Blog
RSS FeedPosted by Michael Wonder on 29 Jun 2022
Ipsen announces U.S. FDA priority review for palovarotene new drug application in patients with fibrodysplasia ossificans progressiva following resubmission
29 June 2022 - Ipsen today announced that the U.S. FDA has accepted for priority review its resubmitted new drug ...
Posted by Michael Wonder on 28 Jun 2022
Nexviadyme (avalglucosidase alfa) approved by European Commission as a potential new standard of care for the treatment of Pompe disease
28 June 2022 - Approved for the treatment of the full spectrum of both late-onset Pompe disease and infantile-onset Pompe disease. ...
Posted by Michael Wonder on 27 Jun 2022
TLV receives assignments for medicines for rare diseases
27 June 2022 - The Government has commissioned the Swedish Dental and Pharmaceutical Benefits Agency (TLV) to analyse and make ...
Posted by Michael Wonder on 16 Jun 2022
Rhythm Pharmaceuticals announces FDA approval of Imcivree (setmelanotide) for use in patients with Bardet-Biedl syndrome
16 June 2022 - Approval based on Phase 3 trial results that demonstrated statistically significant reductions in weight and hunger in ...
Posted by Michael Wonder on 15 Jun 2022
EFPIA-EURORDIS joint statement on patient access to medicines for rare diseases
15 June 2022 - Today marks the publication of the EFPIA-EURORDIS joint statement on patient access to medicines for rare diseases. ...
Posted by Michael Wonder on 01 Jun 2022
Cancer, rare disorder advocates call for more radical change after damning PHARMAC review
2 June 2022 - Radical action is needed urgently to tackle the delays patients face in getting the latest drugs for ...
Posted by Michael Wonder on 21 May 2022
CHMP recommends approval of Xenpozyme (olipudase alfa), the first and only treatment for ASMD
20 May 2022 - Recommendation based on positive results from two clinical trials in which Xenpozyme provided improvement across multiple non-CNS ...
Posted by Michael Wonder on 05 May 2022
Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over
5 May 2022 - NICE has published evidence based recommendations on the use of selumetinib (Koselugo) for the treatment of children ...
Posted by Michael Wonder on 04 May 2022
Pliant Therapeutics receives FDA fast track designation for PLN-74809 for the treatment of idiopathic pulmonary fibrosis
3 May 2022 - Pliant Therapeutics announced today that PLN-74809, its oral, dual-selective αvß6/αvß1 integrin inhibitor, has received fast track ...
Posted by Michael Wonder on 02 May 2022
Orphalan announces FDA approval of Cuvrior for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine
2 May 2022 - Orphalan announces FDA approval of Cuvrior for the treatment of adult patients with stable Wilson’s disease ...
Posted by Michael Wonder on 21 Apr 2022
Ontario family forced to fight for life-saving drug highlights need for national rare disease strategy
21 April 2022 - Beth Vanstone doesn’t want anyone else to have to fight for access to life saving medications. ...
Posted by Michael Wonder on 07 Apr 2022
ICER publishes white paper evaluating reforms to orphan drug development, pricing and coverage
7 April 2022 - The white paper presents an analysis of the potential risks and benefits of reforms seeking to ...
Posted by Michael Wonder on 31 Mar 2022
Statement from Rare Disorders NZ re Minister Little's comments on PHARMAC funding
31 March 2022 - Rare Disorders NZ is extremely frustrated to hear the Minister of Health this week dismiss the call ...
Posted by Michael Wonder on 01 Mar 2022
ABPI response to England Rare Diseases Action Plan
28 February 2022 - England's Rare Disease Action plan is published today with details of how to improve diagnosis, care and ...
Posted by Michael Wonder on 25 Feb 2022
Protalix BioTherapeutics and Chiesi Global Rare Diseases announce the submission of a marketing authorisation application to the European Medicines Agency for PRX-102 for the treatment of Fabry disease
24 February 2022 - Protalix BioTherapeutics and Chiesi Global Rare Diseases today announced the submission of a marketing authorisation application via ...