Regulation

Abeona announces FDA approval for cell-based gene therapy for RDEB patients

Abeona Therapeutics has announced receipt of the US Food and Drug Administration (FDA) approval for gene-modified cellular sheet therapy Zevaskyn for the wound treatment in both adult and paediatric patients suffering from recessive dystrophic epidermolysis bullosa (RDEB), a serious genetic skin condition.

EMA grants orphan drug status to Purespring’s IgA nephropathy treatment

The European Medicines Agency (EMA) has granted orphan drug designation to UK-based Purespring Therapeutics’ lead candidate, PS-002, aimed at treating IgA nephropathy (IgAN).

NICE issues final guidance endorsing Pharming’s APDS therapy

The National Institute for Health and Care Excellence (NICE) has issued final guidance endorsing Pharming Group’s Joenja (leniolisib) for use and reimbursement in the National Health Service (NHS) in England and Wales.

Ensem Therapeutics’ application for ETX-636 secures FDA clearance

Ensem Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its investigational new drug (IND) application for ETX-636.

Viatris files applications in Japan for approval of Effexor to treat GAD

Viatris has submitted applications to Japan's Ministry of Health, Labor and Welfare (MHLW) for the approval of Effexor SR Capsules (venlafaxine hydrochloride) to treat adults with generalised anxiety disorder (GAD).

ALX Oncology’s ALX2004 gains FDA IND clearance to treat solid tumours

ALX Oncology has received investigational new drug (IND) application clearance from the US Food and Drug Administration (FDA) for ALX2004, a new antibody-drug conjugate (ADC) aimed at treating solid tumours that express the epidermal growth factor receptor (EGFR).

FDA grants breakthrough therapy status for Amgen’s Uplizna to treat IgG4-RD

The US Food and Drug Administration (FDA) has granted breakthrough therapy designation for Amgen’s humanised monoclonal antibody Uplizna (inebilizumab-cdon) to treat the adult population with immunoglobulin G4-related disease (IgG4-RD), an immune-mediated inflammatory condition that impacts several organs.

FDA clears Epicrispr’s application for FSHD treatment

The US Food and Drug Administration (FDA) has granted clearance for an investigational new drug (IND) application of Epicrispr Biotechnologies’ EPI-321, intended for treating genetic neuromuscular condition, facioscapulohumeral muscular dystrophy (FSHD).

FDA grants fast track status to Biogen’s BIIB080 for Alzheimer’s

Biogen has received fast track designation from the US Food and Drug Administration (FDA) for its investigational antisense oligonucleotide (ASO) therapy, BIIB080, to treat Alzheimer's disease.