PBC competitive landscape updates

Immagine News

Ocaliva

  • FDA issued  a new safety communication regarding risk of liver injury in non-cirrhotic PBC patients treated with Ocaliva (12/12/24)
    • The FDA identified a significant risk among non-cirrhotic PBC patients taking Ocaliva based on data from the Phase 3b/4 COBALT study (747-302)
    • This update follows the FDA’s 2021 restrictions on Ocaliva’s use for patients with advanced cirrhosis
  • The EU’s General Court reversed its decision to suspend the European Commission’s (EC) revocation of conditional marketing approval for Advanz’s Ocaliva (11/26/24)
    • Recall that the EC General Court had temporarily suspended the EC’s revocation to hear Advanz petitions for “interim measures” that would further delay Ocaliva’s removal from the market
    • The General Court denied the first of Advanz’s petitions but continues to hear arguments in this case, meaning Ocaliva’s marketing status could change again
    • EU patients with active Ocaliva prescriptions can still access treatment in some Countries

Iqirvo

    • Iqirvo’s Phase 2 ELMWOOD trial investigating Iqirvo in PSC was listed as “fully recruited”; data readout expected in 2026 (PCD: Aug 2026), and potential launch in 2031+, following a Phase 3 trial
    • Ipsen discussed its rare disease portfolio as consisting of Iqirvo, Bylvay (odevixibat) and Sohonos (palovarotene)
    • Ipsen also reiterated that data readout for the global Phase 3 ELSPIRE trial (N=72; PCD: Oct 2026) of Iqirvo in 2L PBC is expected in 2026 [known]
  • France’s High Authority of Health (Haute Autorité de Santé, HAS) denied post-market authorization early access for Ipsen’s Iqirvo for 2L PBC in combination with UDCA, or as monotherapy in patients who do not tolerate UDCA (01/07/25)
    • Based on clinical data (Phase 2 GFT505b-216-1 and Phase 3 ELATIVE), the Transparency Committee (CT) concluded that:
      • Iqirvo is not an innovation in PBC treatment
      • Iqirvo demonstrated weak efficacy on PBC progression and symptoms
      • Bezafibrate is a suitable 2L treatment option, minimizing unmet need
    • While the HAS did not fully agree with all CT conclusions, it considers bezafibrate to be an adequate 2L PBC treatment option, consistent with the general sentiment in France
  • Ipsen listed the new Phase 3 ELONSEN trial (N=18; PCD: Mar 2026) in Japan assessing Iqirvo as 2L treatment for adult PBC patients with inadequate response or intolerance to UDCA (12/11/24)
    • The study aims to determine if Iqirvo reduces alkaline phosphatase (ALP) and total bilirubin levels in Japanese patients
    • Assuming data readout and filing occurs soon after the study’s primary completion date (Mar 2026) and a ~12-month review period, Iqirvo could potentially be approved in Japan in H1 2027

Pipeline

Linerixibat

  • CHMP posted the Jan 2025 meeting agenda (Jan 27th-30th), with GSK’s linerixibat (IBATi) listed under Section 8 “Pre-submission issues” (01/27/25)
    • The committee will discuss GSK’s request for accelerated assessment of linerixibat for cholestatic pruritus in PBC
    • Assuming filing acceptance in early 2025 (in line with prior GSK guidance), estimated approval could occur in ~Q3 2025 if the CHMP agrees to an accelerated assessment
    • Notably, GSK does not include linerixibat in its list of 5 key 2025 launches discussed during the 2025 J.P. Morgan Healthcare Conference (01/14), suggesting the company could expect linerixibat revenues to be low compared to other approvals anticipated in 2025
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