top of page

EMA Drug Approvals - CHMP Positive Opinions | March 2024 | iPharmaCenter

Updated: Mar 24

  1. Novartis' Fabhalta Receives Favorable CHMP Opinion for PNH Treatment Advancement

  2. UCB's Bimzelx (bimekizumab) garners favorable CHMP recommendation for treating adults grappling with moderate to severe hidradenitis suppurativa


 

Novartis' Fabhalta Receives Favorable CHMP Opinion for PNH Treatment Advancement

Novartis' Fabhalta (iptacopan) has garnered a favorable opinion from the CHMP, marking a significant milestone as the initial oral monotherapy proposed for adult individuals grappling with paroxysmal nocturnal hemoglobinuria (PNH). This affirmative stance from the CHMP is underpinned by compelling Phase III findings, notably from the APPLY-PNH trial, showcasing notable enhancements in hemoglobin levels sans transfusions among Fabhalta recipients compared to those undergoing anti-C5 therapy. If granted approval, Fabhalta will emerge as the pioneering oral monotherapy accessible in Europe for addressing PNH, a persistent and rare hematologic ailment, applicable to both patients who have received complement inhibitor treatment and those who have not, but suffer from hemolytic anemia.


Despite the application of anti-C5 therapy, a considerable segment of patients continues to grapple with anemia, fatigue, and reliance on blood transfusions. The advanced-stage development initiative for Fabhalta remains underway, targeting various diseases mediated by the complement system.


Novartis made the announcement today regarding the favorable opinion bestowed by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), advocating for the authorization of Fabhalta (iptacopan) for managing hemolytic anemia in adult PNH sufferers. PNH, a rare and incapacitating hematologic condition, ensues when certain blood cells, inherently susceptible to the complement system, proliferate abnormally over healthy blood cells due to conducive immune circumstances. It is characterized by hemolysis, bone marrow malfunction, and thrombotic events, varying in severity and combinations.


Presently, anti-C5 therapies necessitate either infusion or subcutaneous injection and may leave PNH symptoms inadequately controlled. A significant proportion of patients undergoing anti-C5 treatment continue to endure persistent anemia, with a considerable portion relying on blood transfusions. Furthermore, the majority of these patients experience fatigue.


The positive decision from the CHMP is grounded in robust data stemming from the Phase III APPLY-PNH investigation involving patients experiencing lingering anemia despite prior anti-C5 treatment who transitioned to Fabhalta compared to those persisting with anti-C5 therapy, alongside the Phase III APPOINT-PNH trial involving patients naive to complement inhibitors. In APPLY-PNH, by the 24th week, 82.3% of Fabhalta recipients with prior anti-C5 exposure achieved a sustained elevation in Hb levels ≥2 g/dL from baseline without requiring transfusions, in contrast to a mere 2.0% among anti-C5 adherents. In APPOINT-PNH, 92.2% of complement inhibitor-naive patients administered Fabhalta achieved a similar outcome. Additionally, in APPLY-PNH, the transfusion avoidance rate was notably higher among Fabhalta recipients with prior anti-C5 exposure compared to those on anti-C5 therapy.


Both trials underscored Fabhalta's efficacy in mitigating intravascular hemolysis, as indicated by the maintenance of mean lactate dehydrogenase (LDH) levels below the upper limit of normal. Noteworthy enhancements in fatigue were reported by patients in the APPLY-PNH trial, measured by Functional Assessment of Chronic Illness Therapy – Fatigue [FACIT-F] scores. The safety profile of iptacopan remained consistent across both APPLY-PNH and APPOINT-PNH studies.


 

UCB's Bimzelx (bimekizumab) garners favorable CHMP recommendation for treating adults grappling with moderate to severe hidradenitis suppurativa


UCB announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has rendered a positive recommendation for marketing authorization of BIMZELX (bimekizumab) within the European Union (EU)/European Economic Area (EEA) to manage active moderate to severe hidradenitis suppurativa among adults who have not adequately responded to conventional systemic HS therapy. Should the European Commission (EC) grant approval, bimekizumab will emerge as the inaugural IL-17A and IL-17F inhibitor sanctioned in the EU for addressing moderate to severe HS in adults.


The encouraging CHMP opinion stems from evidence presented in the Phase 3 studies, BE HEARD I and BE HEARD II, demonstrating substantial improvements with bimekizumab compared to placebo at Week 16, with sustained benefits observed up to Week 48.


Hidradenitis suppurativa, a profoundly impactful chronic inflammatory skin condition, lacks sufficient approved treatment options, underscoring the significance of UCB's achievement.


The CHMP's affirmative stance on bimekizumab draws from outcomes of the Phase 3 BE HEARD I and BE HEARD II trials assessing the efficacy and safety of bimekizumab in adults with moderate to severe HS. Data showcased statistically significant and clinically meaningful enhancements over placebo in HS signs and symptoms, as measured by HiSCR50 at Week 16, the primary endpoint, with sustained responses up to Week 48. Bimekizumab treatment also yielded improvements over placebo in the HiSCR75 at Week 16, a crucial secondary endpoint, with sustained responses up to Week 48. The safety profile of bimekizumab remained consistent with previous study findings, devoid of new safety concerns.


The CHMP's favorable perspective on bimekizumab for moderate to severe HS will now undergo review by the EC for its final decision. Upon approval, the marketing authorization will extend to all EU member states and countries within the EEA.

0 comments

Comments


bottom of page