Blog
RSS FeedPosted by Michael Wonder on 08 Mar 2023
Genethon given PRIME status by EMA for gene therapy to treat Crigler-Najjar syndrome, a rare liver disease
7 March 2023 - Genethon today announced that the EMA has granted PRIME (Priority Medicines) status to the gene therapy, ...
Posted by Michael Wonder on 28 Feb 2023
My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility.
28 February 2023 - My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the ...
Posted by Michael Wonder on 24 Feb 2023
Chiesi Global Rare Diseases and Protalix BioTherapeutics receive positive CHMP opinion for pegunigalsidase alfa for treatment of Fabry disease
24 February 2023 - European Commission decision anticipated in beginning of May 2023. ...
Posted 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 28 Jan 2023
Ipsen receives CHMP negative opinion for palovarotene as a treatment for fibrodysplasia ossificans progressiva in EU
27 January 2023 - Ipsen to request re-examination of CHMP opinion on palovarotene as a potential treatment for fibrodysplasia ossificans progressiva ...
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 18 Sep 2022
Agios receives positive CHMP opinion for Pyrukynd (mitapivat) for the treatment of pyruvate kinase deficiency in adult patients
16 September 2022 - Agios expects a decision on the marketing authorisation application by the European Commission within 67 days of ...
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 Aug 2022
Patient registries: EMA officials highlight opportunities in orphan drug development
25 August 2022 - Officials from the EMA tout the benefits of patient registries to support regulatory decision making for ...
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 23 Jul 2022
Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
22 July 2022 - Positive opinion based on HELIOS-A Phase 3 study. ...