Sharon Hesterlee, PhD, on Opening Regulatory Pathways for Ultra-Rare Disease Treatments

The executive vice president and chief research officer of MDA discussed the barriers to FDA approval currently facing those developing treatments for ultra-rare diseases.

“One of the challenges in rare disease is that the cost of developing a drug for a common disease is not always that different from the cost of developing a drug for an ultra-rare disease. So it doesn't necessarily scale... And sometimes you can't make the drug expensive enough to make it viable for a company to develop [it]. And so that's the problem when we get to these ultra-rare diseases—these markets just aren't attractive. They're not easy markets for pharma, or biotechs even, to tackle.”

While the advent of adeno-associated virus-vector based gene therapy has opened the possibility to treat many ultra-rare genetic disorders from a technological standpoint, the process of evaluating these treatments in clinical trials and commercializing them is often not financially viable for biotech companies to undertake.

Sharon Hesterlee, PhD, the executive vice president and chief research officer of the Muscular Dystrophy Association (MDA), is heading MDA’s new Kickstart Program, which is seeking to support the development of gene therapy treatments for ultra-rare diseases.1 Hesterlee chaired a session focused on the program at the MDA Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023.2

In an interview with CGTLive™’s sister publication, NeurologyLive™, Hesterlee spoke about the challenges currently facing companies developing treatments for ultra-rare diseases, noting that the costs of developing these treatments is often as expensive as developing treatments for common diseases, despite the smaller population of patients who will benefit from the treatment. Furthermore, she pointed out that the FDA currently requires companies to produce 3 commercial grade batches of gene therapies in order to qualify for approval, and that this is often not cost-effective for companies to do because it could entail the manufacture of substantially more product than is necessary to treat all the patients currently living with the disease. Hesterlee also discussed the gene therapy program that the MDA Kickstart Program is currently supporting for the treatment of congenital myasthenic syndrome.

Read more coverage of the 2023 MDA Conference here.

1. Muscular Dystrophy Association. MDA Kickstart Program. Accessed April 10, 2023.
2. Hesterlee S. MDA Kickstart Program for Ultra-Rare Gene Therapy Development. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Ultra Rare Track session.
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