Jennifer Morganroth, MD, MBA, on the Importance of Preparing Clinical Resources for Genetic Medicines

The neurology resident at Penn Medicine spoke about the preparations taken in the lead up to tofersen’s PDUFA date, which is set for April 25, 2023.

“We often don't think about—when drugs get approved—how that translates to what we need in our clinics. I think this model is one of many that could be used—especially the model I created, [it] has the ability to be used for other genes; it's very flexible. And so, I do think it's very important as these new genetic therapies [and] intrathecal ASOs are becoming approved, that we actually start to think about how we're going to deliver these therapies.”

With many genetic medicines currently in development for the treatment of neuromuscular diseases, it is an important consideration to ensure that healthcare providers have the necessary resources to deliver these treatments to patients in a timely manner, if they are approved for clinical use.

Jennifer Morganroth, MD, MBA, a PGY3 neurology resident at Penn Medicine in the University of Pennsylvania Health System, presented on this topic during a session entitled “Gene Therapy in ALS” at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023. Her presentation focused specifically on the measures taken regarding Biogen’s tofersen, an investigational antisense oligonucleotide (ASO) being evaluated for the treatment of patients with amyotrophic lateral sclerosis (ALS) related to mutations in the SOD1 gene. Tofersen has a PDUFA date approaching on April 25, 2023.

In an interview with CGTLive™, Morganroth spoke about her presentation, noting that she presented on a population model she and her team constructed for patients with SOD1 mutations in the northeastern United States. The model was used to predict the number of patients that would likely seek treatment at their center and the number of lumbar punctures, genetic counselors, and office visits that would be required to meet these patients’ needs. Morganroth emphasized that meeting the clinical capacity to provide lumbar puncture delivery in adequate amounts remains one of the biggest challenges for intrathecal therapies. She also briefly summarized another presentation given during the session that covered similar ground to her own and spoke about the potential implications for patients if tofersen is approved.

Read more coverage of the 2023 MDA Conference here.

Editor’s Note: Morganroth disclosed that she participated in an MD Co-op for the Neuromuscular Clinical Development Unit at Biogen as a paid 5 week elective over the summer of 2022.

1. Biogen provides update on FDA advisory committee meeting on tofersen for SOD1-ALS. News release. Biogen Inc. March 22, 2023. Accessed April 3, 2023.
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