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RSS FeedPosted by Michael Wonder on 13 May 2024
PHARMAC shares the agenda items for the upcoming Rare Disorders Committee meeting (May 2024)
13 May 2024 - PHARMAC is sharing what medicine applications will be considered at the Rare Disorders Advisory Committee meeting ...
Posted by Michael Wonder on 26 Apr 2024
FDA grants Medivir´s MIV-711 rare paediatric disease designation and orphan drug designation for the treatment of Legg-Calvé-Perthes Disease
25 April 2024 - Medivir announced today that its selective cathepsin K inhibitor, MIV-711, has been granted rare paediatric disease designation ...
Posted by Michael Wonder on 08 Apr 2024
Stealth BioTherapeutics announces FDA acceptance of new drug application for elamipretide for the treatment of Barth syndrome
8 April 2024 - If approved, elamipretide would become the first approved therapy for Barth syndrome. ...
Posted by Michael Wonder on 29 Feb 2024
Collaborating to help improve access to drugs for rare diseases
29 February 2024 - On Rare Disease Day — a day when the international community raises awareness about rare conditions ...
Posted by Michael Wonder on 29 Feb 2024
Today is Rare Disease Day
29 February 2024 - PHARMAC understands that people living with rare disorders face many challenges, including access to health care ...
Posted by Michael Wonder on 13 Feb 2024
European Commission approves first CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel), for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
13 February 2024 - Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent ...
Posted by Michael Wonder on 07 Feb 2024
Rhythm Pharmaceuticals announces positive reimbursement decision in Italy for Imcivree (setmelanotide) for the treatment of obesity and control of hunger in Bardet-Biedl syndrome
7 February 2024 - Rhythm Pharmaceuticals today announced that the Italian Medicine Agency (AIFA) approved reimbursement (Gazzetta Ufficiale) for Imcivree (setmelanotide) ...
Posted by Michael Wonder on 18 Jan 2024
New orphan drug for a sub-type of acute myeloid leukaemia: G-BA sees significant additional benefit
18 January 2024 - Since July 2023, there has been one in Germany for patients who suffer from acute myeloid leukaemia ...
Posted by Michael Wonder on 15 Jan 2024
Sebelipase alfa for the treatment of patients with Wolman disease (final guidance)
10 January 2023 - Sebelipase alfa is recommended as an option for the treatment of patients with Wolman disease only if ...
Posted by Michael Wonder on 09 Jan 2024
Orphan drug label expansions: analysis of subsequent rare and common indication approvals
8 January 2024 - We found that 491 novel orphan drugs were approved between 1990 and 2022. ...
Posted by Michael Wonder on 13 Dec 2023
Velmanase alfa for the treatment of patients with alfa mannosidosis
13 December 2023 - NICE has published final evidence-based recommendations on the use of velmanase alfa (Lamzede) for treatment of ...
Posted by Michael Wonder on 27 Nov 2023
Cost of exempting sole orphan drugs from Medicare negotiation
27 November 2023 - This cross-sectional study identified 25 “sole orphan” drugs qualifying for exemption from Medicare price negotiation. ...
Posted by Michael Wonder on 13 Nov 2023
PHARMAC calls for applications for medicines for rare disorders
13 November 2023 - PHARMAC is calling for applications for medicines to treat rare disorders. ...
Posted by Michael Wonder on 04 Nov 2023
Elexacaftor with tezacaftor and ivacaftor, tezacaftor with ivacaftor and lumacaftor with ivacaftor for the treatment of patients with cystic fibrosis
3 November 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
Posted by Michael Wonder on 01 Nov 2023
X4 Pharmaceuticals announces FDA acceptance with priority review of US NDA for mavorixafor in WHIM syndrome
31 October 2023 - FDA sets a PDUFA target action date of 30 April 2024. ...