Blog
RSS FeedPosted by Michael Wonder on 29 Sep 2023
FDA launches pilot program to help further accelerate development of rare disease therapies
29 September 2023 - Today, the US FDA is taking steps to help further accelerate the development of novel drug and ...
Posted by Michael Wonder on 19 Aug 2023
Veopoz (pozelimab-bbfg) receives FDA approval as the first treatment for children and adults with CHAPLE disease
18 August 2023 - Approval represents tenth FDA approved medicine invented by Regeneron. ...
Posted by Michael Wonder on 15 Aug 2023
Cipaglucosidase alfa and miglustat for the treatment of patients with late-onset Pompe disease
15 August 2023 - NICE has published evidence-based recommendations on the use of cipaglucosidase alfa (Pombiliti) with miglustat (Opfolda) for ...
Posted by Michael Wonder on 10 Aug 2023
PHARMAC’s Rare Disorders Advisory Committee makes new recommendations for funding
10 August 2023 - PHARMAC has published the record of the March Rare Disorders Advisory Committee meeting, sharing details on ...
Posted by Michael Wonder on 04 Aug 2023
Setmelanotide acetate for the treatment of people with obesity and hyperphagia associated with Bardet-Biedl syndrome
3 August 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
Posted by Michael Wonder on 13 May 2023
Orphan drugs: early generation of evidence urgently needed
10 May 2023 - A team led by IQWiG authors, together with Rita Banzi from the Italian Mario Negri Institute, analysed ...
Posted by Michael Wonder on 11 May 2023
FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis
9 May 2023 - The objective of this study was to analyse the US FDA approval, trials, unmet needs, benefit, and ...
Posted by Michael Wonder on 10 May 2023
Reforming EU and national orphan drug regulations to improve outcomes for patients with rare diseases
9 May 2023 - In Europe, the number of approvals for new drugs targeting rare diseases (orphan drugs) has risen ...
Posted by Michael Wonder on 11 Apr 2023
New life saving medicines
11 April 2023 - The Australian Government has added the two medicines, Kanuma (sebelipase alfa) and Cerdelga (eliglustat) to the Life ...
Posted by Michael Wonder on 02 Apr 2023
Survey on patients' access to medicines for the treatment of rare health conditions
31 March 2023 - The TLV has submitted an interim report to the government for the task of proposing how ...
Posted by Michael Wonder on 28 Mar 2023
Amicus Therapeutics announces European Commission approval for Pombiliti in patients with late-onset Pompe disease
27 March 2023 - CHMP opinion for miglustat, the oral enzyme stabiliser component of AT-GAA, on track for 2Q, 2023. ...
Posted by Michael Wonder on 23 Mar 2023
Investments to support access to drugs for rare diseases
22 March 2023 - Today, the Government announced a total investment of up to $1.5 billion over three years in ...
Posted by Michael Wonder on 23 Mar 2023
Government of Canada improves access to affordable and effective drugs for rare diseases
22 March 2023 - Today, the Honourable Jean-Yves Duclos, Minister of Health, announced measures in support of the first-ever National Strategy ...
Posted by Michael Wonder on 11 Mar 2023
Acadia Pharmaceuticals announces US FDA approval of Daybue (trofinetide) for the treatment of Rett syndrome in adult and paediatric patients two years of age and older
10 March 2023 - Company expects Daybue to be available by the end of April 2023. ...
Posted by Michael Wonder on 08 Mar 2023
Genethon given PRIME status by EMA for gene therapy to treat Crigler-Najjar syndrome, a rare liver disease
7 March 2023 - Genethon today announced that the EMA has granted PRIME (Priority Medicines) status to the gene therapy, ...