Specialty and orphan drugs continue to dominate the development pipeline, and a new report from Optum Rx spotlights three products payers should be watching.
The pharmacy benefit manager recently released its summer Notable New Drugs report, highlighting three products: Brinsupri (brensocatib), tolebrutinib and a subcutaneous version of Leqembi (lecanemab-irmb). All three are specialty drugs that aim to treat chronic conditions—non-cystic fibrosis bronchiectasis, multiple sclerosis and Alzheimer's disease, respectively.
Arash Sadeghi, senior clinical pharmacist on OptumRx's pipeline and drug surveillance team, told Fierce Healthcare that specialty products represent between 70% and 75% of novel drugs approved each year, making it critical for health plans to monitor what's coming to market.
"Any time we talk about the pipeline or drugs that are going to be high impact or blockbusters, so much of that time is devoted to specialty products, specifically," Sadeghi said.
All three of the drugs on this quarter's list represent first-in-class products, and, for health insurers, that can represent new spending, he said. By comparison, drugs that come to market to compete against leading, established therapies may cut down existing spending.
Sadeghi said medicines in this category are of the most interest to clients who are looking to manage potential increases to drug spend.
Insmed's Brinsupri secured an FDA approval in mid-August, marking the first green light of a DPP1 inhibitor. In the report, Optum Rx analysts note that this drug will fill a niche for a condition that has a significant need for new therapies.
Bronchiectasis cannot be cured but can be managed well with effective treatment, according to the report. Sadeghi said that about 500,000 individuals in the U.S. have the lung condition, where bronchi and airways become thickened. The report said the wholesale acquisition price for competing medicines can range between $40,000 and $96,000.
Similarly, tolebrutinib would be the first to treat non-relapsing forms of multiple sclerosis, said Sadeghi. The BTK inhibitor from Sanofi is set to receive an approval decision from the FDA in late September, after being granted breakthrough status in December 2024.
The report noted that the drug has been shown to "significantly slow advancing disability," though patients would likely need monitoring to detect potential liver damage following side effect reports in previous clinical trials.
"There's a lot of drugs approved for MS, but there's no drug specifically approved for this niche of patients with non-relapsing disease," Sadeghi said.
As for subcutaneous Leqembi, it represents a new, easier-to-administer form of an existing drug that could broaden the market. The injectable from Biogen and Eisai secured an FDA approval for autoinjector dosing Aug. 29.
Leqembi has generally been administered in clinics intravenously, which requires coordinating visits for Alzheimer's patients to receive ongoing treatment. A subcutaneous formulation can instead be taken at home.
Sadeghi noted that the new formulation still requires a patient to start with IV administration in clinic before transitioning. He added that while the subcutaneous version may make it easier for people to access Leqembi, there are still significant questions around the efficacy of this class of drugs.
"I think it may increase demand just by virtue of reducing some of those access issues," Sadeghi said. "But that demand, I think, is also going to depend on how the evidence continues to accrue for the class overall, and how comfortable providers are with using these products in the real world, given that sort of benefit-versus-risk and some of those safety concerns."