Blog
RSS FeedPosted by Michael Wonder on 01 Mar 2026
US FDA approves BioMarin’s Palynziq (pegvaliase-pqpz) for adolescents 12 years of age and older with phenylketonuria
27 February 2026 - BioMarin today announced that the US FDA has approved the company's supplemental biologics license application for Palynziq ...
Posted by Michael Wonder on 24 Feb 2026
US FDA has granted accelerated approval of Loargys (pegzilarginase-nbln) for the treatment of hyperargininemia in patients 2 years and older with arginase 1 deficiency
23 February 2026 - Immedica Pharma today announced that the US FDA has granted accelerated approval of Loargys (pegzilarginase-nbln), an ...
Posted by Michael Wonder on 24 Feb 2026
FDA launches framework for accelerating development of individualised therapies for ultra-rare diseases
23 February 2026 - The U.S. FDA today issued draft guidance for sponsors seeking approval for targeted individualised therapies by ...
Posted by Michael Wonder on 18 Feb 2026
NICE recommends first ever disease modifying treatment for ARG1 deficiency
17 February 2026 - NICE has recommended the first disease modifying treatment for arginase 1 deficiency (ARG1-D), an ultra-rare, inherited and ...
Posted by Michael Wonder on 01 Feb 2026
Ultragenyx resubmits biologics license application for UX111 AAV gene therapy to treat Sanfilippo syndrome type A (MPS IIIA) to US FDA
30 January 2026 - Company expects up to six month review period per FDA guidelines. ...
Posted by Michael Wonder on 28 Nov 2025
PHARMAC proposes funding for rare disorder treatment
26 November 2025 - PHARMAC is proposing to fund nitisinone for people with the rare disorders - tyrosinaemia type 1 ...
Posted by Michael Wonder on 09 Nov 2025
Rhythm Pharmaceuticals announces public reimbursement for Imcivree (setmelanotide) in Canada in five provinces and under the Federal Non-Insured Health Benefits Program
5 November 2025 - Rhythm Pharmaceuticals today announced that it has entered into Product Listing Agreements in the provinces of Ontario, ...
Posted by Michael Wonder on 03 Nov 2025
US FDA approves Kygevvi (doxecitine and doxribtimine), the first and only treatment for adults and children living with thymidine kinase 2 deficiency
3 November 2025 - UCB today announced that Kygevvi has been granted approval by the US FDA for the treatment ...
Posted 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 ...