Blog
RSS FeedPosted by Michael Wonder on 23 Feb 2023
FDA continues important work to advance medical products for patients with rare diseases
23 February 2023 - Patients with rare diseases are experts in their health condition. They provide a unique perspective, and ...
Posted by Michael Wonder on 17 Feb 2023
Chiesi Global Rare Diseases announces FDA approval of Lamzede (velmanase alfa-tycv) for alfa mannosidosis
16 February 2023 - First and only enzyme replacement therapy for the treatment of non-central nervous system manifestations of alfa mannosidosis ...
Posted by Michael Wonder on 23 Jan 2023
Despite court ruling, FDA will continue with its approach to approving orphan drug exclusivity
23 January 2023 - In an unexpected move, the FDA will continue to apply exclusive marketing rights for so called ...
Posted by Michael Wonder on 04 Oct 2022
Rare disease endpoint advancement pilot program
3 October 2022 - FDA is establishing a Rare Disease Endpoint Advancement (RDEA) Pilot Program to support novel efficacy endpoint development ...
Posted by Michael Wonder on 12 Sep 2022
Chiesi Global Rare Diseases announces FDA acceptance of BLA filing for velmanase alfa for the proposed treatment of alfa mannosidosis
12 September 2022 - FDA grants priority review designation with PDUFA date in the first half of 2023. ...
Posted by Michael Wonder on 07 Sep 2022
The FDA needs to be more flexible in assessing treatments for rare diseases, like the one that seemed to help my son
7 September 2022 - Every time I read about clinical trials testing possible treatments for rare diseases, I think of ...
Posted by Michael Wonder on 31 Aug 2022
FDA approves first treatment for acid sphingomyelinase deficiency, a rare genetic disease
31 August 2022 - Today, the US FDA approved Xenpozyme (Olipudase alfa) for intravenous infusion in paediatric and adult patients with ...
Posted by Michael Wonder on 26 Jul 2022
‘No magic bullet’: For drug makers and the FDA, clinical trials on ultra rare diseases pose thorny challenges
26 July 2022 - Walker Burger is beside himself with worry. ...
Posted by Michael Wonder on 07 Jul 2022
US FDA awards rare paediatric disease designation to paxalisib for AT/RT, a rare form of childhood brain cancer
6 July 2022 - Kazia Therapeutics is pleased to announce that the U.S. FDA has awarded rare paediatric disease designation ...
Posted 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 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 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 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 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 ...