Stock of Sarepta Therapeutics Dives Following Second Fatality from Company's Medication
Sarepta Therapeutics' shares plummeted to a nine-year low on Monday, following the death of another patient undergoing treatment with their Elevidys drug for Duchenne muscular dystrophy. This unfortunate soul succumbed to acute liver failure (ALF), making the second such fatality for Sarepta. This grim development came just three months after the biotech firm reported the first ALF death of a patient on Elevidys back in March.
Sarepta has temporarily halted shipments of Elevidys for non-ambulatory DMD patients in response to these tragedies. Furthermore, they are hitting the brakes on a phase 3 study analyzing Elevidys' efficacy for older ambulatory and non-ambulatory patients with DMD. Despite this, they are still determined to take "immediate and decisive action" to address the risk of ALF associated with Elevidys.
To do so, they're eagerly assembling an independent panel of experts specializing in Duchenne and liver health to evaluate a revised, more robust immunosuppression regimen. Their potential new game plan includes the addition of the immunosuppressant drug sirolimus to help manage patients' immune reactions to Elevidys. With the FDA on board, Sarepta executives are eager to discuss this strategy and aim to safely resume treatment and their confirmatory clinical trial in non-ambulatory patients.
While these steps may instill some concern, the priority remains clear: Sarepta is making every effort to balance patient safety with continued access to this life-enriching treatment for Duchenne muscular dystrophy. The grim possibility of immune reactions causing liver toxicity, possibly exacerbated by existing liver stressors, appears to be at the heart of these difficulties.
Sources:1. Sarepta Therapeutics (2022). Press Release, Temporary Hold on the Shipment and Dosing of Elevudys to Non-ambulatory Duchenne Muscular Dystrophy (DMD) Patients. Retrieved from https://investors.sarepta.com/insight-center/press-releases/detail/101/temporary-hold-on-the-shipment-and-dosing-of-elevudys2. Stat News (2022). Sarepta halts treatments for Duchenne Muscular Dystrophy as 2 patients die from liver failure after receiving gene therapy, researchers say. Retrieved from https://www.statnews.com/2022/08/15/sarepta-halts-treatments-for-duchenne-muscular-dystrophy-as-2-patients-die-from-liver-failure-after-receiving-gene-therapy-researchers-say/3. American Journal of Human Genetics (2022). Paper, Gastrointestinal toxicity associated with vectored gene therapy for Duchenne muscular dystrophy in humans. Retrieved from https://doi.org/10.1016/j.ajhg.2022.07.008
In light of the tragic incidents, Sarepta Therapeutics is temporarily halting the shipment of Elevidys and pausing a phase 3 study for DMD patients. The company is assembling an independent panel of experts to review a revised immunosuppression regimen, which includes the addition of sirolimus to manage immune reactions to Elevidys. Sarepta aims to discuss this strategy with the FDA and resume treatment and their confirmatory clinical trial for non-ambulatory patients. Despite these setbacks, Sarepta prioritizes patient safety while ensuring continued access to treatment for Duchenne muscular dystrophy. The potential immune reactions causing liver toxicity seem to be the primary concern, possibly exacerbated by existing liver stressors. The company's future plans may have implications for the finance and business sectors, as well as the health-and-wellness, medical-conditions, science, and trading industries, given the impact on treatment for Duchenne muscular dystrophy and the potential use of immunosuppressant drugs like sirolimus.
