- The Food and Drug Administration has approved the Blueprint Medicines and Roche drug Gavreto to treat tumors with abnormalities in a gene known as RET.
- Gavreto, also known as pralsetinib, is now cleared for use in the estimated 1% to 2% of non-small cell lung cancer patients whose tumors are confirmed to have RET fusions through an FDA-approved blood or tissue-based diagnostic test. Until this week, there had been no such test available. But on Monday, Thermo Fisher said the agency had given its Oncomine Dx Target Test a premarket approval to be used as a companion diagnostic to identify patients eligible for Gavreto.
- Roche paid $675 million for rights to Gavreto in July and will now get most of the drug's profits, aside from the U.S., where it will split revenue with Blueprint. The companies said their drug will be available in U.S. pharmacies this week, and come at a monthly list price of $19,250. Gavreto will compete for market share with Eli Lilly's Retevmo, which was approved in May to treat multiple tumor types with RET fusions.
The FDA approved Gavreto two and a half months ahead of its November review deadline. Even so, Blueprint and Roche are playing from behind.
Lilly has enjoyed about four months without competition to Retevmo. Blueprint and Roche will try to make up ground as they launch Gavreto, but the data backing their rival could present an obstacle to carving out market share. Christopher Raymond, an analyst at Piper Sandler who covers Blueprint, noted that Lilly's drug appears to hold an advantage when looking at clinical trial results.
Updated data included in Gavreto's label show that, for RET fusion-positive non-small lung cancer patients who had previously received platinum-based chemotherapy, the overall response rate after taking Blueprint's drug was 57%. For patients who hadn't received prior systemic therapy, the rate was 70%. Those rates were lower than what Bluebird presented at a medical conference this summer.
Conversely, Retevmo's label shows overall response rates of 64% and 85%, respectively, in patients who had received platinum-based chemotherapy and those who had not.
On safety, Raymond wrote it "doesn't seem to bode well" that Gavreto's label includes a warning for interstitial lung disease, or pneumonitis. Pneumonitis occurred in 10% of Gavreto-treated patients, according to the drug's label, with 2.7% them experiencing Grade 3 or 4 adverse events and 0.5% having fatal reactions.
Blueprint does have a few things going in its favor. The recently inked deal between Blueprint and Roche, for example, provides commercialization support from one of the world's biggest cancer drugmakers.
The company will also launch its drug at a slightly lower list price than the $20,600 set for a 30-day supply of Retevmo.
What's more, the approval of Thermo Fisher's companion diagnostic could make the testing process smoother. Without an FDA-approved option, doctors have been diagnosing RET-mutated cancers through local laboratory tests like those typically available at major cancer centers.
Still, it's not clear how well Blueprint's drug can challenge Lilly's.
"We do believe the recent Roche deal for Gavreto will help drive faster commercialization and more efficient patient identification, but despite this, we think the competitive edge for RET inhibitors continues to lie with [Lilly's] Retevmo, with its better efficacy and safety profile," Raymond wrote in a Sept. 7 note to clients.
Blueprint shares were down about 3% Monday morning, to trade at roughly $71 apiece.