Sarepta has suffered another regulatory setback after the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys (delandistrogene moxeparvovec), in Europe.
In the latest of a string of issues Sarepta has faced, the CHMP said it was recommending against approval as the pivotal EMBARK trial (NCT05096221) of the drug failed to show an effect on movement abilities after 12 months.
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Sarepta said it has accepted the CHMP’s decision on Elevidys, with Dr Louise Rodino-Klapac, president of R&D and technical operations, saying: “While we are disappointed by the CHMP’s negative opinion, we understand the urgent need for continued dialogue and collaboration to bring transformative therapies to people with DMD who live with a relentless disease that steals their mobility, independence and ultimately life – often by early adulthood.”
While this news will come as a blow to Sarepta, the licence for commercialising the therapy lies with Roche outside the US. The Swiss company said it plans to work with the EMA to explore a potential path forward.
Roche’s chief medical officer and head of global product development Dr Levi Garraway said: “We are disappointed by the CHMP’s negative opinion, given the urgent need for disease-modifying therapies for children in the EU living with Duchenne.
“With an average life expectancy of only 28 years, achieving disease stabilisation is a major advance for individuals living with DMD, their families and caregivers. We are confident in the value Elevidys can bring to ambulatory patients.”
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By GlobalDataWhile the primary endpoint in the EMBARK trial was not met, secondary endpoints, which include functional outcomes, were both clinically and statistically significant.
The CHMP said that while many patients treated with Elevidys were shown to produce a shorter form of the dystrophin protein, “the levels of dystrophin could not be linked to an improvement in movement abilities”.
Sarepta recently agreed to pause Elevidys shipments to the US after a request from the US Food and Drug Administration (FDA).
This came after the company confirmed a third gene therapy patient death, the first two in patients dosed with Elevydis, and the third in a 51-year-old non-ambulant limb-girdle muscular dystrophy (LGMD) patient dosed with an investigational gene therapy SRP-9004. Both Elevidys and SRP-9004 are adeno-associated virus (AAV) gene therapies that use the same AAVrh74 serotype.
Speaking after the third patient death, GlobalData healthcare analyst Momna Ali said the recent setbacks for Sarepta put the cell and gene therapy (CGT) sector at a “crossroads”.
Ali said: “While the scientific potential of CGT therapies remains extraordinary, this moment serves as a reminder of the complexity, cost, and responsibility involved. There is a lot of buzz around therapies beyond the pill; however, for the landscape to keep evolving at the pace it has been in the last 3-5 years, there must be greater transparency, patient safety, and sustainable innovation – otherwise, it’ll be met with more setbacks.”
According to GlobalData, the CGT market was estimated to be $5.88bn in 2023 and is projected to reach $80bn by 2029.
GlobalData is the parent company of Clinical Trials Arena.
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