Vesleteplirsen

After sales stalled for Sarepta's Elivedys, an FDA expansion of its patient population has sparked a new surge as the gene therapy generated $181 million in the third quarter. An FDA expansion of Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) Elevidys has sparked a new surge in sales for the gene therapy.In the third quarter, Sarepta reported revenue from Elevidys at $181 million, routing analysts' consensus estimate of $160 million as it made a 48% sequential leap in sales.It was a significant boost for Elevidys, which got off to a surprisingly quick launch—which is unusual for gene therapies—after its initial approval in June of 2023.In its first two quarters on the market, Elevidys generated sales of $69 million and $131 million in the last two quarters of 2023, respectively. But momentum for the single-use treatment had stalled in the first half of this year with sales reaching $134 million and $122 million separately during the first two quarters.“Elevidys’ performance substantially exceeded our prior guidance,” Sarepta CEO Doug Ingram said in a conference call Wednesday. “We are tracking well into Q4 and 2025 performance consistent with prior guidance.”Dallan Murray, Sarepta's VP of marketing, later added that Elevidys is now “at the inflection point on the launch curve.”Providing the boost was an expanded FDA approval of Elevidys covering all DMD patients age 4 and older. The nod came a year—nearly to the day—after the U.S. regulator initially endorsed Elevidys on an accelerated basis and only for ambulatory boys ages 4 and 5. The expanded nod opened the treatment to non-ambulatory patients. Based on the sales and net price of Elevidys, Leerink Partners figured that Sarepta provided the therapy to 75 patients in the third quarter, which is up from 51 in the previous period, bringing the total number of patients treated to roughly 265. Elevidys is priced at $3.2 million per dose.The growth was driven by “patient demand from both the ambulant and non-ambulant populations, ample site capacity, positive trends in access and reimbursement and consistent [access] conversion rates,” Murray said.Given the momentum, Leerink analysts are projecting Elevidys sales at $331 million in the fourth quarter, which matches up with consensus and company expectations. "Despite the beat and management’s reiteration of guidance, we think Elevidys has significant room for revenue growth in the fourth quarter and into 2025. We look forward to updates on Elevidys’ uptake in ambulatory and non-ambulatory patients in the commercial setting for greater validation of the market opportunity and demand,” Sami Corwin, Ph.D., of William Blair wrote in a note to clients.Along with its third-quarter report, Saretpta also said it was grounding its investigational DMD therapy SRP-5051 after revaluating its risk/benefit profile.