While FDA mulls Zevra’s rare disease med, Rafael merges with Cyclo to push their rival candidate

Fierce Biotech August 22, 2024
James Waldron

With just weeks to go before the FDA decides whether to approve the first treatment for a rare genetic condition, Rafael Holdings is merging with Cyclo Therapeutics to try to push their own candidate over the line.

Cyclo’s lead asset is Trappsol Cyclo, a formulation of hydroxypropyl beta cyclodextrin that is being evaluated in a phase 3 trial in Niemann-Pick disease type C1 (NPC). The rare, genetic lysosomal storage disease prevents the body from moving and using cholesterol and other lipids in cells. This leads to a buildup of cholesterol and other lipids in the liver, spleen or lungs.

After the deal closes, which is expected in late 2024, Rafael said it will continue to fund the phase 3 trial...

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