FDA Updates for the Week of Jan. 23, 2023

FDA approves Lilly’s BTK inhibitor for rare blood cancer.

The FDA has granted accelerated approval to Lilly’s Jaypirca (pirtobrutinib) to treat adult patients with relapsed or refractory mantle cell lymphoma (MCL), a rare blood cancer and a form of non-Hodgkin lymphoma (NHL). Annually, about one in 200,000 people worldwide develop MCL.

The U.S. wholesale acquisition cost of Jaypirca is $21,000 per 30 days of therapy for the 200 mg dose per day. Jaypirca is expected to be available in the United States in the coming weeks. A Lilly spokesperson said that because oncology is a protected class, they anticipate Medicare Part D plans to add Jaypirca to their formularies within 90 days.

Jaypirca is a non-covalent, highly selective kinase inhibitor and uses a novel binding mechanism to target the specific mutations associated with resistance that patients may develop after treatment with covalent Bruton’s tyrosine kinase (BTK) inhibitors.

The accelerated approval was based on response rate from a single-arm, phase 1/2 study. Jaypirca-treated patients who had been treated previously with a covalent BTK inhibitor achieved an overall response rate of 50%, with 13% of patients achieving a complete response.

FDA approves Keytruda for lung cancer after surgery.

The FDA has approved Merck’s Keytruda (pembrolizumab) to treat patients with non-small cell lung cancer (NSCLC) following surgery and chemotherapy. Lung cancer is the leading cause of cancer death worldwide. Non-small cell lung cancer is the most common type of lung cancer, accounting for about 81% of all cases.

This approval marks the fifth indication for Keytruda-based regimens in non-small cell lung cancer and the 34th indication in the United States.

The approval is based on data from the phase 3 KEYNOTE-091 trial, which enrolled 1,177 patients with completely resected stage IB or IIIA non-small cell lung cancer. In patients who received adjuvant platinum-based chemotherapy following surgical resection, Keytruda reduced the risk of disease recurrence or death by 27% versus placebo regardless of PD-L1 expression. The median disease-free survival in patients was nearly five years (58.7 months) for the Keytruda arm versus nearly three years (34.9 months) for the placebo arm.

FDA approves new drug for type 2 diabetes.

The FDA has approved Brenzavvy (bexagliflozin), an oral sodium-glucose cotransporter 2 (SGLT2) inhibitor. Developed by TheracosBio, Brenzavvy is a SGLT2 inhibitor that is indicated to improve glycemic control in adults with type 2 diabetes. SGLT2 inhibitors are a class of prescription medicines that lower blood sugar by causing the kidneys to remove sugar from the body through urine.

Brenzavvy will be available as 20 mg oral tablets to be taken once daily. The details on its availability and pricing are still in development, a company spokesperson said.

The FDA approval is based on results from a clinical program that evaluated the safety and efficacy in 23 clinical trials enrolling more than 5,000 adults. Phase 3 studies showed that Brenzavvy significantly reduced hemoglobin A1c and fasting blood sugar after 24 weeks, either as a monotherapy and in combination with metformin. In one phase 3 trial that enrolled 426 patients, Brenzavvy was found noninferior to glimepiride, an oral antidiabetic, in lowering hemoglobin A1c and was associated with significantly fewer hypoglycemic events than glimepiride. In another phase 3 trial of 312 patients, bexagliflozin lowered hemoglobin A1c levels by 0.37% compared with placebo.

FDA removes Evusheld’s EUA for COVID-19.

AstraZeneca’s Evusheld is no longer authorized for use in the United States, according to a notice from the FDA. Evusheld had been granted an emergency use authorization (EUA) in December 2021 for COVID-19 pre-exposure prophylaxis. It is a combination of two long-acting antibodies — tixagevimab and cilgavimab — derived from B-cells donated by convalescent patients after SARS-CoV-2 infection.

Data show Evusheld is unlikely to be active against certain SARS-CoV-2 variants, including the omicron subvariants currently circulating in the United States. According to the most recent CDC modeling data, these variants are projected to be responsible for more than 90% of current infections in the United States. This means that Evusheld is not expected to provide protection against developing COVID-19 if exposed to those variants.

In a statement, AstraZeneca said it will continue to work with the FDA to assess and share relevant data regarding Evusheld and SARS-CoV-2 variants. Evusheld currently remains authorized in other countries where it is approved for COVID-19 pre-exposure prophylaxis and treatment, including the EU and Japan.

FDA to hold advisory committee meeting for ALS drug.

FDA plans to hold an advisory committee meeting to review data submitted as part of the new drug application for tofersen, an investigational product to treat patients with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The Peripheral and Central Nervous System Drugs Advisory Committee meeting is scheduled for March 22, 2023.

Developed by Ionis Pharmaceuticals and licensed to Biogen, tofersen is an antisense drug that binds to SODI mRNA. SOD1 was the first gene in which mutations were found to cause the neurodegenerative disease. SOD1-ALS is an ultra-rare form of ALS that affects about 330 people in the United States. It is progressive, leads to the loss of everyday functions and is fatal.

Biogen’s NDA for tofersen was accepted for priority review by the FDA under the accelerated approval pathway and has a Prescription Drug User Fee Act action date of April 25, 2023. Biogen is seeking approval based on the use of a neurofilament as a surrogate biomarker. Neurofilaments are normal proteins found in healthy neurons; they are increased in blood and cerebrospinal fluid when damage has been done to neurons or their axons and are a marker of neurodegeneration. In ALS, higher levels of neurofilaments have been found to predict more rapid decline in clinical function and shortened survival.

FDA accepts Jardiance sNDA for CKD indication.

The FDA has accepted a supplemental new drug application (sNDA) for Jardiance (empagliflozin), which is being investigated as a potential treatment to reduce the risk of kidney disease progression and cardiovascular death in adults with chronic kidney disease (CKD). An FDA decision is in the second half of 2023.

Developed by Boehringer Ingelheim and Eli Lilly, Jardiance is an oral SGLT2 inhibitor used to lower blood sugar in adults with type 2 diabetes, as well as to reduce the risk of cardiovascular death for those with type 2 diabetes and those with cardiovascular disease. The current list price for a month supply of Jardiance is $570.48.

The application is based on results from the landmark EMPA-KIDNEY phase 3 trial, in which Jardiance significantly reduced the risk of kidney disease progression or cardiovascular death in adults with CKD by 28% compared with placebo. The trial showed a significant reduction in risk of hospitalization for any cause, with a 14% relative risk reduction with Jardiance versus placebo in a pre-specified key secondary endpoint.