Novartis snags FDA nod for Fabhalta in another kidney disease but leaves room for rival Apellis

New FDA Approval for Fabhalta

• The FDA approved Novartis' Fabhalta (iptacopan) for the treatment of adults with C3 glomerulopathy (C3G), to reduce proteinuria10.

• This is the first and only FDA-approved treatment for C3G10.

• Fabhalta is an oral inhibitor of the alternative complement pathway, thought to target the underlying cause of C3G10.

Clinical Trial Results

• In the Phase 3 APPEAR-C3G trial, Fabhalta showed sustained proteinuria reduction at one year with a favorable safety profile10.

• Specifically, Fabhalta achieved a 35.1% relative proteinuria reduction versus placebo after 6 months7.

Competitive Landscape

• Apellis Pharmaceuticals is developing a rival drug, pegcetacoplan (branded as Empaveli), for C3G11.

• In Apellis' Phase 3 VALIANT trial, pegcetacoplan demonstrated a 68% reduction in proteinuria versus placebo at 26 weeks11.

• The stronger efficacy results for pegcetacoplan suggest it may have an advantage over Fabhalta if approved7.

Market Outlook

• C3G is an ultra-rare kidney disease typically diagnosed in young adults, with no previously approved treatments10.

• Analysts see the rare kidney disease space as a potential "blockbuster opportunity"11.

• Both Novartis and Apellis are advancing multiple kidney disease treatments to address high unmet needs1011.

In summary, while Novartis has secured the first FDA approval in C3G with Fabhalta, Apellis' pegcetacoplan has shown stronger efficacy data, setting up potential competition in this rare disease market if it gains approval.

Sources:

7. https://www.fiercepharma.com/pharma/apellis-and-novartis-drop-dueling-data-sets-they-race-approval-rare-form-kidney-disease

10. https://www.novartis.com/us-en/news/media-releases/novartis-receives-third-fda-approval-oral-fabhalta-iptacopan-first-and-only-treatment-approved-c3-glomerulopathy-c3g

11. https://www.fiercepharma.com/pharma/apellis-and-sobis-pegcetacoplan-wins-big-rare-kidney-disease-study-offering-renewed-hope-new