Blog
RSS FeedPosted 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NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy
2 June 2021 - NICE has today published draft guidance for public consultation which does not recommend risdiplam (Evrysdi, Roche) for ...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 ...
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 ...
Posted by Michael Wonder on 28 Apr 2021
Biogen to expand access to its ALS drug, but move may come too late for some patient
27 April 2021 - After weeks of controversy, Biogen has agreed to provide an experimental drug for combating ALS to ...
Posted by Michael Wonder on 20 Apr 2021
Alnylam submits new drug application with U.S. FDA for vutrisiran for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults
19 April 2021 - Alnylam has submitted a new drug application to the U.S. FDA for the approval of vutrisiran for ...
Posted by Michael Wonder on 19 Apr 2021
Citrine Medicine and Sinopharm Group announce strategic partnership to broaden and accelerate access to rare disease drugs in China
19 April 2021 - Citrine Medicine and Sinopharm Group today announced a strategic partnership to accelerate the commercialisation of rare ...
Posted by Michael Wonder on 11 Apr 2021
Masterton woman feels abandoned after medical funding rejected
9 April 2021 - Masterton's Allyson Lock feels she is not part of the "team of five million" after her ...
Posted by Michael Wonder on 11 Mar 2021
Denali Therapeutics announces fast track designation granted by the U.S. FDA to ETV:IDS (DNL310) for the treatment of patients with Hunter syndrome
11 March 2021 - Denali Therapeutics today announced that the U.S. FDA has granted fast track designation to ETV:IDS (DNL310) for ...
Posted by Michael Wonder on 01 Mar 2021
Rare Disease Day 2021: FDA shows sustained support of rare disease product development during the public health emergency
1 March 2021 - Rare Disease Day is a time to reflect on both the progress that has been made, and ...