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 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 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
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
Investment in rare disease biotechs has increased following renewal of the FDA’s Priority Review Voucher (PRV) programme, according to SynaptixBio, but the United States remains dominant compared to the UK. In February 2026, the PRV programme was signed into law and will remain in place until reviewed again in September… 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
The US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee has voted that available evidence does not support the effectiveness of Capricor Therapeutics’ Deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). DMD is a rare and life-threatening disease for… Read More
The US Food and Drug Administration (FDA) has granted Fast Track Designation for HAYA Therapeutics’ HTX-001 for the treatment of symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM). The first-in-class investigational long non-coding RNA (lncRNA)-targeting therapy is in a Phase Ia/b trial, with the first cohort dosed this past May. HTX-001 is a… Read More
DDW Editor Reece Armstrong speaks to David Crean, Chief Business Officer of biopharmaceutical company MediciNova, about recent turmoil within the FDA and what the pharma sector should expect from the agency going forward. It’s been a volatile couple of years for the United States. President Trump’s second term… Read More
The US Food and Drug Administration (FDA) has expanded the use of Casgevy (exagamglogene autotemcel) for the treatment of people ages two years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). The approval makes Casgevy the first… Read More
The US Food and Drug Administration (FDA) has approved Lumvoa (veligrotug-vvze) as a treatment for thyroid eye disease (TED). TED is a rare, debilitating autoimmune disease characterised by inflammation and tissue remodelling around and behind the eyes, which can lead to proptosis, diplopia, pain and vision impairment. Read More
The US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for Beam Therapeutics’ BEAM-304 for the treatment of phenylketonuria (PKU). PKU is a rare, inherited metabolic disorder that results in toxic accumulation of phenylalanine (Phe), leading to serious neurologic and neurocognitive impairments… Read More
Bruno Quinney speaks to Adrian Kells, Senior Vice President and Integrated Product Team Lead PD at AskBio, about the company’s gene therapy programme and pioneering treatment for Parkinson’s. The drug discovery landscape for Parkinson’s disease is becoming one of the most exciting frontiers in the industry. It is unsurprising to… Read More
GSK’s momelotinib, a JAK inhibitor with a differentiated mechanism of action, has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) syndrome. VEXAS syndrome is a clonal… Read More
