With a spring approval in hand, Reata Pharmaceuticals has been waiting patiently to launch its first commercial product, Skyclarys. Now, after an impurity problem stalled the rare disease med’s debut, the Plano, Texas-based drugmaker has cleared things up with the FDA well ahead of schedule.
In turn, analysts feel confident the newly commercial-stage Reata can meet—or potentially even exceed—a prospective sales target for 2023.
One and a half months ahead of schedule, the FDA has given the thumbs-up to a prior approval supplement (PAS) to update the drug substance specification for Skyclarys, which became the first and only FDA-approved drug for Friedreich's ataxia back in March.
Friedreich's ataxia is a hereditary neurological disorder that strikes during adolescence and leaves patients wheelchair-bound, often cutting their lives short.
Following the drug’s approval, Reata disclosed a process impurity in May, noting in a press release that FDA clearance of its Skyclarys supplement would be required ahead of the product’s launch. At the time, Reata said it expected to receive an updated regulatory decision sometime around mid-August. Without approval of the supplemental application, Reata’s drug could not be released to patients.
Reata’s PAS—which sought to circumvent the process glitch by increasing Skyclarys’ impurity specification—was accepted in mid-June and approved this week, clearing the way for the rare disease med’s launch. The drug is now available to patients with Friedreich ataxia in the U.S., Reata said late Tuesday.
The green light was to be expected, according to analysts at SVB Securities, who wrote in a note to clients that they figured the manufacturing problem was “likely to be resolved in the near term.”
With that final regulatory hurdle cleared, the SVB Securities team contends Reata’s launch is “worth owning,” with Skyclarys on track to meet or exceed the analysts’ 2023 sales forecast of $45.9 million.
“On the heels of the PAS approval, we expect [Reata] shares to trade higher, and believe the stock can continue to work as the launch gets underway,” SVB Securities added.
Reata has priced Skyclarys at $370,000 annually before discounts. With that cost in mind, Jefferies analyst Maury Raycroft previously estimated U.S. sales potential at $400 million by 2030. Reata has also submitted an application for approval in Europe, with the goal to launch across the pond in early 2024.