Akcea Faces Tricky Risk/Benefit Scenario In Orphan Disease FCS
The Ionis subsidiary's volanesorsen shows robust efficacy in a pivotal Phase III trial for familial chylomicronemia syndrome, but with high dropout levels for platelet reduction. Firm says platelet reductions are manageable and stem from the drug working as expected in the rare disease.
You may also be interested in...
Akcea terminates top three execs while praising their accomplishments in building up the Ionis spinout. Stock finished the trading day down nearly 20%.
Biohaven restructures its migraine partnership with Bristol to reduce its downstream royalty commitments. Meanwhile, the pharma partners with GRYT Health's cancer app for patient/caregiver insights into cancer therapy.
The latest drug approval and development news from our US FDA Performance Tracker.