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RSS FeedPosted 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 24 Aug 2021
FDA, drug makers propose new pilots aimed at speeding rare disease drugs, as part of user fee update
23 August 2021 - The FDA and the drug industry have jointly agreed to create several new pilot programs aimed ...
Posted by Michael Wonder on 27 Jul 2021
Health Canada releases What We Heard Report from the public engagement on the National Strategy for Drugs for Rare Diseases
26 July 2021 - Too many Canadians are struggling to access the drugs they need, especially if they have a rare ...
Posted 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 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 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 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 28 Apr 2021
Vertex announces European Commission approval for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor to treat cystic fibrosis patients 12 years and older with at least one F508del mutation in the CFTR gene
28 April 2021 - New indication includes people ages 12 years and older who have one copy of the F508del mutation ...