Blog
RSS FeedPosted by Michael Wonder on 22 Jul 2021
bluebird bio receives EC approval for Skysona (elivaldogene autotemcel, Lenti-D) gene therapy for patients less than 18 years of age with early cerebral adrenoleukodystrophy without matched sibling donor
21 July 2021 - Skysona is the first and only gene therapy approved in the European Union to treat early CALD. ...
Posted by Michael Wonder on 14 Jul 2021
PHARMAC 'cruel' for failing to fund drugs for rare illnesses — advocate
14 July 2021 - New Zealand’s drug-buying agency doesn’t value the lives of all people equally, according to those advocating for ...
Posted by Michael Wonder on 01 Jul 2021
LEXEO Therapeutics receives rare paediatric disease designation and orphan drug designation for LX2006 for the treatment of Friedreich’s ataxia
30 June 2021 - Phase I/II clinical trial in patients with cardiomyopathy associated with Friedreich’s ataxia expected to initiate in 2021. ...
Posted by Michael Wonder on 28 Jun 2021
AskBio receives FDA fast track designation for LION-101, a novel investigational AAV gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)
28 June 2021 - LGMD2I/R9 is a rare form of muscular dystrophy with no approved therapies. ...
Posted by Michael Wonder on 27 Jun 2021
BioMarin receives positive CHMP opinion in Europe for vosoritide for the treatment of children with achondroplasia from age 2 until growth plates close
25 June 2021 - Temporary Authorization for Use (ATU) granted in France to allow access and reimbursement of vosoritide to begin ...
Posted by Michael Wonder on 23 Jun 2021
FDA should lead the way on new ALS treatments, not Canada and Europe
22 June 2021 - Following the FDA’s recent decision to give the green light to aducanumab, the first treatment approved ...
Posted by Michael Wonder on 23 Jun 2021
Rare disease patient advocates continue battle for affordable drugs
23 June 2021 - Beth Vanstone, mother of local Cystic Fibrosis Warrior Madi Vanstone and director of the CF Get ...
Posted by Michael Wonder on 18 Jun 2021
Orphazyme provides regulatory update from FDA on arimoclomol for Niemann-Pick disease type C
18 June 2021 - Orphazyme today announced it has received a complete response letter from the U.S. FDA following its ...
Posted by Michael Wonder on 02 Jun 2021
NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy
2 June 2021 - NICE has today published draft guidance for public consultation which does not recommend risdiplam (Evrysdi, Roche) for ...
Posted by Michael Wonder on 31 May 2021
Examining the impact of different country processes for appraising rare disease treatments: a case study analysis
31 May 2021 - Conventional appraisal and reimbursement processes are being challenged by the increasing number of rare disease treatments with ...
Posted by Michael Wonder on 29 May 2021
Ipsen confirms U.S. FDA accepts new drug application for palovarotene as the first potential treatment worldwide for fibrodysplasia ossificans progressiva
28 May 2021 - New drug application granted priority review status, with a decision anticipated on 30 November 2021. ...
Posted by Michael Wonder on 25 May 2021
Scholar Rock receives fast track designation from the U.S. FDA for apitegromab for the treatment of patients with spinal muscular atrophy
24 May 2021 - Builds on Priority Medicines (PRIME) designation recently granted by the EMA recognising the unmet medical needs of ...
Posted by Michael Wonder on 15 May 2021
Apellis announces U.S. FDA approval of Empaveli (pegcetacoplan) for adults with paroxysmal nocturnal hemoglobinuria
14 May 2021 - Empaveli was superior to Soliris for the change from baseline in haemoglobin level at Week 16 in ...
Posted by Michael Wonder on 13 May 2021
Fulcrum Therapeutics announces U.S. FDA grants fast track designation to losmapimod for the potential treatment of facioscapulohumeral muscular dystrophy
12 May 2021 - Fulcrum Therapeutics today announced that the U.S. FDA has granted fast track designation to losmapimod for ...
Posted by Michael Wonder on 05 May 2021
Gilead readies to sell anticancer drug Trodelvy in Korea
4 May 2021 - Gilead’s antibody-drug conjugate Trodelvy (sacituzumab govitecan) has won the Ministry of Food and Drug Safety’s designation ...