Sarepta surges as investors cheer expanded use of gene therapy

June 21 – Sarepta Therapeutics shares surged as much as 40% to a more than three-year high on Friday as an expanded use approval cemented its dominant position in the gene therapy market for Duchenne muscular dystrophy .

At current levels of $163.88, stock was set to add nearly $4 billion to the company’s market value if gains hold.

On Thursday, the U.S. FDA granted traditional approval for the therapy, Elevidys, in patients four years and above who can walk, as well as an accelerated approval for those who cannot.

The health regulator’s expanded approval was based on the therapy meeting its secondary goals of improvement in factors like time taken by patients to rise from the floor and a 10-meter walk or run, even though it had failed to meet its primary goal of improving motor functions as assessed by a scoring system.

The FDA decision was the best case scenario predicted by analysts, with BMO Capital Market’s Kostas Biliouris estimating that Elevidys could be given to about 13,000 patients, or nearly 90% of U.S. patients.

Given the high demand and acceptable safety, analysts flagged manufacturing concerns and insurance coverage for newly added patients as limiting factors.

The company is working on a new so-called suspension process for commercial production of Elevidys, which could be more beneficial as the therapy moves to a broader outside U.S. population, CEO Doug Ingram said in a conference call with analysts and investors.

“We’ve already done engineering runs up to 2,000 liters… and it will give us not only more materials down the road years from now, but also significantly reduce the cost of goods,” Ingram added.

The company uses Catalent and Thermo Fisher Scientific as contract manufacturers for its gene therapy.

Elevidys is among the most expensive treatments in the world with a list price of $3.2 million, and Ingram said on Friday the company does not intend to modify the price.

Sales of the therapy came in at $200.4 million in 2023. BMO’s Biliouris said those sales showed that Sarepta had “done an amazing job”.

“Commercially, they have been perhaps more successful than any other gene therapy, with the only exception being Zolgensma from Novartis,” Biliouris said in an interview ahead of the approval.

Biliouris now expects Elevidys to be the “dominant” DMD gene therapy with no near-term competition until later than 2027.

DMD is an inherited progressive muscle-wasting disorder that affects an estimated one-in-3,500 male births worldwide, according to the U.S.-based National Organization for Rare Disorders.

The decision also follows concerns around an “uphill battle” to secure traditional approval after the therapy failed to meet the main goal in a key confirmatory late-stage trial.

The global treatment market for DMD – including gene therapies and other drugs – is expected to grow to $11.47 billion by 2034, according to ResearchandMarkets.com.

RegenxBio is testing a rival gene therapy in early- to mid-stage, while Pfizer’s treatment recently failed in a late-stage study.

This article was generated from an automated news agency feed without modifications to text.

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Published: 21 Jun 2024, 11:01 PM IST