The response to COVID-19 likely slowed the development of pricey gene therapy drugs, but those products are now making their way back into the approval pipeline, according to a new report from Optum Rx.
The pharmacy benefit manager released its quarterly look at the drug development pipeline. The latest analysis spotlights two gene therapy products headed to the Food and Drug Administration's approval table this fall: elivaldogene autotemcel, under the brand Skysona, and betibeglogene autotemcel, as Zynteglo.
Both therapies are for rare conditions, with Skysona targeting cerebral adrenoleukodystrophy (CALD) in young boys and Zynteglo treating beta thalassemia patients who depend on blood transfusions.
Orphan drugs currently account for nearly half of FDA approvals, and gene therapies like Skysona and Zynteglo carry particularly high price tags. Should they be approved, these drugs would double the number of gene therapies on the market to four, and it's a trend payers should be keeping a close—and early—eye on, said Bill Dreitlein, senior director of pipeline and drug surveillance at Optum Rx, in an interview.
"COVID has had somewhat of an impact, delaying the development of some of those products but now I think we’re starting to see a small wave of those products coming to market," he said.
He said if the wave of gene therapies swells, it could signal a "tipping point" for these drugs.
Both Zynteglo and Skysona are developed by bluebird bio. Analysts forecast that a single dose of Zynteglo could cost $2.1 million, and there are an estimated 3,000 people in the U.S. with beta thalassemia, with about half dependent on regular transfusions.
The report doesn't list a potential price for Skysona but notes that Zolgensma, a gene therapy currently on the market for the ultra-rare condition spinal muscular atrophy, costs $2.125 million per dose. There are about 40 people in the U.S. with CALD annually, according to the report.
And because these drugs treat conditions that are so rare, the data on long-term efficacy is limited, but that information would be another consideration for payers weighing coverage. For both drugs, there is data available from seven years of follow-up.
Insurers and PBMs "only know as much as the data available to us," which makes tracking these products key, Dreitlein said.
"Now is a good time to get back into monitoring the gene therapy pipeline," he added.
The report also highlights two other drugs: teclistamab and deucravacitinib. Teclistamab is an antibody treatment for relapsed or refractory multiple myeloma, while deucravacitinib is an oral drug for psoriasis.
The active nature of the psoriasis market has been noted in previous pipeline reports, and OptumRx states that if approved deucravacitinib would enter a crowded space. It would compete most directly with Otezla, another oral drug, and would likely appeal most to patients with milder psoriasis or those who have not yet advanced to an injectable biologic.
Dreitlein said it's not likely to unseat any of the popular biologics for psoriasis, but deucravacitinib would offer another option for people who prefer an oral therapy to treat psoriasis. An FDA decision is expected on Sept. 10.
"For people who are early in their journey with psoriasis and looking to start therapy, this could be a good option for them before going to a biologic," he said.