Sarepta Working to Ensure Treatment Access, Clinical Trials Continuity Amid COVID-19 Pandemic
Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and Exondys 51 (eteplirsen), both approved for the treatment of Duchenne muscular dystrophy (DMD), during the COVID-19 pandemic.
Also, the company continues to work with regulatory authorities to guarantee the continuation of ongoing clinical trials, its Patient Affairs team said in an update to the DMD and limb-girdle muscular dystrophy (LGMD) communities.
COVID-19 (COrona VIrus Disease 2019) is an infectious disease that is believed to spread chiefly from person to person through small droplets of moisture that are produced when sneezing or coughing. Infection may also occur by touching a contaminated surface or object and then touching the mouth, nose, or eyes.
Common symptoms include fever, dry cough, and difficulty breathing, which can occur up to 14 days after exposure.
While this pandemic is causing medical and economic uncertainty, Sarepta said it will continue to monitor the evolving situation and do everything possible to meet the needs of the DMD and Limb-girdle communities.
Both Vyondys 53 and Exondys 51 are exon-skipping therapies, as they work by masking the mutated DNA bits that cause disease to enable production of a shortened but functional version of dystrophin. People with DMD have little to none of this key protein for muscle health.
Patients with questions about these therapies can call SareptAssist at 1-888-727-3782.
The company said it will continue to “provide an uninterrupted supply of its therapies,” and has activated additional plans to minimize disruption to patients. Home infusion nurses have hygiene and equipment protocols in place to protect both patients and themselves, Sarepta said. These protocols follow guidance from the Centers for Disease Control and Prevention.
The company is also working on the development of gene therapies for DMD and LGMD, with clinical trials currently ongoing.
“Sarepta is closely monitoring our studies and working to mitigate disruption and delay in both dosing or follow-up visits,” the company stated. Specifically, this includes working with regulatory authorities, such as the U.S. Food and Drug Administration, to minimize risk to trial participants and then to the trial itself. Mitigation strategies, such as conducting follow-up visits online, are being considered.
Participants are being encouraged to stay in contact with appropriate coordinators at clinical trial sites, so that details can be discussed. Individuals receiving trial-associated healthcare at home should be able to continue to do so. “We are also working to expand access to home health services in trials where and when possible,” Sarepta said.
The company will update the community when trials, previously scheduled to start in 2020, actually begin.
“During a time of much ambiguity, we assure you that Sarepta remains steadfast in our focus and commitment to advancing and delivering innovative therapies for the Duchenne and Limb-girdle communities,” Sarepta’s team wrote. “We wish you all strength, health and safety through this difficult time.”