Blog
RSS FeedPosted 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. ...
Posted by Michael Wonder on 30 Oct 2023
Switzerland restricts reimbursement of orphan drugs
30 October 2023 - New rules for the reimbursement of orphan drugs and other unlisted medicines will become effective in Switzerland ...
Posted by Michael Wonder on 30 Oct 2023
Egetis announces EMA validation of marketing authorisation application for Emcitate for the treatment of MCT8 deficiency
27 October 2023 - Egetis Therapeutics today announced that its marketing authorisation application to the EMA for Emcitate (tiratricol) for ...
Posted by Michael Wonder on 27 Oct 2023
Catalyst Pharmaceuticals reports FDA approval of Agamree (vamorolone) for Duchenne muscular dystrophy granted to Santhera Pharmaceuticals
26 October 2023 - Catalyst expects to commercially launch Agamree in Q1, 2024. ...
Posted by Michael Wonder on 26 Oct 2023
Olipudase alfa for the treatment of patients with acid sphingomyelinase deficiency (Niemann-Pick disease type B and AB)
26 October 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
Posted by Michael Wonder on 24 Oct 2023
Ipsen updates on EU marketing authorisation application for odevixibat in Alagille syndrome
23 October 2023 - Ipsen plans to submit a new marketing authorisation application for the treatment of Alagille syndrome by the ...
Posted by Michael Wonder on 10 Oct 2023
Patient access to medicines for rare health conditions at sustainable costs can be strengthened
2 October 2023 - The Swedish Dental and Medicines Benefits Agency, TLV, today submits a report to the government on how ...