The US Food and Drug Administration (FDA) has granted accelerated approval for Kresladi (marnetegragene autotemcel), an autologous haematopoietic stem cell-based gene therapy. It is indicated for the treatment of paediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human… Read More
The US Food and Drug Administration (FDA) has granted accelerated approval for Avlayah (tividenofusp alfa-eknm), the first FDA-approved biologic specifically designed to cross the blood-brain barrier. Avlayah is an enzyme replacement therapy indicated for the treatment of neurologic manifestations of Hunter syndrome (mucopolysaccharidosis type II, or MPS… Read More
The European Medicines Agency (EMA) has launched three major new features of PRIME, the Agency’s scheme to enhance support for the development of medicines targeting an unmet medical need. After the completion of a two-year pilot, the EMA has integrated these additional tools to support continued scientific… Read More
Aldeyra Therapeutics has received a Complete Response Letter (CRL) from the US Food and Drug Administration (FDA) for the New Drug Application (NDA) of reproxalap, an investigational drug candidate, for the treatment of dry eye disease. The CRL states that there is “a lack of substantial evidence… Read More
Australia’s Therapeutic Goods Administration (TGA) has approved a first-in-human trial of a T-cell therapy in multiple myeloma. The therapy, developed by HaemaLogiX in collaboration with the Peter MacCallum Cancer Centre (Peter Mac), is a KMA.CAR T-cell immunotherapy. The novel autologous CAR-T cell therapy targets Kappa Myeloma Antigen (KMA), a receptor… Read More
The US Food and Drug Administration (FDA) has granted Fast Track Designation to a treatment for the rare paediatric disease late-infantile neuronal ceroid lipofuscinosis (LINCL/CLN2 disease). PLX-200, developed by biotechnology company Polaryx Therapeutics, will now undergo an expedited review with the potential to address the paediatric disease with unmet… Read More
The US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for hypertension drug lorundrostat. Biopharmaceutical company Mineralys Therapeutics announced the NDA for lorundrostat was accepted for the treatment of adult patients with hypertension in combination with other antihypertensive drugs. The FDA has assigned a Prescription Drug User Fee Act… Read More
Novo Nordisk has received a Warning Letter from the US Food and Drug Administration (FDA) related to violations of postmarketing adverse drug experience (PADE) regulations. The letter relates to an inspection that took place in early 2025, in which the FDA found serious violations of PADE reporting… Read More
The US Food and Drug Administration (FDA) has granted Qualified Infectious Disease Product (QIDP) designation to a novel treatment of Gram-negative bacterial infections. CTX-187, developed by immunotherapy company Centauri Therapeutics, is a novel immunotherapeutic approach to treat Gram-negative bacterial infections. It is hoped the therapeutic, currently in preclinical… Read More
The European Commission (EC) has approved Zynyz (retifanlimab) in combination with carboplatin and paclitaxel for the first-line treatment of adult patients with metastatic or with inoperable locally recurrent squamous cell carcinoma of the anal canal (SCAC). Rretifanlimab is the first systemic treatment for adult patients with advanced… Read More
The European Commission has approved an indication extension for Akeega (niraparib and abiraterone acetate dual action tablet) with prednisone or prednisolone (AAP) in combination with androgen deprivation therapy (ADT). The regimen is now also approved for the treatment of patients with metastatic hormone-sensitive prostate cancer (mHSPC) and… Read More
The US Food and Drug Administration (FDA) has approved a clinical study of Pentixapharm’s CXCR4-targeted theranostic programme in haemato-oncology, following a 30-day safety review of two Investigational New Drug (IND) applications. The two IND submissions support the single PENTHERA Phase I/II protocol, which evaluates the combined use… Read More
The US Food and Drug Administration (FDA) has removed the clinical hold on the Investigational New Drug application (IND) for the MAGNITUDE Phase III clinical trial of nexiguran ziclumeran (nex-z) for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The clinical holds on the INDs for MAGNITUDE and… Read More
The European Medicines Agency (EMA)’s Committee for Medicinal Products (CHMP) has adopted a positive opinion recommending marketing authorisation for a chronic spontaneous urticaria (CSU) treatment. The opinion means remibrutinib, a highly selective oral BTKi developed by Novartis, could be the first targeted therapy approved for CSU in Europe. … Read More
The US Food & Drug Administration (FDA) has granted approval for Yuviwel (navepegritide) under its Accelerated Approval Program. Navepegritide is the only once-weekly treatment indicated to increase linear growth in children one years of age and older with achondroplasia with open epiphyses and the only one to… Read More
The US Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to TSRA-196, Tessera Therapeutics’ lead in vivo gene editing programme. TSRA-196 is being jointly developed with Regeneron for the treatment of adults with alpha-1 antitrypsin deficiency (AATD) who are homozygous… Read More
