FDA Updates for the Week of Jan 30, 2023

FDA approves Trodelvy for HR+/HER2- breast cancer.

The FDA has approved Gilead Sciences’ Trodelvy (sacituzumab govitecan-hziy) to treat patients with locally advanced or metastatic breast cancer. It is indicated for patients who are HR+/HER2-, the most common type of breast cancer. It accounts for about 70% of all new cases. Five-year relative survival rate for these patients is 30%.

Trodelvy has also been recommended as a preferred treatment for metastatic HR+/HER2- breast cancer by the National Comprehensive Cancer Network.

The approval is based on progression-free survival and overall survival data from the phase 3 TROPiCS-02 study. Trodelvy demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit of 3.2 months compared with the treatment of physician’s choice. Trodelvy also demonstrated a 34% reduction in risk of disease progression or death. Three times as many people treated with Trodelvy were progression free at one year versus those treated with chemotherapy.

The safety profile for Trodelvy was consistent with prior studies, with no new safety signals identified in this patient population.

FDA approves novel therapy for CKD-related anemia.

The FDA has approved Jesduvroq (daprodustat) for the once-a-day treatment of anemia due to chronic kidney disease (CKD) in adults on dialysis. Chronic kidney disease is characterized by progressive loss of kidney function and is a risk factor for cardiovascular disease. Anemia is a frequent complication of CKD and affects about 700 million patients worldwide, with an estimated one in seven patients also developing anemia. In the United States, about 810,000 patients have end-stage renal disease, of which 558,000 patients receive dialysis.

Developed by GSK, Jesduvroq is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI). It works to increase production of erythropoietin (EPO), which stimulates production of hemoglobin and red blood cells.

A spokesperson for GSK said they are working to make Jesduvroq available as soon as possible. A wholesale acquisition cost has not yet been set for Jesduvroq. Because 87% of dialysis patients are on Medicare, GSK is in the process of submitting the required documentation with Centers for Medicare & Medicaid Services (CMS) to help ensure timely reimbursement and patient access to Jesduvroq. This is expected in the fourth quarter 2023/first quarter 2024 time frame.

The approval is based on the results of the ASCEND-D trial, which were published in the New England Journal of Medicine in December 2021. The study compared Jesduvroq with Aranesp (darbepoetin alfa), and Jesduvroq demonstrated noninferiority based on mean change in hemoglobin. The label includes a boxed warning about the increased risk of death, myocardial infarction, stroke, venous thromboembolism, and thrombosis of vascular access. Jesduvroq increases the risk of thrombotic vascular events, including major adverse cardiovascular events (MACE).

FDA approves Orserdu to treat metastatic breast cancer.

The FDA has approved Orserdu (elacestrant) to treat postmenopausal women or adult men with advanced or metastatic breast cancer. It is indicated for patients with ER-positive, HER2-negative, ESR1-mutated disease with disease progression following at least one line of endocrine therapy.

Developed by Stemline Therapeutics (a subsidiary of Menarini Group), Orserdu is an oral selective estrogen receptor degrader (SERD). SERDs work by blocking the effects of estrogen on hormone receptor-positive breast cancer cells. ESR1 mutations are present in up to 40% of ER+, HER2- advanced or metastatic breast cancers.

Approval was based on data from the phase 3 EMERALD study, a multicenter trial that enrolled 478 postmenopausal women and men with ER-positive, HER2-negative advanced or metastatic breast cancer of which 228 patients had ESR1 mutations. The study compared Orserdu with AstraZeneca’s Faslodex (fulvestrant), the only other FDA-approved SERD, plus letrozole, anastrozole, exemestane.

In the 228 (48%) patients with ESR1 mutations, median progression-free survival was 3.8 months in the Orserdu arm and 1.9 months in the Faslodex arm.

FDA approves Tezspire in pre-filled pen.

The FDA has approved Tezspire (tezepelumab-ekko) in a pre-filled, single-use pen for patients aged 12 years and older with severe asthma. First approved by the FDA in December 2021, Tezspire is a first-in-class biologic for severe asthma. It targets thymic stromal lymphopoietin (TSLP), which plays a role in the inflammation associated with asthma.

Developed by Amgen and AstraZeneca, the original Tezspire delivered via a single-use syringe has a list price of $3,847.35 per 210 mg/mL, which is given every four week. A spokesperson for the companies indicated that the pricing of the pre-filled will be made available when it launches.

The approval was based on results from the PATHFINDER clinical trial program, which included results from the PATH-BRIDGE phase 1 trial and the PATH-HOME trial phase 3 trial. The majority (92%) of healthcare providers, patients and caregivers were able to successfully administer Tezspire both in the clinic and at home.