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 ...

Read more →

NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy

2 June 2021 - NICE has today published draft guidance for public consultation which does not recommend risdiplam (Evrysdi, Roche) for ...

Read more →

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 ...

Read more →

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. ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →