28 Jun 2021
Posted in Pharma
Teplizumab’s approval will be paradigm changing for T1D as first disease-modifying therapeutic, says GlobalData
The Food and Drug Administration (FDA)’s Endocrinologic and Metabolic Drugs Advisory Committee voted in favor of Provention Bio’s teplizumab 10-7 to delay the onset of type 1 diabetes (T1D). In the eight major markets (8MM*) the number of diagnosed prevalent cases of T1D will grow at an annual growth rate (AGR) of 1.78%, from 3.3 million cases in 2019 to 3.9 million cases by 2029, according to GlobalData. The leading data and analytics company notes that, as these cases increase, there is still no approved disease-modifying treatment for T1D, and teplizumab will likely not only be the first preventative therapeutic, but the first disease-modifying therapeutic for T1D.
With a PDUFA date of July 2, it is likely that the first preventative treatment for T1D could be available by the end of the year. Provention based its application on results from the pivotal TN-10 study. Results from that study showed a single fourteen-day course of teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least two years in presymptomatic patients with stage 2 T1D compared to placebo. Since the trial’s completion, extension studies have extended that median delay to three years.
Samisha Khangaonkar, Pharma Analyst at GlobalData, comments: “Teplizumab’s protection may last even longer as studies continue. However, as T1D screening is not clinical practice in the US, the trial criteria focused on the subset of T1D patients who have relatives with T1D. According to key opinion leaders (KOLs) interviewed by GlobalData, only 10–15% of T1D patients have a first- or second-degree relative who also has T1D. Unless T1D antibody screening becomes standard of care, the eligible patient population for teplizumab remains quite small.”
Screening panels can be costly, and according to interviewed payers, will unlikely be reimbursed unless preventative screening for T1D is incorporated into the US Task Force of Preventive Services as a recommendation. Even then, however, the reimbursement criteria will likely be limited to the trial patient population, leaving out the 85–90% of newly diagnosed T1D patients that do not have a first or second degree relative with T1D.
Khangaonkar continues: “Many KOLs have expressed uncertainty about teplizumab’s longevity, cost, psychological impacts of delaying T1D, and the long-term toll teplizumab may have. FDA Committee members said that there needed to be more safety data, particularly regarding long-term malignancies and severe adverse events such as the risk of diabetic ketoacidosis and the three deaths documented in the study. The Provention study did not meet its enrollment goal, and thus was assessed in only 44 patients. Given the issues with enrollment and the lack of T1D screening, it will likely be difficult to identify a clear patient population in practice.”
Although the prevention population is small, teplizumab is also being investigated as a treatment at early onset of T1D, as it was shown to significantly reverse the decline of C-peptide levels, which suggests that teplizumab can not only delay the destruction of beta cells, but also restore insulin production in dysfunctional beta cells.
Khangaonkar adds: “Increased screening and early treatments, such as teplizumab, are a first step to drive preventative and patient specific care in T1D. However, increased innovation in the space is a critical need to address the underlying causes of T1D and advance care for all T1D patients, not just those ‘at risk’ of developing T1D.”
*8MM – The US, France, Germany, Italy, Spain, the UK, Japan, and Canada