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.

test tube

Ionis Pharmaceuticals Inc. subsidiary Akcea Therapeutics Inc. now has Phase III efficacy data in hand for volanesorsen in familial chylomicronemia syndrome (FCS), but market analysts are mixed on the implications of safety data showing frequent instances of reduced platelet counts, potentially leading to thrombocytopenia.

FCS, a rare disease affecting an estimated 3,000-5,000 patients worldwide, results in extremely high triglyceride levels and can lead to...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Clinical Trials

More from R&D

Sanofi Revels In More Positive Rilzabrutinib Data

 
• By 

The oral BTK inhibitor impresses in a Phase II trial for IgG4-related disease.

Is Zydus’s Agenus Deal Opportunistic Or Strategic?

 
• By 

Will Zydus’s acquisition and licensing deal with Agenus turn out to be a strategic move towards building an oncology portfolio or an opportunistic one driven by a venture capital mindset?

How AstraZeneca Aims To Stay Ahead In Lupus As New Competition Looms

 

The company has established the efficacy of its biologic Saphenlo in systemic lupus erythematosus but is now investing across modalities to stay ahead of rivals.