How big is the LSD patient population market?

Based on current treatments and survival estimates, we expect about 28,000 cases of diagnosed prevalent cases of LSDs in 2015 in the mature markets. That number is expected to remain stable over the next ten years. This figure captures 27 of over 50 distinct LSDs.

We estimate that the United States and France will see an increase in the number of diagnosed prevalent cases of LSDs whereas Germany, Italy, Spain, the United Kingdom and Japan will experience a slight decrease in the number of diagnosed prevalent cases due to changes in population demographics.

Which LSDs are most common?

Sphingolipidoses like Gaucher’s disease comprise just about half of all prevalent LSD cases in 2015, over 14,000 cases combined, in the mature markets. Mucopolysaccharidoses are the second most common class of

LSDs followed by glycogen storage diseases and LAL deficiencies like Wolman’s disease.

How might improvements in treatment impact the number of patients living with an LSD?

The dynamics of diagnosed prevalent LSD patient populations are inherently linked to survival. Many LSDs, especially early-onset disorders, have a high mortality and the earlier treatment begins, the better the outcome.

Even small increases in patient survival can have a large impact on diagnosed prevalent populations. As such, we have explicitly built these dynamics into our forecast model in anticipation of future improvements among available therapies. For example, studies have shown ERT to be effective in extending survival among patients with Pompe disease.

Opportunities for treatment

To date, most LSDs do not have an approved therapy available for treatment. When we think about investment in drug-development and the market price that orphan drugs can command, we can see how there is still tremendous untapped market potential among these rare inborn errors of metabolism. While promising, gene therapy is still a long ways off leaving LSDs to offer opportunities today for novel, targeted therapies.

For more information, check out my upcoming webinar on the Epidemiology of Lysosomal Storage Disorders in the Mature Markets.

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