Blog
RSS FeedPosted by Michael Wonder on 31 Aug 2025
FDA approval of Leqembi subcutaneous formulation charts path to combination therapies for Alzheimer's disease
29 August 2025 - Subcutaneous delivery increases accessibility and introduces potential for at home administration, following model of diabetes and ...
Posted by Michael Wonder on 29 Nov 2020
First jabs could take place within days in the UK
29 November 2020 - A COVID-19 vaccine could be rolled out in the UK within days, with reports approval of ...
Posted by Michael Wonder on 29 Nov 2020
PBS support for Australians living with leukaemia
28 November 2020 - Patients living with one of Australia’s most common forms of leukaemia are set to benefit from expanded ...
Posted by Michael Wonder on 28 Nov 2020
Alnylam announces innovative value-based agreement framework for Oxlumo (lumasiran) to accelerate access for patients with primary hyperoxaluria type 1 and deliver ultra-rare orphan disease pricing solutions to U.S. payers
24 November 2020 - Expedited access to Oxlumo aims to support children and adults living with PH1 who face inevitable disease ...
Posted by Michael Wonder on 28 Nov 2020
Vertex announces European Commission approval for Symkevi (tezacaftor/ivacaftor) with Kalydeco (ivacaftor) for eligible children with cystic fibrosis ages 6-11 years
27 November 2020 - The combination therapy is a new treatment option for CF patients who are homozygous for F508del. ...
Posted by Michael Wonder on 28 Nov 2020
Polaryx Therapeutics receives both rare paediatric disease and orphan drug designations for the treatment of GM2 gangliosidosis with PLX-300
25 November 2020 - Polaryx Therapeutics announced today that it has received from the U.S. FDA both rare paediatric disease and ...
Posted by Michael Wonder on 28 Nov 2020
FDA approves first treatment for weight management for people with certain rare genetic conditions
27 November 2020 - The U.S. FDA has approved Imcivree (setmelanotide) for chronic weight management (weight loss and weight maintenance for ...
Posted by Michael Wonder on 28 Nov 2020
Protalix BioTherapeutics and Chiesi Global Rare Diseases announce extension of PDUFA date for pegunigalsidase alfa for the proposed treatment of Fabry disease
27 November 2020 - Protalix BioTherapeutics today announced that the U.S. FDA has extended the Prescription Drug User Fee Act (PDUFA) ...
Posted by Michael Wonder on 27 Nov 2020
Global regulators urge continuation of COVID-19 vaccine trials for longer-term safety and efficacy follow-up
27 November 2020 - EMA has endorsed a statement by the International Coalition of Medicines Regulatory Authorities that urges all stakeholders, ...
Posted by Michael Wonder on 27 Nov 2020
Dr Crawshaw takes over as PHARMAC’s medical director after COVID challenges in the UK
27 November 2020 - PHARMAC is pleased to announce that Dr Shirley Crawshaw, seven months after she was appointed, has arrived ...
Posted by Michael Wonder on 27 Nov 2020
Proposal for funded multiple sclerosis treatments
27 November 2020 - PHARMAC is proposing some changes to the way that multiple sclerosis treatments are funded, as well ...
Posted by Michael Wonder on 26 Nov 2020
TGA announces approval of new medicine from ASX listed company
26 November 2020 - Afamelanotide acetate (Scenesse) was approved on 22 October 2020. ...
Posted by Michael Wonder on 26 Nov 2020
After admitting mistake, AstraZeneca faces difficult questions about its vaccine
25 November 2020 - Some trial participants only got a partial dose of AstraZeneca’s vaccine. Experts said the company’s spotty disclosures ...
Posted by Michael Wonder on 26 Nov 2020
Bowel disease patients to march on PHARMAC and Parliament to present 30,000+ signature petition
27 November 2020 - On 2 December 2020, Crohn’s and Colitis NZ will lead a march of patients, supporters and medical ...
Posted by Michael Wonder on 26 Nov 2020
Takhzyro is not included in the high-cost protection
26 November 2020 - Takhzyro (lanadelumab) is a subcutaneous injection intended for routine prevention of recurrent hereditary seizures, angioedema in patients ...
Posted by Michael Wonder on 26 Nov 2020
European Medicines Agency validates application for tepotinib for the treatment of advanced NSCLC with METex14 skipping alterations
26 November 2020 - Merck today announced that the EMA has validated for review, the application for tepotinib for the ...