UK – NICE recommends Biomarin’s Vimizim for patients with rare life-limiting metabolic disorder

The National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending elosulfase alfa for routine use in the NHS, treating...

UK – Previously untreatable lung cancer patients offered new therapy

The National Institute for Health and Care Excellence (NICE) has published a final appraisal document recommending Amgen’s Lumykras (sotorasib) for use within the Cancer...
FDA: 2019 Continues Uptick in Orphan Drug Approvals

USA – Study: One-fifth of recent FDA approved drugs not authorized, reimbursed in other...

More than one-fifth of drugs recently approved by the US Food and Drug Administration (FDA) were not approved or recommended for public reimbursement in...

Europe – EMA Q&A addresses submission of data elements for raw data pilot

The European Medicines Agency (EMA) this week issued a question-and-answer guidance to address sponsors’ questions on its pilot testing the review of raw clinical...

USA – FDA draft guidance on controlled correspondence reflects GDUFA III commitments

The US Food and Drug Administration (FDA) on Wednesday issued a draft guidance to assist generic drug manufacturers in requesting information from the agency...

USA – Industry groups seek changes to nonprescription drug access proposal

Drugmakers, pharmacists and others are calling for flexibility in the US Food and Drug Administration’s (FDA) proposed rule on requirements for nonprescription drug products...

USA – Global regulators discuss how they are adapting to AI, real-world data in...

Regulators across the globe have been preparing for the arrival of new artificial intelligence (AI) technologies and advances in real-world data (RWD) they say...
Advice for Management of Clinical trials in relation to Coronavirus

UK – SKYCovion COVID-19 vaccine authorised by MHRA

SKYCovion, the COVID-19 vaccine developed by SK Chemicals, has today been authorised by the Medicines and Healthcare products Regulatory Agency (MHRA). It becomes the 8th...

Europe – Convergence: EMA official urges transition of existing clinical trials to CTIS portal

Sponsors should plan to transition existing clinical trials into the new Clinical Trial Information System (CTIS) portal by 30 January 2025, Anabela Marcal, of...

UK – Recommendation from NICE for PTC Therapeutics gene therapy

Eladocagene exuparvovec, the only gene therapy for children with the ultra-rare genetic disorder, aromatic L-amino acid decarboxylase (AADC) deficiency, has been recommended by the...