Rare Disease Spotlight: A genetic seizure disorder with a growing market, pipeline

Dravet syndrome has something many other severe, rare diseases do not — multiple approved treatments. Yet, much unmet need remains. With the indication’s first products establishing a growing market, opportunity exists for companies to develop safer or more effective next-generation therapies. The pipeline is accordingly moving toward more selective small molecules and therapies aimed at the disease’s root cause.

Each of the first three therapies approved for Dravet syndrome are in a different drug class, and each of those drug classes contain at least one more active drug development program, although the pipeline is also moving beyond those classes...