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Sentynl secures US-first approval for rare Menkes disease
The US biopharma company can now offer a new treatment option for eligible paediatric patients with the genetic disease.
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The US Food and Drug Administration (FDA)
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Generic manufacturer safety records revealed by new consumer app
News organisation ProPublica’s Rx Inspector tool connects FDA information on factory location and regulatory records to individual generic drugs.
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FDA to encourage new manufacturing sites with its PreCheck programme
The scheme will provide more support and a streamlined application process to companies constructing new US facilities.
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First-in-class therapy approved for chronic skin disease
The biologic treatment targets a key driver of prurigo nodularis, with trial data demonstrating improved itch as well as skin nodules for patients with the condition.
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Regulating therapies for rare diseases – recent approvals
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
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FDA approves innovative engineered cell therapy
The accelerated approval by the US Food and Drug Administration (FDA) authorises the first engineered cell therapy for a solid tumour in the US.
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EC approves treatment for rare haematological disorder
The European Commission has approved Europe's first treatment for adults with indolent systemic mastocytosis, a rare haematological disorder.
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FDA approves first cell-based gene therapies for sickle cell disease
The US FDA has approved Vertex’s Casgevy and bluebird bio’s Lyfgenia, for one-time treatment of sickle cell disease in patients 12 years and older.
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First chikungunya vaccine approved
Future regulatory authorisation is expected in Europe, following the US Food and Drug Administration (FDA)’s accelerated approval of IXCHIQ®.
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FDA approves first Stelara biosimilar, Wezlana
The US FDA has approved Amgen’s Wezlana (ustekinumab-auub) as the first biosimilar to reference blockbuster drug Stelara (ustekinumab).
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Guidance on machine learning-enabled medical devices published
The Medicines and Healthcare products Regulatory Agency (MHRA), US Food and Drug Administration (FDA) and Health Canada have issued guidelines to reduce the regulatory burden for machine learning-enabled medical devices.
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Takeda ulcerative colitis biologic receives FDA approval
A new route of administration for a biologic to treat ulcerative colitis (UC) has been approved in the US.
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FDA approval for Janssen’s prostate cancer treatment
The US FDA approval of Akeega (niraparib and abiraterone acetate) is based on positive results from the Phase III MAGNITUDE study.
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FDA warning letters highlight CAPA concerns
Several FDA warning letters distributed to pharmaceutical manufacturers in 2023 have warned of numerous corrective and preventive action (CAPA) compliance concerns.
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EMA revises guidance on nitrosamine impurities
The European Medicines Agency (EMA) has updated its guidance on nitrosamine impurities in human medicinal products, amending Q&A 10 and adding three appendices.
