July 16, 2026

Novartis’ Fabhalta Receives FDA Traditional Approval as First and Only Complement Inhibitor to Slow Kidney Function Decline in Primary IgAN

Novartis reported that the FDA has granted traditional approval to Fabhalta (iptacopan) to slow kidney function decline in adults with primary IgAN at risk of disease progression. The approval, granted under Priority Review, converts Fabhalta’s August 2024 accelerated approval, which was based on proteinuria reduction, into traditional approval and is supported by the P3 APPLAUSE-IgAN study. Over two years, Fabhalta reduced the rate of eGFR decline by 48% vs pbo, with an annualized mean eGFR change of 3.0 vs – 5.7 mL/min/1.73 m²/year, demonstrating preservation of kidney function; reductions in proteinuria were observed as early as two weeks and sustained throughout treatment. The safety profile was consistent with prior data, with abdominal pain, dizziness, and nausea among the most common adverse events; treatment carries a risk of serious infections from encapsulated bacteria and is available through a REMS program requiring appropriate vaccination before treatment. Novartis also highlighted its broader IgAN portfolio, including Vanrafia (atrasentan) and investigational zigakibart, as well as patient access programs, with nearly 100% of U.S. patients paying $10 or less per month.

Source: GlobeNewswire PR Jul 16, 2026

July 17, 2026

Boehringer Ingelheim’s JASCAYD Gains EU Approval as Novel Oral Treatment for IPF and PPF

BI received approval from the EC for JASCAYD (nerandomilast) to treat idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) in adults in the EU. The approval was based on results from the P3 FIBRONEER program, comprising the FIBRONEER-IPF and FIBRONEER-ILD studies. Both trials met their primary endpoints, demonstrating that JASCAYD successfully slowed lung function decline, measured by the absolute change in forced vital capacity (FVC) from baseline to wk52 vs. pbo. Although the key secondary endpoint was not met in either trial, a numerical reduction in mortality was observed across both studies, reaching nominal statistical significance in FIBRONEER-ILD. JASCAYD demonstrated similar discontinuation rates to pbo and a favourable safety and tolerability profile, with no requirement for routine liver monitoring.

Source: GlobeNewswire PR Jul 17, 2026

July 20, 2026

FDA Grants FTD to Acadia’s Remlifanserin for Psychosis Associated with Alzheimer’s Disease

Acadia Pharmaceuticals reported that the U.S. FDA has granted FTD to remlifanserin, an investigational, highly selective 5-HT2A receptor inverse agonist, for the treatment of hallucinations and delusions associated with Alzheimer’s disease psychosis (ADP), a serious condition with no FDA-approved therapies. The designation is intended to facilitate development and expedite regulatory review for therapies addressing serious conditions with unmet medical needs. Acadia also announced that enrolment in the P2 RADIANT study has been completed, with topline results expected in Sep – Oct 2026, while screening and enrolment for the P3 portion of the RADIANT program are already underway.

Source: Business Wire PR Jul 20, 2026

July 21, 2026

Kyowa Kirin’s CRYSVITA Receives EU Label Extension for Infants and Children with X-linked Hypophosphataemia

Kyowa Kirin reported that the EC has approved a label extension for CRYSVITA (burosumab) to treat infants and children from 1 year of age with X-linked hypophosphataemia (XLH) and radiographic evidence of bone disease in EU. This approval expands the use of CRYSVITA to younger patients and is based on clinical data demonstrating that treatment can improve rickets severity and lower-limb deformities, while supporting improvements in phosphate homeostasis and bone health. The decision was supported by results from P1/2 BUR-CL207 study, which demonstrated a safety profile consistent with previously established data in infants. The approval also grants Crysvita a 2-year extension of orphan market exclusivity in the EU for XLH, extending protection from Feb 2028-30.

Source: Business Wire PR Jul 21, 2026

July 22, 2026

Affinia Therapeutics’ AFTX-201 Receives FDA Orphan Drug Designation for BAG3-Associated Dilated Cardiomyopathy

Affinia Therapeutics reported that the U.S. FDA has granted ODD to AFTX-201, an investigational AAV-based gene therapy, for the treatment of BAG3-associated dilated cardiomyopathy (DCM), a rare and potentially fatal genetic heart disease with no currently available disease-modifying treatments. AFTX-201 is designed as a one-time IV infusion to deliver a functional copy of the BAG3 gene to cardiac muscle cells, with the goal of addressing the underlying genetic cause of the disease. The therapy utilizes Affinia’s proprietary rationally designed capsid, engineered to enable efficient cardiac transduction at doses 5-10 fold lower than those typically used with conventional capsids such as AAV9 or AAVrh74. AFTX-201 is currently being evaluated in the P1/2 UPBEAT study in adults aged 18-55 years with BAG3-associated DCM.

Source: Business Wire PR Jul 22, 2026

July 23, 2026

Nuvectis Reported Ciprocopan Receives Approval in China for Treatment-Naive PNH

Nuvectis Pharma reported that its partner Haisco received NMPA’s (China) marketing approval for ciprocopan (NXP100) to treat paroxysmal nocturnal hemoglobinuria (PNH) in patients previously untreated with complement inhibitors, marking the first global approval of a once-daily oral Factor B inhibitor. The approval was supported by results from a H2H P3 study assessing ciprocopan vs. eculizumab (Soliris), in which ciprocopan met all primary and secondary endpoints.

Key Efficacy Measures: