Blog
RSS FeedPosted by Michael Wonder on 04 Sep 2022
Australia among world's best in treating debilitating and often fatal muscle disorder
4 September - Australia has become one of the first countries in the world to fund a new, next-generation treatment ...
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. ...
Posted by Michael Wonder on 18 Jul 2022
Velmanase alfa for the treatment of alfa mannosidosis
15 July 2022 - The Department of Health and Social Care has asked NICE to produce guidance on using velmanase alfa ...
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 06 Jul 2022
Setmelanotide for the treatment of patients with obesity caused by LEPR or POMC deficiency
6 July 2022 - NICE has issued fvidence-based recommendations on setmelanotide (Imcivree) for treating obesity caused by LEPR or POMC ...
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 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 ...