Teplizumab is the first immunotherapy to launch for T1DM and is a landmark drug given its potential ability to preserve beta cell function and delay the need for insulin treatment.

About teplizumab

  1. Provention Bio Inc
  2. Anti-CD3 monoclonal antibody
  3. Daily intravenous administration for 12-14 days to delay progression to clinical T1DM in at-risk people
  4. ~1.8 million cases of T1DM in the G7 markets in 2021

Why is it a drug to watch?

Teplizumab is an Fc receptor–nonbinding anti-CD3 monoclonal antibody that was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) based on results from the phase 2 At-Risk trial in at-risk children and adolescents aged 8 years to 17 years without a diagnosis of T1DM but with a relative with T1DM. In the study, the onset of T1DM was delayed or prevented. The phase 3 PROTECT is assessing the efficacy and safety of teplizumab in children and adolescents aged 8 years to 17 years with a diagnosis in the previous six weeks. Findings from this study will also support the request for additional pharmacokinetic/pharmacodynamic (PK/PD) data in the complete response letter (CRL) from the U.S. FDA in 2021.

  1. Provention Bio has partnered with Sanofi SA to launch teplizumab for T1DM. Sanofi will co-promote the drug and has exclusive right of first negotiation to in-license the drug.

Review and approval status

August 2019:
• Breakthrough Therapy Designation: U.S. FDA

October 2019:
• Priority Medicines (PRIME) designation: European Medicines Agency (EMA)

February 2022:
• Biologics License Application (BLA)

November 17, 2022:
• Approved: U.S. FDA

December 2022:
• U.S. launch

Expected launch:

2024: Europe

Patents estimated to expire beginning in 2026

How will teplizumab impact the market for T1DM?

  1. Mainstay of treatment remains insulin for glycemic control.
  2. Few immunotherapies have made it to late-stage clinical development.
  3. Specialists are optimistic about immunotherapies that can prevent or slow beta cell deterioration, which they feel could shift the treatment paradigm.
  4. If approved when expected, teplizumab will have the advantage of being the first immunotherapy to market.

What gaps in treatment does teplizumab fill?

Insulin is the cornerstone of T1DM treatment to maintain glycemic control and avoid glucose-related complications such as retinopathy, nephropathy and neuropathy that present a significant disease burden. However, many patients with T1DM struggle to maintain recommended glycemic levels due to high treatment costs and complicated disease management (e.g., dose calculations, devices, sensors, pens, multiple daily injections). Disease-modifying drugs such as teplizumab have the potential to prolong disease progression and improve quality of life.

What hurdles might it need to overcome to reach blockbuster status?

Identification of the eligible population might prove to be challenging in practice, given the need for large-scale screening for high-risk individuals especially when testing for early T1DM antibodies is not routinely conducted. Another consideration is that not all individuals with the relevant antibodies progress to T1DM, which might indicate the need for an additional screening stage to determine eligibility. Physicians also view the daily infusion for 12-14 days as burdensome and expect it to be a barrier to uptake.

expected sales in 2027
probability of success for teplizumab in Europe.

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