Blog
RSS FeedPosted 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 ...
Posted 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 ...