Blog
RSS FeedPosted 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
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 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 ...
Posted by Michael Wonder on 26 Jan 2021
Recordati Rare Diseases: Carbaglu (carglumic acid) receives U.S. FDA approval for a new indication to treat acute hyperammonaemia associated with propionic acidemia and methylmalonic acidemia
26 January 2021 - Carbaglu is first and only FDA approved medication for hyperammonaemia associated with these rare conditions. ...
Posted by Michael Wonder on 02 Dec 2020
Amicus Therapeutics initiates rolling biologic license application to the U.S. FDA for AT-GAA in late-onset Pompe disease
1 December 2020 - On track for completing the BLA submission in 1H2021. ...
Posted by Michael Wonder on 28 Nov 2020
Alnylam announces innovative value-based agreement framework for Oxlumo (lumasiran) to accelerate access for patients with primary hyperoxaluria type 1 and deliver ultra-rare orphan disease pricing solutions to U.S. payers
24 November 2020 - Expedited access to Oxlumo aims to support children and adults living with PH1 who face inevitable disease ...
Posted by Michael Wonder on 24 Nov 2020
FDA approves first drug to treat rare metabolic disorder
23 November 2020 - Today, the U.S. FDA approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1, a ...
Posted by Michael Wonder on 21 Nov 2020
FDA approves first treatment for Hutchinson-Gilford progeria syndrome and some progeroid laminopathies
20 November 2020 - Today, the U.S. FDA approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford ...