Regeneron’s Pasatru wins FDA approval in ultra-rare ‘stone man disease’
Pasatru demonstrated strong efficacy in a Phase III trial, helping patients reduce lesions and flare-ups.
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Kynexis pockets €97m to advance cognition-focused schizophrenia drug
Kynexis will now prepare for registrational studies on lead asset, KYN-5356 in cognitive impairment associated with schizophrenia (CIAS).
FDA grants accelerated approval to Ultragenyx’s Genglycos gene therapy
Ultragenyx Pharmaceutical has received accelerated approval from the FDA for its gene therapy, Genglycos, to treat GSDIa.
SandboxAQ introduces AQPotency for virtual drug screening
SandboxAQ has introduced AQPotency, a LQM designed to virtually screen and predict the potential of drug candidates without the need for a solved protein structure.
BioMarin spends up to $490m for Alesta’s bone disease candidate
The deal provides BioMarin with a potential rival to AstraZeneca’s blockbuster Strensiq in hypophosphatasia.
Trump expected to nominate Heidi Overton as new FDA commissioner
Overton could take the helm at the FDA during a tumultuous period for the agency, defined by several resignations, firings and redundancies.
Andelyn Biosciences to develop GM2 gangliosidosis therapy
Andelyn Biosciences is set to develop and manufacture a gene therapy known as AAV9-GM2, for Queen’s University to treat GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases.
BeOne Medicines and partners renew Brukinsa access programme for CLL
BeOne Medicines, the BeOne Care Foundation, and The Max Foundation have extended their Brukinsa access programme for CLL until 2028.
Reformed pharmaceutical regulation across India’s drug landscape
One of the major changes made to India’s primary legal framework for drug price regulation affects the pricing of essential drugs.
Updated UK guidance for finerenone introduces new treatment for HF patients
New guidance from NICE provide a framework for UK care providers using finerenone to manage the treatment of certain types of heart failure.