TAKEDA PHARMACEUTICAL COMPANY LIMITED

Health Canada Expands HyQvia Authorization for CIDP Maintenance Therapy

2 months ago

• Health Canada has expanded marketing authorization for Takeda's HyQvia as a maintenance therapy for chronic inflammatory demyelinating polyneuropathy (CIDP) in adults after stabilization with intravenous immunoglobulin. • HyQvia is now the only subcutaneous immunoglobulin treatment in Canada that can be administered as infrequently as once monthly and can be self-administered at home after proper training, reducing hospital visits. • The approval is based on the Phase 3 ADVANCE-1 trial, which demonstrated a significant reduction in CIDP relapse rates with HyQvia compared to placebo (15.5% vs 31.7%), with an estimated treatment difference of -16.2%.

FDA Approves Tablet Formulation of LIVMARLI for Rare Pediatric Liver Diseases

2 months ago

• Mirum Pharmaceuticals' LIVMARLI (maralixibat) has received FDA approval for a new tablet formulation to treat cholestatic pruritus in patients with Alagille syndrome and Progressive Familial Intrahepatic Cholestasis. • The tablet version provides a convenient one-tablet per dose option for older patients, while maintaining the liquid formulation for younger patients, enhancing treatment flexibility for physicians and patients. • LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is now approved in over 40 countries for ALGS and 30 countries for PFIC, with the new tablet formulation expected to be available in June 2025.

FDA Foregoes Advisory Committee for Cabozantinib's Neuroendocrine Tumor Application

4 months ago

• The FDA has canceled the Oncologic Drugs Advisory Committee meeting for Exelixis' cabozantinib sNDA, streamlining the review process. • The sNDA seeks approval for cabozantinib to treat advanced pancreatic and extra-pancreatic neuroendocrine tumors in previously treated adults. • The FDA's decision is based on Phase III CABINET trial results, which showed significant progression-free survival improvements with cabozantinib. • The Prescription Drug User Fee Act (PDUFA) target action date for the FDA's decision on cabozantinib remains April 3, 2025.

Rusfertide Achieves Primary Endpoint in Phase 3 VERIFY Trial for Polycythemia Vera

5 months ago

• Rusfertide significantly reduced the need for phlebotomies in polycythemia vera (PV) patients, with 77% achieving clinical response compared to 33% on placebo. • The VERIFY trial met all key secondary endpoints, including hematocrit control and improved patient-reported outcomes, demonstrating comprehensive efficacy. • The hepcidin mimetic was well-tolerated, with no new safety concerns, suggesting a favorable risk-benefit profile for PV patients. • Protagonist and Takeda plan to submit detailed results for presentation at upcoming medical conferences and to regulatory agencies for potential approval.