Latest news with #ALF
Yahoo
2 days ago
- Health
- Yahoo
Sarepta refuses to pull gene therapy despite FDA order
Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the US Food and Drug Administration (FDA). Speaking on 18 July, shortly after the FDA ordered the pull for Elevidys, Sarepta said it will continue to ship Elevidys to the ambulant population as studies show no signs of new or changed safety signals, adding it first heard of this potential request earlier in the day through media reports. A statement from the company reads: 'At Sarepta, patient safety and well-being are always our top priority. We are committed to upholding the highest safety standards for all of our therapies. "This guides every decision we make, as evidenced by our conservative decision to pause shipments of Elevidys for non-ambulant patients while we work with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure (ALF).' Trial of LGMD therapy also put on hold The FDA has also put a hold on Sarepta's gene therapy SRP-9004 in limb girdle muscular dystrophy (LGMD) after a patient died in an early-stage study due to ALF. Speaking about the patient death in the Phase I trial of SRP-9004, the company's statement added: 'We recognise that the death of any patient is heartbreaking, including the recent death of a 51-year-old non-ambulant LGMD patient. "We also want to clarify that this tragic event occurred in a Phase I clinical trial for an investigational gene therapy called SRP-9004. SRP-9004 is a clinical-stage therapy that is intended to treat a different disease, is administered using a different dose, and is manufactured using a different process. The LGMD study participant who passed away was not treated with Elevidys, and the dosing for the SRP-9004 trial had concluded at the time of his death.' This patient's death, which was reported to the FDA on 3 July, is the third that has impacted Sarepta's gene therapy programme, with the first two deaths earlier this year in patients treated with Elevidys, both also due to ALF. Both Elevidys and SRP-9004 are adeno-associated virus (AAV) gene therapies that use the same AAVrh74 serotype. The FDA's Center for Biologics Evaluation and Research (CBER) director Dr Vinay Prasad, said: 'Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits. The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury.' This comes just days after Sarepta said it has agreed to change the black box on the Elevidys label to include ALF and acute liver injury (ALI) warnings. In the same announcement, Sarepta said it would be cutting 500 jobs as part of company restructuring. Speaking after the third patient death was announced, GlobalData healthcare analyst Momna Ali said the recent setbacks for Sarepta put the cell and gene therapy (CGT) sector at a 'crossroads'. Ali said: 'Following Sarepta's announcement of a third patient death, a new label for Elevidys and them laying off 500 employees, or 36% of its workforce, shelving parts of its pipeline to save $420m, and shifting focus from gene therapy to siRNA programmes, this is going to put the CGT sector at a pivotal crossroad. '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 has to be greater transparency, patient safety, and sustainable innovation – otherwise, it'll be met with more setbacks.' The news of the LGMD death came to light on 17 July in media reports. After this was reported, Sarepta's stock has dropped 43.21%, from $22.54 at market open on 17 July to $12.80 at market open on 21 July. The FDA also said it has revoked the platform technology designation for Sarepta's AAVrh74 Platform Technology. "Sarepta refuses to pull gene therapy despite FDA order" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Yahoo
16-06-2025
- Business
- Yahoo
Sarepta and Roche halt DMD gene therapy's use after second death
Serepta and global partner Roche have discontinued the commercial and clinical use of Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec) in non-ambulatory patients after a safety profile re-assessment following two patient deaths. Roche said that the dosing restrictions are effective immediately, with commercial use stopped and enrolment in clinical trials paused until additional risk mitigation measures are implemented in the study protocol. In a separate announcement, Sarepta said it is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for the therapy. The biopharma added it is temporarily suspending shipments of Elevidys for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated. The re-assessment of the benefit-risk ratio for non-ambulatory patients follows a second fatal acute liver failure (ALF) case in a DMD patient administered with Elevidys. Two fatal ALF cases have occurred in the gene therapy's use to date. Shares in Swiss-listed Roche fell 1% at market open following the development, while Sarepta was down 31.34% in pre-market trading on the Nasdaq. 'While we ultimately believe the implementation of a modified immunosuppression regimen could prevent future safety events in non-ambulatory patients, we recognise that another patient death will amplify investor concerns surrounding the potential removal of Elevidys from the commercial market, which we continue to view as an unlikely scenario,' said William Blair analyst Sami Corwin in a research note. Corwin added that Elevidys's label will likely be updated to reflect the risk of ALF. Elevidys is approved in the US for the treatment of ambulatory and non-ambulatory individuals aged four and older with DMD with a confirmed mutation in the DMD gene. It is the only gene therapy approved in the country for the rare musculoskeletal disease. Elevidys works by inserting a functional copy of the DMD gene, providing the missing instructions used to make the protein dystrophin. Sarepta Therapeutics, who developed the therapy, holds US rights to the drug, while Roche gained rights to other global territories through a 2019 agreement. The first death due to ALF, announced in March 2025, prompted the suspension of multiple clinical trials evaluating the gene therapy in different patient populations with DMD. The clinical holds are still in effect, whilst dosing will be paused in trials operating outside of Europe. Roche and Sarepta jointly manage the clinical studies for Elevidys. The companies did not release further details on the second death. Sarepta stated the event has been reported to the US Food and Drug Administration (FDA) and global health authorities. While elevated liver enzymes are a known side effect of adeno-associated virus-based gene therapies, the exact mechanism behind liver toxicity remains unclear. "We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,' Garraway said "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect,' he added. 'Our paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory,' said Sarepta's chief scientific officer and head of R&D Louise Rodino-Klapac. "Sarepta and Roche halt DMD gene therapy's use after second death " was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
16-06-2025
- Business
- Yahoo
Sarepta and Roche halt DMD gene therapy's use after second death
Serepta and global partner Roche have discontinued the commercial and clinical use of Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec) in non-ambulatory patients after a safety profile re-assessment following two patient deaths. Roche said that the dosing restrictions are effective immediately, with commercial use stopped and enrolment in clinical trials paused until additional risk mitigation measures are implemented in the study protocol. In a separate announcement, Sarepta said it is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for the therapy. The biopharma added it is temporarily suspending shipments of Elevidys for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated. The re-assessment of the benefit-risk ratio for non-ambulatory patients follows a second fatal acute liver failure (ALF) case in a DMD patient administered with Elevidys. Two fatal ALF cases have occurred in the gene therapy's use to date. Shares in Swiss-listed Roche fell 1% at market open following the development, while Sarepta was down 31.34% in pre-market trading on the Nasdaq. 'While we ultimately believe the implementation of a modified immunosuppression regimen could prevent future safety events in non-ambulatory patients, we recognise that another patient death will amplify investor concerns surrounding the potential removal of Elevidys from the commercial market, which we continue to view as an unlikely scenario,' said William Blair analyst Sami Corwin in a research note. Corwin added that Elevidys's label will likely be updated to reflect the risk of ALF. Elevidys is approved in the US for the treatment of ambulatory and non-ambulatory individuals aged four and older with DMD with a confirmed mutation in the DMD gene. It is the only gene therapy approved in the country for the rare musculoskeletal disease. Elevidys works by inserting a functional copy of the DMD gene, providing the missing instructions used to make the protein dystrophin. Sarepta Therapeutics, who developed the therapy, holds US rights to the drug, while Roche gained rights to other global territories through a 2019 agreement. The first death due to ALF, announced in March 2025, prompted the suspension of multiple clinical trials evaluating the gene therapy in different patient populations with DMD. The clinical holds are still in effect, whilst dosing will be paused in trials operating outside of Europe. Roche and Sarepta jointly manage the clinical studies for Elevidys. The companies did not release further details on the second death. Sarepta stated the event has been reported to the US Food and Drug Administration (FDA) and global health authorities. While elevated liver enzymes are a known side effect of adeno-associated virus-based gene therapies, the exact mechanism behind liver toxicity remains unclear. "We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,' Garraway said "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect,' he added. 'Our paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory,' said Sarepta's chief scientific officer and head of R&D Louise Rodino-Klapac. "Sarepta and Roche halt DMD gene therapy's use after second death " was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Business Insider
15-06-2025
- Business
- Business Insider
Sarepta suspends non-ambulatory Elevidys shipments after second death
Sarepta (SRPT) Therapeutics provided a safety update regarding Elevidys, an FDA approved gene therapy for patients with Duchenne muscular dystrophy. The company announced a second reported case of acute liver failure resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta is temporarily suspending shipments of Elevidys for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place. For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after Elevidys infusion, along with post-treatment monitoring, remains the same. Sarepta has also voluntarily paused dosing in the ENVISION clinical study. FDA concurs with this action, the company said. 'The pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from the FDA. Regulatory alignment is needed before screening and dosing in ENVISION may resume,' Sarepta added. The company is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for Elevidys.
Buzz Feed
12-06-2025
- Entertainment
- Buzz Feed
Anybody Remember Cartoon All-Stars To The Rescue?
In 1990, a special animated program was created entitled Cartoon All-Stars To The Rescue, which was created to teach kids about the dangers of marijuana and drugs. It follows a teenager named Michael, who is using cannabis, and various cartoon characters, from their perspective shows that were popular at the time, who confront him after coming to life from items in his sister's room. The program featured characters from 10 different shows, including The Smurfs, ALF, Garfield and Friends, Alvin and the Chipmunks, Winnie the Pooh, Muppet Babies, The Real Ghostbusters, Looney Tunes, and Teenage Mutant Ninja Turtles. The special aired on all superstations, along with other independent stations. The special is considered a time capsule of animation history due to it featuring various characters from various Saturday morning shows. Some even consider it one of the greatest cartoon crossovers of all time. Do you remember watching this? Comment below!
