Injection buys valuable time to formulate care game plans in diabetes management.
Type 1 diabetes (T1D) is a chronic autoimmune disease that reportedly affects about 1.6 million Americans. Lighting the way with the potential to change the practice of medicine for people with T1D is Provention Bio, a Sanofi company, and the FDA approval of its drug, Tzield (teplizumab-mzwv). Cleared in November 2022, the injection is approved to delay the onset of stage 3 T1D in adults and pediatric patients eight years and older who have stage 2 T1D.
Tzield is a CD3-directed antibody that binds to and may deactivate pancreatic beta cell auto-reactive T-lymphocytes and is administered by intravenous infusion once daily for 14 consecutive days.
According to FDA’s news release of the approval, “Tzield is the first disease-modifying therapy for T1D…[The] treatment can delay the need for exogenous insulin therapy and its associated risks and intensive regimen. This delay is clinically meaningful, particularly because T1D often presents in patients younger than 10 years who may face challenges with complex disease management.”
Most T1D patients are diagnosed after they have already hit stage 3, which is when they have likely lost such a significant portion of insulin-producing cells that they’ll need insulin for the rest of their lives.
“We believe the impact is profound,” Olivier Bogillot, head of US general medicines, Sanofi, tells Pharmaceutical Executive®. “Delaying the onset of stage 3 T1D gives people time to educate themselves, prepare for T1D management, and develop a care plan with their healthcare providers—without the challenges that come with stage 3 T1D.”
A 14-vial Tzield regimen has a current list price of $193,900, and an individual vial has a list price of $13,850.
“These are gross prices and certain segments of the market are eligible for rebates and discounts, such as Medicaid,” explains Bogillot. “Our price was determined after consideration of a number of factors, including pricing market research.”
Provention Bio works with COMPASS, a patient support program with a staff of dedicated personnel available to answer questions and help navigate coverage, reimbursement, and access for patients that are prescribed Tzield. Additionally, they offer financial assistance options to eligible patients for out-of-pocket costs.
During an assessment soon after approval, Seeking Alpha called Tzield a “potential blockbuster,” implying a $6 billion market opportunity, with further growth possible if patient populations expand.1
Tzield’s safety and efficacy were evaluated in a randomized, double-blind, event-driven, placebo-controlled trial with patients who have stage 2 T1D. Participants received Tzield or a placebo once daily via intravenous infusion for 14 days and were then followed until they were diagnosed with stage 3 T1D. Findings show the Tzield median follow-up time was 51 months or just over four years, as compared to 25 months or just over two years for the placebo group. Those not diagnosed with stage 3 T1D were followed for different periods of time.
“T1D can precipitate DKA (diabetic ketoacidosis), a serious complication that requires immediate attention,” says Bogillot. “T1D screening has been shown to lower rates of DKA.” The condition, in which the body produces excess blood acids (ketones), can be triggered by infection or other illness.
The safety of Tzield was studied in five clinical trials with more than 1,000 participants, not all diagnosed with stage 2 T1D. Side effects were monitored in 773 patients who were given the drug and 245 patients who were administered the placebo. The most common side effects include rash, leukopenia (decrease in white blood cell counts), and headache. The use of Tzield comes with warnings and precautions, including premedicating and monitoring for symptoms of cytokine release syndrome; risk of serious infections; decreased levels of a type of white blood cell called lymphocytes; risk of hypersensitivity reactions; the need to administer all age-appropriate vaccinations prior to starting Tzield; and avoiding concurrent use of live, inactivated, and mRNA vaccines with Tzield.
Future development plans for Tzield are already coming down the pipe. According to Provention Bio, “The topline results of the PROTECT Phase III clinical trial showed a positive result on the primary endpoint and will be presented at a major congress later this year.”
1. Ingham, E. Provention Bio: Assessing the Investment Case After Teplizumab Approval. Seeking Alpha. November 25, 2022. https://seekingalpha.com/article/4560410-provention-bio-stock-assessing-investment-case-after-fda-teplizumab-approval