 people will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease in the United States.

FDA clears novel liver cancer combo treatment.

The FDA has cleared AstraZeneca’s Imjudo (tremelimumab) in combination with the blockbuster drug Imfinzi (durvalumab) for unresectable hepatocellular carcinoma (HCC), the most common type of liver cancer.

Imjudo will be commercially available in the United States in early November 2022, Chelsea Ford, director of external communications for AstraZeneca U.S. Corporate Affairs, told FormularyWatch.

The cost of the new Imjudo-Imfinzi regimen, which will be published in a few days, will be “comparable to other approved combination treatments in the liver cancer space,” Ford said.

The novel dose and schedule of the combination, called STRIDE regimen (Single Tremelimumab Regular Interval Durvalumab), includes a single dose of the anti-CTLA-4 antibody Imjudo 300 mg added to the anti-PD-L1 antibody Imfinzi 1500 mg, followed by Imfinzi every four weeks, AstraZeneca said in a news release.

FDA postpones advisory committee meeting for OTC birth control.

The FDA has postponed the joint meeting of the Nonprescription Drugs Advisory Committee and the Obstetrics, Reproductive and the Urologic Drugs Advisory Committee to discuss Perrigo’s application for the nonprescription oral contraceptive. The meeting was originally scheduled for Nov. 18, 2022, to discuss the Opill Rx-to-OTC switch. The date of the new meeting has not yet been determined.

The FDA has postponed the meeting in order to review additional information the agency requested. The agency has also extended the Prescription Drug User Fee Act (PDUFA) date for Opill by 90 days.

Opill is a progestin-only daily birth control pill, which is also referred to as a mini pill or non-estrogen pill. While progestin-only birth control pills can still increase your risk of blood clots and stroke, they’re generally seen as safer for some women, including those who smoke, have high blood pressure or those at risk for blood clots.

FDA grants priority review for novel C. difficile therapeutic.

The FDA has accepted for review Seres Therapeutics’ biologics license application (BLA) for SER-109 for the prevention of recurrent C. difficile infection (rCDI), a hard-to-treat infection. The application has been granted priority review designation with a Prescription Drug User Fee Act (PDUFA) action date of April 26, 2023.

SER-109 is microbiome therapeutic composed of purified Firmicutes spores for the treatment of recurrent C. difficile infection. Firmicutes spores normally live in a healthy microbiome. SER-109 is designed to modulate the disrupted microbiome to a state that resists C. difficile colonization and growth. The SER-109 manufacturing purification process is designed to remove unwanted microbes to the risk of pathogen transmission. The FDA has granted SER-109 breakthrough therapy designation and orphan drug designation for the prevention of recurrent C. difficile infection.

With nearly 170,000 cases of recurrent C. diff each year in the United States, it is one of the top three most urgent bacterial threats in the country, according to the Centers for Disease Control and Prevention, and is a leading cause of hospital-acquired infection. The CDC estimates that there are about half a million infections a year, and one in six patients will have a second infection within two to eight weeks. In those over the age of 65, one in 11 patients with C. difficile will die within one month.

FDA grants priority review for acute myeloid leukemia drug.

The FDA has accepted Daiichi Sankyo’s new drug application (NDA) of quizartinib to treat adult patients with newly diagnosed acute myeloid leukemia (AML) who are FLT3-ITD positive. The application has been granted priority review with a Prescription Drug User Fee Act date (PDUFA) of April 24, 2023. If approved, quizartinib will be used in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation monotherapy following consolidation.

Mutations of the FLT3 gene occur in about 30% of AML patients. FLT3-ITD (internal tandem duplication) is the most common type of FLT3 mutation in AML and is associated with increased risk of relapse and shorter overall survival. The conventional treatment for newly diagnosed AML is intensive chemotherapy with hematopoietic stem-cell transplantation for eligible patients.

FDA accepts NDA for Novaliq’s new dry eye therapy.

The FDA has accepted the new drug application for Novaliq’s CyclASol (cyclosporine ophthalmic solution), to treat the signs and symptoms of dry eye disease (DED). The Prescription Drug User Fee Act (PDUFA) target action date set by the FDA is June 8, 2023.

Novaliq submitted the NDA for CyclASol in August 2022, and the application was based on two pivotal studies in more than 2,000 patients.

CyclASol is water-free, preservative-free solution based on EyeSol technology. Cyclosporine is not water-soluble, but the EyeSol excipient perfluorobutylpentane allows for improved bioavailability and better efficacy on the target tissue. The product contains no oils, no surfactants and is preservative-free due to the novel carrier. This provides additional clinical benefits for patients, such as improved tolerability and decreased visual disturbances.

Dry eye is one of the most common ocular surface disorders, with about 18 million Americans diagnosed with dry eye disease. Inflammation and immunologic processes play a key role in the pathology of the disease.

Genmab submits applications for bispecific therapy for large B-cell lymphoma.

Genmab has submitted a biologics license application (BLA) to the FDA for subcutaneous epcoritamab to treat patients with relapsed/refractory large B-cell lymphoma (LBCL) after two or more lines of systemic therapy. Large B-cell lymphoma (LBCL) is a fast-growing type of non-Hodgkin’s lymphoma (NHL), a cancer that develops in the lymphatic system and affects B-cell lymphocytes, a type of white blood cell. There are an estimated 150,000 new LBCL cases each year globally.

Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology, which is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B-cells and induces T cell mediated killing of CD20+ cells. CD20 is expressed on B-cells and a clinically validated therapeutic target in many B-cell malignancies.

Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies' oncology collaboration. AbbVie has submitted a marketing authorization application (MAA) for epcoritamab for the treatment of patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), which has been validated by the European Medicines Agency (EMA).