Blog
RSS FeedPosted by Michael Wonder on 23 Oct 2025
New life saving medicine free for rare disease
22 October 2025 - Australians with the extremely rare and life-threatening inherited disease, acid sphingomyelinase deficiency (ASMD) will now have ...
Posted by Michael Wonder on 26 Sep 2025
HIRA calls for insurance overhaul to ensure access to high cost orphan drugs
26 September 2025 - The Government has proposed that a new health insurance reimbursement system must be designed to ensure ...
Posted by Michael Wonder on 01 Sep 2025
Care for people with rare and less common cancers ‘delayed and fragmented’, new report reveals
1 September 2025 - A national survey of nearly 2,500 people affected by cancer has found those with a rare ...
Posted by Michael Wonder on 21 Aug 2025
Rhythm Pharmaceuticals announces FDA acceptance of sNDA for setmelanotide in acquired hypothalamic obesity
20 August 2025 - US FDA accepts sNDA for filing with priority review; sets PDUFA goal date of 20 December 2025. ...
Posted by Michael Wonder on 25 Mar 2025
Government of Canada signs bilateral agreement with Quebec for Drugs for Rare Diseases
21 March 2025 - In Canada, one in 12 people live with a rare disease, and for most people affected, the ...
Posted by Michael Wonder on 13 Mar 2025
First licensed treatment for ultra-rare immune disorder recommended
13 March 2025 - Leniolisib is the first ever treatment for activated phosphoinositide 3-kinase delta syndrome licensed for use in the ...
Posted by Michael Wonder on 03 Mar 2025
Canada’s Drug Agency publishes new guidance for rare disease registries in Canada
28 February 2025 - On Rare Disease Day — a global event dedicated to raising awareness for the millions of ...
Posted by Michael Wonder on 03 Mar 2025
Supporting the development of drugs for rare diseases - the importance of regulatory transparency
1 March 2025 - Transparency regarding the information submitted to the FDA and the agency’s decision making could have far-reaching ...
Posted by Michael Wonder on 03 Mar 2025
Vertex receives CHMP positive opinion for expanded label for Kaftrio in combination with ivacaftor for people with cystic fibrosis to include rare mutations
28 February 2025 - If approved, approximately 4,000 people with cystic fibrosis in the European Union will be eligible for ...
Posted by Michael Wonder on 01 Feb 2025
High quality evidence for the treatment of very rare diseases: IQWiG is involved in the EU project RealiseD
29 January 2025 - Developing clinical studies for good evidence on very rare diseases in order to arrive at effective therapies ...
Posted by Michael Wonder on 28 Jan 2025
Drugs for rare diseases – Ontario agreement
24 January 2025 - Today, the Governments of Canada and the province of Ontario signed the National Strategy for Drugs ...
Posted by Michael Wonder on 09 Jan 2025
Denali Therapeutics announces US FDA breakthrough therapy designation granted to tividenofusp alfa for the treatment of Hunter syndrome
8 January 2025 - Denali Therapeutics today announced that the US FDA has granted breakthrough therapy designation for tividenofusp alfa (DNL310) ...
Posted by Michael Wonder on 22 Dec 2024
Vertex announces US FDA approval of Alyftrek, a once daily next in class CFTR modulator for the treatment of cystic fibrosis
20 December 2024 - In head to head clinical trials, Alyftrek was non-inferior on ppFEV1 and further decreased sweat chloride ...
Posted by Michael Wonder on 22 Dec 2024
Vertex announces US FDA approval for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include additional non-F508del Trikafta responsive variants
20 December 2024 - Approximately 300 more people with cystic fibrosis in the US are now eligible for a medicine ...
Posted 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 ...