Blog
RSS FeedPosted by Michael Wonder on 21 Dec 2024
Ultragenyx submits biologics license application to the US FDA for UX111 AAV gene therapy for the treatment of Sanfilippo syndrome type A
19 December 2024 - If approved, UX111 would be the first approved therapy in the US for Sanfilippo syndrome type ...
Posted by Michael Wonder on 14 Dec 2024
FDA approves new treatment for congenital adrenal hyperplasia
13 December 2024 - Today, the US FDA approved Crenessity (crinecerfont) to be used together with glucocorticoids to control androgen levels ...
Posted by Michael Wonder on 10 Dec 2024
Chiesi Global Rare Diseases and Protalix BioTherapeutics announce validation of variation submission by EMA for pegunigalsidase alfa
9 December 2024 - Application to label a less frequent dosing regimen at a dose of 2 mg/kg body weight ...
Posted by Michael Wonder on 06 Dec 2024
Jessica Lawrence’s meds cost her family $75k a year because they’re not listed on the PBS
6 November 2024 - Medications that are subsidised for cancer sufferers are critical to Jess Lawrence’s wellbeing, but because the 25-year-old ...
Posted by Michael Wonder on 06 Dec 2024
Government of Canada signs bilateral agreement with Alberta to improve access to drugs for rare diseases
5 December 2024 - Today, the Honourable Mark Holland, Canada's Minister of Health, and the Honourable Adriana LaGrange, Alberta's Minister ...
Posted by Michael Wonder on 05 Dec 2024
Results of health technology assessments of orphan drugs in Germany—lack of added benefit, evidence gaps, and persisting unmet medical needs
3 December 2024 - The number of orphan drug approvals has increased sharply in Europe. In Germany, all orphan drugs are ...
Posted by Michael Wonder on 21 Nov 2024
ImmuneSensor Therapeutics receives orphan drug and rare paediatric disease designations from the US FDA for cGAS inhibitor drug candidate, IMSB301, for the treatment of Aicardi Goutières syndrome
20 November 2024 - ImmuneSensor Therapeutics announced that the US FDA has granted both orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 24 Oct 2024
Canada’s Drug Agency announces recipients of funding to enhance rare disease registries
21 October 2024 - Canada’s Drug Agency is pleased to announce the successful applicants from our 2024 Request for Proposals (RFP) ...
Posted by Michael Wonder on 20 Sep 2024
FDA approves first treatment for Niemann-Pick disease, type C
20 September 2024 - Today, the US FDA approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type ...
Posted by Michael Wonder on 14 Aug 2024
FDA approves new drug for hypoparathyroidism, a rare disorder
9 August 2024 - The US FDA has approved Yorvipath (palopegteriparatide) injection for subcutaneous use in adults with hypoparathyroidism. ...
Posted by Michael Wonder on 03 Aug 2024
Drugs in rare paediatric disease priority review voucher program have similar revenue to brand drugs
2 August 2024 - Drugs issued rare paediatric disease priority review vouchers that were redeemed had revenues that were similar ...
Posted by Michael Wonder on 06 Jul 2024
Four decades of orphan drugs and priorities for the future
6 July 2024 - The Orphan Drug Act was enacted in the United States in 1983 in response to growing awareness ...
Posted by Michael Wonder on 25 May 2024
Issues, challenges and opportunities for economic evaluations of orphan drugs in rare diseases: an umbrella review
25 May 2024 - There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. ...
Posted by Michael Wonder on 23 May 2024
Setmelanotide acetate for the treatment of patients with obesity and hyperphagia in Bardet-Biedl syndrome (final guidance)
22 May 2024 - NICE has published final evidence-based recommendations on the use of setmelanotide acetate (Imcivree) for treating obesity ...
Posted 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 ...