Dive Brief:

• One year after an initial regulatory rejection, Provention Bio’s injectable drug teplizumab has won Food and Drug Administration approval for preventive treatment of Type 1 diabetes.

• The drug, which will be sold under the name Tzield, is the first treatment cleared by the FDA to delay the onset of Type 1 diabetes. It is approved for individuals over the age of 8 who are considered to be in stage 2 of the autoimmune disease.

• The agency based its approval on data from a study in 2019 that showed patients with stage 2 Type 1 diabetes who received Tzield went longer before receiving a stage 3 diagnosis than those given placebo.

Dive Insight:

Originally developed by MacroGenics, Tzield is an antibody that targets a protein known as CD3 in order to regulate the immune system. The drug has been in testing for years, passing from MacroGenics’ hands to Eli Lilly in 2007, then back to MacroGenics again after a Phase 3 study failure in 2010.

In 2018, the New Jersey biotechnology company Provention acquired rights to the drug and has been faithfully betting on the drug’s promise to prevent the full onset of Type 1 diabetes.

Following the FDA’s rejection of the drug last year, Provention worked to gather the necessary information to meet the agency’s concern. The rejection was in part tied to a “bridging study” looking at the effects of the drug on the body, as well as certain deficiencies found in the manufacturing facilities.

Anticipating an approval of its experimental drug, Provention in October entered a marketing agreement with Sanofi. Under the deal, Sanofi paid Provention $20 million for rights to negotiate a potential license of teplizumab, as well as to secure a co-promotion contract. Upon approval of the drug, Sanofi agreed to make a $35 million equity investment in Provention.

As of Friday morning, Provention’s shares are trading about $8 a share, up 25% compared to one year ago but slightly down compared to last week.

Teplizumab will be sold at a price of $13,850 a vial, with a 14-vial regimen costing $193,900 at list price. Provention has launched a patient support program called Compass to help answer patient questions regarding coverage and to help patients get access to the drug.

The price is higher than some analysts had expected, according to Cantor Fitzgerald’s Prakhar Agrawal.