Travere Therapeutics

Ed Arce of H.C. Wainwright maintains Buy rating on Mirum Pharmaceuticals (MIRM) with $66.00 price target, citing FDA's Breakthrough Therapy Designation for volixibat in treating cholestatic pruritus in PBC patients, and its potential to offer substantial improvement over existing therapies.

September 2024 FDA updates include approvals for Embecta's tubeless patch pump for diabetes, Travere Therapeutics' sparsentan for IgA nephropathy, Johnson & Johnson's guselkumab for ulcerative colitis, and the first over-the-counter hearing aid software device. Other approvals cover treatments for conditions like chronic rhinosinusitis with nasal polyps, atopic dermatitis, eosinophilic granulomatosis with polyangiitis, Niemann-Pick disease type C, and schizophrenia. The FDA also issued warnings and accepted NDAs for various treatments, including delgocitinib cream for chronic hand eczema and roflumilast foam for psoriasis.

Travere Therapeutics pauses subject enrolment in Phase III HARMONY Study of pegtibatinase for classical homocystinuria to implement manufacturing scale-up improvements, aiming to resume enrolment in 2026.

Travere Therapeutics pauses enrollment in Phase 3 HARMONY Study for pegtibatinase due to manufacturing scale-up issues, aiming to resume in 2026. Current patients continue receiving treatment unaffected by the pause.

IgA nephropathy (IgAN) is a common autoimmune kidney disease characterized by IgA deposits, leading to progressive damage and chronic kidney disease (CKD). 40-53% of patients progress to end-stage renal disease (ESRD) within two decades, necessitating interventions like dialysis or transplantation. The treatment landscape for IgAN is evolving with ongoing phase 3 trials for 11 agents, including budesonide and sparsentan, which have received FDA approval. Early detection and intervention are crucial to delay progression, reduce healthcare costs, and improve patient outcomes.

FDA commissioner Robert Califf defends agency's standards amid criticism for granting full approval to Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy Elevidys and Travere Therapeutics’ drug Filspari for IgAN, despite failed Phase 3 trials.

Canaccord Genuity raises Travere Therapeutics' price target to $23.00 following FDA's full approval of Filspari for IgAN, expanding its indication. Travere's FILSPARI sales grew 37% to $27.1 million in Q2 2024, with a projected market penetration of 13.8% by 2031. Citi and BofA Securities maintain Buy ratings, anticipating market expansion.

FDA approves Travere Therapeutics’ Filspari (sparsentan) for IgA nephropathy, the first non-immunosuppressive treatment to significantly slow kidney function decline. Based on the PROJECT study, Filspari outperformed irbesartan in preserving kidney function over two years. Common adverse events include hyperkalemia and hypotension.