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Sarepta + Roche

Sarepta Therapeutics and Roche: First Gene Therapy to Receive FDA Approval

June 23, 2023

The Food and Drug Administration (FDA) in the United States has approved ELEVIDYS (delandistrogene moxeparvovec-rokl, also known as SRP-9001) for the treatment of ambulatory patients 4-5 years old with Duchenne muscular dystrophy (DMD). This micro-dystrophin adeno-associated viral vector (AAV) gene therapy is developed by Sarepta and Roche.

ELEVIDYS is the first approved gene therapy for Duchenne, marking a significant milestone that encourages a path toward identifying treatments for all those living with this rare genetic disease. 

 “The approval of Elevidys is a watershed moment for the treatment of Duchenne. Elevidys is the first and only gene therapy approved for Duchenne and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease.”

Doug Ingram, President and CEO of Sarepta

Defeat Duchenne Canada has funded over 17 million in Duchenne research, including Dr. Jerry Mendell’s work at Nationwide Children’s Hospital’s Research Institute, which led to the development of ELEVIDYS. We congratulate Dr. Mendell on this milestone in his tireless, lifelong dedication to Duchenne science.

What does this mean for Canada?

For individuals residing outside the United States, Roche is actively engaging with health authorities in various countries to facilitate the approval and availability of delandistrogene moxeparvovec-rokl:

“Following this announcement, we appreciate that many families, caregivers and people living with DMD will be seeking to understand if and when this medicine is likely to receive approval in Canada.

Delandistrogene moxeparvovec is currently not authorized for sale in Canada. If the EMBARK data are positive, Roche Canada intends to file a new drug submission to Health Canada for regulatory approval of delandistrogene moxeparvovec. While today marks an important milestone for the DMD community, we are keenly aware that regulatory approval is only the first step towards people being able to access medicines. Further assessments of clinical- and cost-effectiveness by national Health Technology Assessment bodies (i.e. CADTH and INESSS) must also be undertaken. Roche will then work with payers towards sustainable access to delandistrogene moxeparvovec across the country.

The FDA approval is the first of what we hope will be many more encouraging updates to the Duchenne community as delandistrogene moxeparvovec continues its journey to reach those who need it. Our sincere gratitude and appreciation goes out to the many patients and their families who are participating in DMD research.”  

Roche Canada

Questions and Answers

How do Roche and Sarepta work together?

  • As part of the 2019 license and collaboration agreement, Roche and Sarepta work together in a partnership and joint development programme with the objective of making delandistrogene moxeparvovec available globally.1
  • Sarepta is responsible for organizing and conducting clinical studies as well as managing regulatory approval and the commercialisation of delandistrogene moxeparvovec in the US.
  • Roche is responsible for regulatory approval, Health Technology Assessment appraisal and bringing delandistrogene moxeparvovec to patients across the rest of the world.
  • Sarepta is responsible for the manufacturing of delandistrogene moxeparvovec and will supply the product to Roche for their distribution.

What does the FDA approval mean?

  • The FDA has provided regulatory approval for delandistrogene moxeparvovec to be marketed in the US, under the agency’s accelerated approval pathway for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.1

What is the safety profile of delandistrogene moxeparvovec?

To date, the safety profile of delandistrogene moxeparvovec is consistent across three Phase 1 and 2 clinical trials: Study 101 (NCT03375164), Study 102 (NCT03769116) and Study 103 (also known as ENDEAVOR, NCT04626674 ). Across these three clinical trials:9

  • There were no deaths.
  • No adverse events (AEs) led to study discontinuation.
  • 7 people (8.3%) experienced treatment-related SAEs:
  • Vomiting (N=2); increased liver enzymes (transaminases) (N=2); breakdown of muscle tissue (rhabdomyolysis) (N=2); liver injury (N=1); immune-mediated myositis (N=1); heart inflammation (myocarditis) (N=1).
  • The most frequently observed treatment-emergent AE was vomiting and nausea.
  • No clinically relevant complement activation was observed.
  • There have been no signs of liver failure reported across the delandistrogene moxeparvovec clinical studies.

When are you planning to apply for regulatory approval in Canada?

  • Delandistrogene moxeparvovec is currently not authorized for sale in Canada.
  • If the results of the ongoing Phase 3 EMBARK study (Study 301, NCT05096221) are positive, we intend to file a new drug submission to Health Canada for regulatory approval of delandistrogene moxeparvovec.

Do you expect delandistrogene moxeparvovec to be approved for the same indication/label outside the US?

  • Different health authorities act independently, following distinct processes with different filing requirements. Although their ultimate goals are the same for their geographical regions, they have distinct regulations and procedures, meaning there are differences in how medicines are approved.
  • Whether these health authorities will impose the same or a more restrictive label is yet to be determined. Roche is engaging with health authorities to discuss the registration pathway for delandistrogene moxeparvovec. Final labelling will depend on the health authorities’ decisions for each country.

In countries where delandistrogene moxeparvovec is not yet approved, will it be possible for patients outside of the US to access it?

  • In a select number of countries, physicians are able to request access to a treatment for a charge on an individual named patient basis, ahead of regulatory approval in their country.
  • It is not a Roche or Sarepta-led programme, and local regulations must be followed when accessing a medicine in this way.
  • Roche recognizes that there is a significant unmet medical need for those living with DMD, and we believe in helping to deliver innovations to the patient community. It is our intention to have and foster dialogue with key decision-makers to advocate for access for eligible DMD patients.

How many patients have been treated with delandistrogene moxeparvovec so far?

  • More than 150 people have been treated with delandistrogene moxeparvovec in clinical trials to date.

Which delandistrogene moxeparvovec clinical trials are currently open for recruitment?

  • ENVISION (also known as Study 303, (NCT05881408) is a global Phase 3 clinical study that aims to evaluate the safety and efficacy of delandistrogene moxeparvovec, in non-ambulatory (those who are not able to walk unassisted) and older ambulatory (those who are able to walk unassisted) boys. Roche will be sharing further information on the study timelines and locations outside of the US after the summer.

When can we expect to hear the price of delandistrogene moxeparvovec? Will it be the same in all countries?

  • Roche is responsible for bringing delandistrogene moxeparvovec to patients outside of the US. Roche is not involved in setting the US price.
  • We are committed to appropriately pricing delandistrogene moxeparvovec in a sustainable way, reflecting the value it brings to patients, their families and health systems and society.
  • At this time, there is no set price for delandistrogene moxeparvovec in Canada.

Why did the FDA approve the use of delandistrogene moxeparvovec to ambulatory patients aged 4–5 years?

  • The BLA for delandistrogene moxeparvovec included safety and efficacy data from Study 102. In this phase 2, double-blind placebo-controlled Phase 2 study, a statistically significant part of the data package that was submitted to the FDA by Sarepta.
  • In this study, a statistically significant increase in functional benefit was seen in the 4–5 year-old age group treated with delandistrogene moxeparvovec compared to placebo. Results from the ongoing Phase 3 EMBARK study (NCT05096221) are due towards the end of 2023. The FDA may consider an expansion of the delandistrogene moxeparvovec label based upon the review of the Phase 3 EMBARK data.7

*Propensity-score-weighted external control is a statistical method used to estimate the effectiveness of a medical treatment or intervention when a large enough placebo controlled group is not feasible, using real-world data as an external control.

**The FDA Accelerated Approval programme allows treatments for serious conditions that fill an unmet medical need to be approved based on a surrogate endpoint, or biomarker data that are likely to be predictive of clinical benefit. Pharmaceutical companies are still required to conduct studies to confirm the anticipated clinical benefit. If the confirmatory trial shows that the treatment actually provides a clinical benefit, then the FDA grants traditional approval.

References

  1. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy. Last accessed: June 2023.
  2. Duan D, et al. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021;7(1):13.
  3. Sarepta Therapeutics. Available at: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-us-fda-has-accepted-filing-and. Last accessed: May 2023.
  4. ClinicalTrials.gov. NCT03375164: A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD). Available at: https://clinicaltrials.gov/ct2/show/NCT03375164. Last accessed: May 2023
  5. ClinicalTrials.gov. NCT03769116: A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 (Delandistrogene Moxeparvovec) for Duchenne Muscular Dystrophy (DMD). Available at: https://clinicaltrials.gov/ct2/show/NCT03769116. Last accessed: May 2023.
  6. ClinicalTrials.gov. NCT04626674: A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) (ENDEAVOR). Available at: https://clinicaltrials.gov/ct2/show/NCT04626674. Last accessed: May 2023.
  7. ClinicalTrials.gov. NCT05096221: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK). Available at: https://clinicaltrials.gov/ct2/show/NCT05096221. Last accessed: May 2023.
  8. Sarepta Therapeutics. Available at: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-partnership-roche-territories. Last accessed: May 2023.
  9. Zaidman C, et al. Integrated analyses of data from clinical trials of delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD). Presented at ICNMD 2022.