The US Food and Drug Administration (FDA), a division within the US Department of Health and Human Services (HHS), has announced the Rare Disease Framework Initiative to accelerate the development of drugs and biologics for rare diseases. According to the regulator, the initiative will develop an optimised,… Read More
The Medicines and Healthcare products Regulatory Agency (MHRA) has published its 2030 Strategy, setting out ambitious plans to translate scientific and technological innovation into better health outcomes. The regulator says that the commitments will enable it to keep pace with rapid advances in science, technology and personalised… Read More
The US Food and Drug Administration (FDA) has given the go-ahead to Genentech’s Gazyva (obinutuzumab) in adult and paediatric patients two years of age and older. It is approved to reduce the risk of relapse in patients with frequently relapsing or steroid dependent childhood-onset idiopathic nephrotic syndrome… Read More
The European Commission (EC) has granted marketing authorisation under exceptional circumstances for Nezglyal (leriglitazone), an orally bioavailable, brain penetrating, selective PPAR gamma agonist, as a treatment for male cALD patients. It is approved in patients aged two to 12 years with Gadolinium (Gd)-negative brain lesions and is… Read More
AstraZeneca’s fixed-dose triple-combination Trixeo Aerosphere (budesonide/glycopyrronium/formoterol fumarate dihydrate or BGF) has been approved in the European Union (EU) as a maintenance treatment for asthma. It is approved in patients 12 years of age and older who are not adequately controlled by a combination of a medium dose… Read More
MaaT Pharma has received a negative trend vote from the Committee for Medicinal Products for Human Use (CHMP) for MaaT013 (Xervyteg) following re-examination for its conditional marketing authorisation application. The committee’s concerns relate primarily to the attribution of clinical benefit and safety in the absence of a… Read More
The US Food and Drug Administration (FDA) has approved Ionis Pharmaceuticals’ Zanvastro (zilganersen) for the treatment of Alexander disease (AxD) in paediatric and adult patients. It is the first and only disease modifying treatment for AxD, an ultra-rare, progressive and often fatal neurological disorder that can affect… Read More
The US Food and Drug Administration (FDA) has approved Lilly’s Mounjaro (tirzepatide), a dual GIP and GLP-1 hormone receptor agonist, to lower the risk of major adverse cardiovascular (CV) events (MACE) in adults with type 2 diabetes. The drug is already approved as an adjunct to diet… Read More
The US Food and Drug Administration (FDA) has approved Revolution Medicines’ Rasonque (daraxonrasib) once-daily oral tablets, for the treatment of adults with metastatic pancreatic adenocarcinoma (PDAC). It is approved in patients who have received at least one prior systemic therapy or who are not candidates for multiagent… Read More
Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved GSK’s Hibsago (bepirovirsen), an antisense oligonucleotide (ASO), as a functional cure for chronic hepatitis B (CHB) virus infection. It is approved in adult patients who have received at least six months of prior nucleos(t)ide analogue therapy and… Read More
The European Commission (EC) has approved Johnson & Johnson’s Tecvayli (teclistamab) plus daratumumab for relapsed/refractory multiple myeloma, offering a potential new standard of care. The indication extension in combination with daratumumab is approved for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) who have… Read More
In June 2026, China’s National Medical Products Administration cleared satricabtagene autoleucel (satri-cel), CARsgen’s Claudin18.2 CAR-T, for advanced gastric cancer. It became the world’s first CAR-T cell therapy approved for a solid tumour. The science is genuinely impressive. But the geography is the story. Satri-cel is not a Chinese… Read More
The US Food and Drug Administration (FDA) granted accelerated approval for Ultragenyx Pharmaceutical’s Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and paediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). Dr Eric Crombez, Chief Medical Officer at Ultragenyx, commented: “As our… Read More
AC Immune has received Fast Track designation and clearance of its Investigational New Drug (IND) application from the US Food and Drug Administration (FDA) for its anti-alpha-synuclein (a-syn) active immunotherapy candidate, ACI-7104, for the treatment of early-stage Parkinson’s disease (PD). ACI-7104 is designed to induce… Read More
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has authorised orforglipron (Foundayo), making it the first country in Europe to approve the GLP-1 tablet for both weight management and type 2 diabetes. Alongside a reduced-calorie diet and increased physical activity, orforglipron is authorised for weight loss and… Read More
Reckitt’s Mucinex 12HR Cold & Fever Multi-Symptom is the first US Food and Drug Administration (FDA)-approved over-the-counter (OTC) drug in the cold and flu category in 14 years. Mucinex that treats fever, cough, chest congestion and body pain for 12 hours. According to The Centers… Read More
