Regulation

FDA grants fast track status to Zai Lab’s lung cancer treatment

The US Food and Drug Administration (FDA) has granted fast track designation to Zai Lab’s delta-like ligand (DLL3) antibody-drug conjugate (ADC), ZL-1310, for treating extensive-stage small cell lung cancer (ES-SCLC).

FDA grants RMAT to BrainChild Bio’s CAR T-cell therapy for DIPG

BrainChild Bio's B7 homolog 3 (B7-H3) targeting autologous chimeric antigen receptor (CAR) T-cell therapy, BCB-276, has received the regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for treating diffuse intrinsic pontine glioma (DIPG).

FDA to review Regenxbio’s gene therapy BLA for Hunter syndrome

Regenxbio has announced the acceptance of its biologics license application (BLA) by the US Food and Drug Administration (FDA) of its gene therapy clemidsogene lanparvovec (RGX-121) for review to potentially treat Mucopolysaccharidosis II (MPS II), also referred as Hunter syndrome.

InnoCare’s Mesutoclax obtains breakthrough therapy designation in China

InnoCare’s B-cell lymphoma-2 (BCL2) inhibitor, Mesutoclax (ICP-248), has secured breakthrough therapy designation (BTD) from the China National Medical Products Administration’s (NMPA) Center for Drug Evaluation (CDE).

FDA grants fast track status to Alterity’s multiple system atrophy treatment

The US Food and Drug Administration (FDA) has granted fast track designation to Alterity Therapeutics' oral agent ATH434 for treating multiple system atrophy (MSA).

CHMP endorses Roche’s breast cancer therapy for administration out of clinical settings

Roche has received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for updating the European Union (EU) label of Phesgo.

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.