Blog
RSS FeedPosted 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7 December 2021 - Today, the FDA is issuing a draft guidance to provide recommendations for managing the administration of individualised ...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 ...
Posted by Michael Wonder on 27 Nov 2021
It’s time to better support the three million Canadians with a rare disease
25 November 2021 - After finding out her private health insurer would no longer pay for a drug helping with ...
Posted by Michael Wonder on 20 Nov 2021
FDA approves first drug to improve growth in children with most common form of dwarfism
19 November 2021 - Today, the U.S. Food and Drug Administration approved Voxzogo (vosoritide) injection to improve growth in children five ...
Posted by Michael Wonder on 13 Nov 2021
NICE does not recommend elosulfase alfa for NHS use following evaluation of new evidence
12 November 2021 - NICE has today published draft guidance for public consultation which does not recommend elosulfase alfa (also called ...
Posted by Michael Wonder on 08 Nov 2021
Drugs against rare diseases: Libmeldy gene therapy shows additional benefits - Zolgensma cell therapy, on the other hand, does not
4 November 2021 - The Federal Joint Committee (G-BA) today assessed the additional benefit of two active ingredients against very rare, ...
Posted by Michael Wonder on 09 Oct 2021
FDA approves innovative treatment for paediatric patients with congenital athymia
8 October 2021 - Today, the U.S. FDA approved Rethymic for the treatment of paediatric patients with congenital athymia, a rare ...
Posted by Michael Wonder on 29 Sep 2021
U.S. FDA approves Livmarli (maralixibat) as the first and only approved medication for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older
29 September 2021 - Mirum Pharmaceuticals today announced that the U.S. FDA has approved Livmarli (maralixibat) oral solution for the treatment ...
Posted by Michael Wonder on 15 Sep 2021
Aduhelm backlash threatens to reverse progress in FDA’s reviews of rare and ultra-rare disease drugs
14 September 2021 - The FDA’s approval of Aduhelm to treat Alzheimer’s disease has unleashed criticism about the decision and ...
Posted by Michael Wonder on 09 Sep 2021
Albireo receives UK MHRA approval of Bylvay (odevixibat)
8 September 2021 - Bylvay now approved in U.S., EU and UK as first drug treatment for patients with progressive familial intrahepatic ...