Sarepta Therapeutics Halts Elevidys Shipments for Non-Ambulatory Dmd Patients After Second Death
Sarepta Therapeutics has temporarily suspended shipments of Elevidys (delandistrogene moxeparvovec-rokl) for infusion in non-ambulatory patients. This action follows teh second reported death in three months of a patient with Duchenne Muscular Dystrophy (Dmd) after receiving the gene therapy.
The company is initiating an enhanced immunosuppressive regimen evaluation to ensure patient safety after the recent Elevidys treatment complications.
Patient Death Leads To Shipment Suspension
the affected patient, described as a non-ambulatory individual with Dmd, experienced acute liver failure (Alf). Sarepta reported this as the second such case following Elevidys treatment, the only gene therapy approved by the Fda for Dmd.
The first patient, a 16-year-old male, died in march. Following that initial incident,Sarepta committed to updating the prescribing details for Elevidys.
“we are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient’s family and his care team during this incredibly difficult time,” said Louise Rodino-Klapac, Phd, Sarepta’s Chief Scientific Officer and Head of Research & Progress.
Enhanced Safety Measures And Trial Adjustments
Sarepta’s temporary shipment suspension specifically affects non-ambulatory patients.The pause will remain in effect while the company evaluates and discusses an enhanced immunosuppressive regimen with regulatory bodies before implementing it.
There will be no immediate changes for ambulatory patients. The existing protocol of administering corticosteroids before and after Elevidys infusion, along with post-treatment monitoring, will continue.
Pro Tip: Always consult with a qualified healthcare professional for personalized medical guidance.This article is for informational purposes only and dose not constitute medical advice.
Independent Expert Panel To Review Immunosuppression
Sarepta plans to immediately assemble an independent panel of leading experts in Duchenne and liver health. The purpose is to evaluate and refine the immunosuppression strategy for Elevidys.
The panel will analyze data and assess a proposed regimen involving sirolimus, supported by preclinical data indicating the effectiveness of additional immunosuppression in managing liver enzyme elevations. Sarepta has pledged to share the panel’s findings with the Fda for further guidance and approval.
Phase Iii Envision Trial Paused
Sarepta has also voluntarily paused dosing in its phase iii Envision trial (Srp-9001-303, Nct05881408). This pause allows for the evaluation of a protocol amendment.The amendment would incorporate the enhanced immunosuppressive regimen for the non-ambulatory patient cohort, pending Fda feedback.
The Fda has agreed with the voluntary pause and must approve the protocol amendment before screening and dosing can resume in the Envision trial.
Did You No? Duchenne Muscular Dystrophy primarily affects males, with symptoms typically appearing between the ages of 2 and 5.
Fda Approval History Of Elevidys
The Fda’s former director of its Center for Biologics Evaluation and Research (Cber), Peter Marks, Md, overruled staff reviewers twice to grant elevidys an initial accelerated approval for ambulatory boys ages 4 to 5 in 2023. This was followed by an expanded approval last year for all Dmd patients with a confirmed mutation in the Dmd gene who are ages 4 and older, including accelerated approval for non-ambulatory patients.
The full approval came even after Elevidys missed its primary endpoint of statistically significant improvement vs.placebo on the North Star Ambulatory Assessment (Nsaa) total score 52 weeks after treatment (2.6 points Elevidys vs. 1.9 points placebo). The Fda accepted Sarepta’s argument that Elevidys merited a broader label due to “robust evidence for a clinically meaningful treatment benefit” across age groups in key pre-specified functional secondary endpoints.
However,the Fda made continued approval of Elevidys for non-ambulatory Duchenne patients contingent upon verification of clinical benefit in a confirmatory trial,which the Envision trial is designed to provide.
Marks resigned abruptly in march following a policy dispute. He accused U.s. Health and Human Services Secretary Robert F. Kennedy Jr. of seeking confirmation of misinformation on vaccine safety. vinay Prasad, Md, has since succeeded Marks as Cber director.
elevidys Market Performance
Elevidys generated $375 million in net product revenue during the first quarter, reflecting an increase of 180% year over year. In the previous year, net product revenue totaled $820.8 million. Roche holds the rights to Elevidys outside the United States and reported $4 million in royalty revenue during Q1 and $16.8 million in 2024 on sales of the gene therapy.
Key Events Summary
| Date | Event | Impact |
|---|---|---|
| March 2024 | First Patient Death | Sarepta announces an update to Elevidys prescribing information. |
| June 2024 | Second Patient Death | Sarepta halts Elevidys shipments for non-ambulatory patients, initiating enhanced immunosuppression evaluation. |
| Ongoing | Envision Trial | Phase Iii trial paused to incorporate new immunosuppressive regimen amendment. |
Understanding Duchenne Muscular Dystrophy (dmd)
Duchenne Muscular Dystrophy (Dmd) is a genetic disorder characterized by progressive muscle degeneration and weakness due to alterations or absence of the protein dystrophin. It primarily affects males, with symptoms usually appearing in early childhood.
While there is no cure for Dmd, treatments such as gene therapies like Elevidys aim to slow disease progression and improve quality of life. Ongoing research and clinical trials offer hope for further advancements in managing this condition. Support groups and advocacy organizations play a crucial role in providing resources and community for individuals and families affected by Dmd.
Frequently Asked Questions About Elevidys and Dmd
- Why did Sarepta halt Elevidys shipments?
Sarepta halted Elevidys shipments to non-ambulatory patients after a second death linked to acute liver failure, prompting a safety review.
- What is Elevidys used to treat?
Elevidys is a gene therapy used to treat Duchenne Muscular Dystrophy (Dmd) in patients with a confirmed mutation in the Dmd gene.
- Are there any changes for ambulatory patients receiving Elevidys?
No treatment changes are being implemented for ambulatory patients. The current practice of administering corticosteroids before and after Elevidys infusion remains the same.
- What is the role of the Fda in the Elevidys situation?
The Fda granted accelerated and expanded approval for Elevidys and must approve the protocol amendment for the Envision trial before screening and dosing can resume.
- what is Duchenne Muscular Dystrophy (Dmd)?
Dmd is a genetic disorder causing progressive muscle degeneration and weakness, primarily affecting males.
What are your thoughts on sarepta’s decision? How do you think this situation will impact the future of gene therapy treatments for Dmd?
Disclaimer: This article provides information about a developing situation and should not be considered medical advice.Consult with healthcare professionals for personalized guidance.