Zydus receives USFDA ODD for Desidustat for the treatment of sickle cell disease

This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop a therapy for sickle cell disease

Shilpa Medicare & mAbTree Biologics win FDA nod for rare blood cancer therapy

The pioneering monoclonal antibody is for Essential Thrombocythemia (ET) and Polycythemia Vera (PV), rare chronic blood cancers with limited treatment options

AGC Biologics powers commercial launch of its life-changing gene therapy for rare childhood immune disorder

Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector

Takeda & Protagonist submit FDA application for groundbreaking polycythemia vera therapy

The investigational subcutaneous hepcidin mimetic peptide is designed to regulate iron and red blood cell production

Savara resubmits FDA application for potential treatment in rare lung disease patients

The resubmission of BLA for MOLBREEVI names Fujifilm as the drug substance manufacturer

Diamyd Medical accelerates Phase 3 Type 1 diabetes trial following FDA guidance

The interim efficacy readout, involving around 170 participants with 15-month data, remains on track for the end of March 2026

CPHI & PMEC India 2025 ignites pharma innovation and partnerships

For 2026 onwards, CPHI and PMEC India are evolving into two distinct, specialized shows

US House passes Bill reauthorizing FDA rare disease incentive program

Chugai’s Tecentriq gets green light in Japan for hard-to-treat Thymic Carcinoma

The drug had previously received orphan drug designation for this rare cancer on March 31, 2025

EU nod to Wayrilz as breakthrough treatment for hard-to-treat ITP

Wayrilz represents a new approach to ITP by targeting the disease at its root through multi-immune modulation