- Sarepta Therapeutics has dosed the first commercial patient with its newly approved gene therapy for Duchenne muscular dystrophy and expects a “handful” more to follow over the next week and a half, executives said on an earnings call Wednesday.
- The therapy, Elevidys, was cleared in June for use in certain 4- and 5-year-old Duchenne patients and given a $3.2 million price tag. UnitedHealthcare issued a coverage policy on Monday and others should be in place in the next three to six months. In the meantime, Sarepta’s working with insurers on a “case by case basis,” said Chief Customer Officer Dallan Murray.
- Early signs from the launch suggest “more rapid uptake than expected,” according to RBC Capital Markets analyst Brian Abrahams. Sarepta told analysts it believes the treatment could eventually be available to up to 95% of Duchenne patients, including those who can no longer walk. But they cautioned that sales will take time to build, and access will hinge on the outcome of a confirmatory trial expected to read out in the fourth quarter.
U.S. regulators have approved eight gene therapies for inherited diseases. A majority of those clearances have come since the start of 2022, a trend that’s expected to accelerate in the years to come.
But selling the complex and expensive treatments remains a challenge. While Novartis has had some success with the spinal muscular atrophy drug Zolgensma, it’s currently an outlier. Launches are slow to accelerate as companies haggle with insurers over seven-figure price tags. Some therapies require human cells to be extracted and modified in a lab, a process that can take weeks or months.
Even those administered with simple infusions can take time to gain traction: It took more than six months for the first U.S. commercial patient to receive CSL and UniQure’s hemophilia gene therapy Hemgenix.
Sarepta has other obstacles with Elevidys. Its therapy hasn’t yet proven in testing that it can improve Duchenne patients’ function. Patients have to be screened for antibodies that can mute Elevidys’ effects, and monitored for years after treatment. Additionally, there’s only a two-year window in which patients are currently eligible. The first to receive therapy did so the day before his sixth birthday, which required “an enormous amount of work,” CEO Doug Ingram told analysts.
Sarepta’s ongoing trial also complicates matters. The Food and Drug Administration has said it will loosen Elevidys’ age restrictions if the study succeeds, and pull the drug from the market if it doesn’t.
Pfizer could quickly take market share, too, as initial Phase 3 results from a similar gene therapy are now expected later this year.
Those uncertainties have led Sarepta to shy away from forecasting revenue for Elevidys in the early stages of its launch. Instead, Murray pointed to positive signs, like the activation of more than 50 treatment sites, the “handful” of additional infusions coming and a “strong demand” for therapy.
“We expect this to take some time in the early stages before dosing can begin, in earnest, to generate the launch ramp we are confident will come,” he said.
The dosing of an initial patient already surprised some Wall Street analysts. Leerink Partners’ Joseph Schwartz hiked his projections for the 2023 fiscal year to $122 million from $110 million previously. Abrahams, of RBC, is now estimating $114 million in 2023 and more than $4 billion annually at peak.
“Although we still expect the initial launch to be gated by payer processing, sites, and manufacturing,” Abrahams wrote, “early data suggests potential for the system to move more quickly than we and [Wall] Street had expected.”
Sarepta shares climbed about 3%, to around $108 apiece, in Thursday trading.