Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 22 Feb 2022
NICE recommends Albireo’s Bylvay (odevixibat) for all PFIC types
22 February 2022 - Positive NICE review completed in less than six months post MHRA approval. ...
Posted by Michael Wonder on 24 Jan 2022
Codexis announces FDA orphan drug and rare paediatric disease designations for CDX-6512 for the treatment of homocystinuria
24 January 2022 - Codexis today announced that the U.S. FDA has granted the company orphan drug designation for CDX-6512 for ...
Posted by Michael Wonder on 13 Jan 2022
Orphan drugs: privilege of "fictitious" additional benefit not justified
12 January 2022 - Drugs for orphan diseases should also go through a regular benefit assessment procedure when they enter the ...
Posted by Michael Wonder on 05 Jan 2022
U.S. FDA approves Immix Biopharma rare paediatric disease designation for IMX-110 as a treatment for life-threatening paediatric cancer
3 January 2021 - Immix Biopharma announced today that the U.S. FDA has granted rare paediatric disease designation for IMX-110 for ...
Posted by Michael Wonder on 14 Dec 2021
New hope offered to rare disease patients
14 December 2021 - Early this month, the world's first therapy to treat spinal muscular atrophy, a rare genetic disorder ...
Posted by Michael Wonder on 09 Dec 2021
Canada's national strategy for drugs for rare diseases needs a holistic approach
8 December 2021 - International report summarises drugs for rare disease best practices and recommendations. ...
Posted by Michael Wonder on 08 Dec 2021
FDA takes new steps aimed at advancing development of individualised medicines to treat genetic diseases
7 December 2021 - Today, the FDA is issuing a draft guidance to provide recommendations for managing the administration of individualised ...
Posted by Michael Wonder on 08 Dec 2021
Fortress Biotech, Cyprium Therapeutics and Sentynl Therapeutics announce the initiation of rolling submission of a new drug application for CUTX-101, copper histidinate, for treatment of Menkes disease
7 December 2021 - Cyprium Therapeutics with support from its licensing partner Sentynl Therapeutics today announced the initiation of a ...