Pipeline

Expanding our pipeline through Italfarmaco's internal R&D + external innovation

As the U.S. rare disease affiliate of the family-owned global pharmaceutical company Italfarmaco, we are positioned to support the commercialization of products in Italfarmaco’s pipeline here in the United States. We are also actively seeking partners with high-quality assets backed by strong data so we can bring our focus, agility, speed and expertise to deliver real value for people living with rare diseases in need of treatment options.

Disease Area
Program/Indication
Discovery
IND-enabling
Phase 1/2
Phase 3
Approved

Disease Area

Neuromuscular Disorder

Givinostat
Duchenne muscular dystrophy

Approved

Givinostat
Becker muscular dystrophy

Phase 1/2

Disease Area

Hematological Disease

Givinostat
Polycythemia Vera

Phase 3

Clinical Studies

We remain focused on generating additional data to support the safe and effective use of therapies.

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