Commentary
Video
Pharmacists are crucial in Elevidys administration, ensuring accurate dosing, coordinating treatment timing, and managing adverse effects to optimize patient outcomes.
In an interview with Pharmacy Times®, Craig McDonald, MD, chair of the department of physical medicine and rehabilitation at UC Davis Health, discusses the critical role of pharmacists in the multidisciplinary team for patients with Duchenne muscular dystrophy (DMD) being treated with delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics). In this role, pharmacists contribute to accurate dosing, timely administration, and close post-infusion monitoring while helping to secure treatment authorization and coordinate care, according to McDonald.
As gene therapies for DMD continue to advance, McDonald emphasizes that pharmacists will continue to support clinical trials and expand treatment access to non-ambulatory DMD patients.
Pharmacy Times: How can pharmacists collaborate with other members of a patient’s care team to ensure accurate dosing, proper monitoring, and optimized treatment with Elevidys?
1. Pharmacists ensure precise dosing, assist with insurance approvals, and prevent unnecessary drug waste due to the high cost of Elevidys.
2. Close communication between the pharmacist and care teams prevents treatment during acute illness, ensuring safe administration.
3. While thrombocytopenia risk is low, pharmacists should watch for myocarditis and liver injury, optimizing patient outcomes.
Craig McDonald, MD: I think the pharmacists are really an important component of the team. We have to really ensure that we have an accurate, calculated dose that we're going after in terms of approval. These are very expensive therapeutics, and so we also have to ensure that we're doing close communication and monitoring. When a patient, for instance, may have an acute illness prior to the scheduled day of admission and treatment, we may have to cancel the therapeutic administration. We don't want to be treating patients on top of an acute illness. In addition, on the day of treatment, we also have to be in communication with the pharmacist, because if the patient is actually on site, healthy, and ready to receive the treatment, we don't want the pharmacy team, who may be thawing the medication, to be commencing with that preparation when the patient may not be in an optimum state of health to receive the treatment. I think the pharmacy team also helps us in terms of procuring the authorization for the treatment, and I think they're very helpful in terms of being part of that team when we're doing the day-of infusion monitoring as well as the post-treatment monitoring with Elevidys.
In addition, the pharmacist helps us in terms of the peri-infusion steroid doses and dose calculations there. These patients have to be on higher doses of peri-infusion steroids, higher than their standard of care steroids, and so doses that are typically around 1 mg/kg of prednisone, or if they require acute management of something like liver injury or other side effects such as myocarditis, we may need to increase the steroid dose up to 2 mg/kg. Also, we typically treat patients with an appropriate dose of ondansetron (Zofran; Pfizer) to help prevent the nausea and vomiting, which frequently occur in up to 50% to 60% of patients after treatment. Other side effects that we see rarely can be viral-mediated myocarditis and then, as I mentioned, acute liver injury. Fortunately, with Elevidys, we haven't seen really significant thrombocytopenia, which is usually a complement-mediated toxicity that has been shown as a class effect in gene therapy and can sometimes lead to complement-mediated toxicity, such as hemolytic uremic syndrome and so forth. Fortunately, with Elevidys, we haven't seen too much of an issue there. I would say the pharmacists are really a critical component of our team that is treating these patients with this complex gene therapy.
Pharmacy Times: Is there anything else that you want to add?
McDonald: I would just like to say that we're excited about the conduct of future clinical trials. Many of those trials, the Elevidys trials in the non-ambulatory population, are being conducted at European sites. We're now commercially treating non-ambulatory patients based on accelerated approval criteria that allow us to treat patients if we obtain insurance authorization to try to get the patients to the drug treatment for these non-ambulatory patients sooner. We're really looking forward to getting more safety and efficacy data on the older, non-ambulatory patients with DMD in terms of preservation of arm function, upper limb function, and also preservation of breathing function and heart function.
