Blog
RSS FeedPosted by Michael Wonder on 02 Nov 2022
ICER publishes evidence report on gene therapies for haemophilia A and B
2 November 2022 - Duration of benefit with gene therapies and risks for rare side effects remain major uncertainties. ...
Posted by Michael Wonder on 27 Oct 2022
AvroBio receives rare paediatric disease designation from the US FDA for first in class gene therapy for Gaucher disease
27 October 2022 - AVR-RD-02 has previously received fast track status from FDA, orphan drug designation in the US and EU, ...
Posted by Michael Wonder on 18 Oct 2022
AvroBio granted ILAP designation from UK MHRA for first in class gene therapy for Gaucher disease
18 October 2022 - ILAP intended to accelerate regulatory review process and facilitate patient access in UK for seriously debilitating and ...
Posted by Michael Wonder on 17 Oct 2022
Biogen announces FDA’s 3 month extension of review period for the new drug application for tofersen
17 October 2022 - The new Prescription Drug User Fee Act action date set by the FDA is 25 April ...
Posted by Michael Wonder on 17 Oct 2022
Gene therapy Luxturna now reimbursed in Quebec for people with previously untreatable inherited vision loss
17 October 2022 - Quebec leads the way as the first province to reimburse Luxturna (voretigene neparvovec) for previously untreatable inherited ...
Posted by Michael Wonder on 13 Oct 2022
FDA accepts BioMarin's biologics license application for valoctocogene roxaparvovec AAV gene therapy for adults with severe haemophilia A
12 October 2022 - PDUFA target action date is 31 March 2023 ...
Posted by Michael Wonder on 12 Oct 2022
‘Current standard for stopping insurance benefits for SMA treatment harsh on adult patients’
12 October 2022 - Spinraza (nusinersen sodium), the first spinal muscular atrophy treatment that won the insurance coverage quickly since ...
Posted by Michael Wonder on 07 Oct 2022
Alnylam announces FDA approval of supplemental new drug application for Oxlumo (lumasiran) in advanced primary hyperoxaluria type 1
6 October 2022 - Approval is based on positive efficacy and safety results of the ILLUMINATE-C Phase 3 study of Oxlumo ...
Posted by Michael Wonder on 03 Oct 2022
‘Underlying guilt’ turns to delight after PHARMAC decision
30 September 2022 - Lani McLeod has had an underlying feeling of guilt for two years that her boys had ...
Posted by Michael Wonder on 29 Sep 2022
BioMarin resubmits biologics license application for valoctocogene roxaparvovec AAV gene therapy for severe haemophilia A to the FDA
29 September 2022 - BLA includes substantial body of data from pivotal Phase 3 and on-going Phase 1/2 studies. ...
Posted by Michael Wonder on 29 Sep 2022
Auckland toddler may be able to dance again after PHARMAC proposes funding drug
29 September 2022 - An Auckland toddler who was losing the ability to walk may be able to dance again, ...
Posted by Michael Wonder on 27 Sep 2022
Carvykti (ciltacabtagene autoleucel) receives approval from Japan’s MHLW for the treatment of patients with relapsed or refractory multiple myeloma
28 September 2022 - Legend Biotech announced today that Japan’s MHLW has approved Carvykti (ciltacabtagene autoleucel), a B-cell maturation antigen ...
Posted by Michael Wonder on 27 Sep 2022
PHARMAC seeking feedback on nusinersen sodium and adrenaline auto-injectors funding
28 September 2022 - PHARMAC has initiated two consultations today, seeking feedback on the funding of two different medicines that we've ...
Posted by Michael Wonder on 21 Sep 2022
Alnylam receives approval in Europe for Amvuttra (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
20 September 2022 - Amvuttra demonstrated halting or reversal in neuropathy impairment with subcutaneous administration once every three months. ...
Posted by Michael Wonder on 21 Sep 2022
Novartis and the pan-Canadian Pharmaceutical Alliance conclude negotiations for Luxturna, a gene therapy for previously untreatable inherited vision loss
20 September 2022 - Novartis Pharmaceuticals Canada and the pan Canadian Pharmaceutical Alliance have successfully concluded negotiations for Luxturna (voretigene neparvovec), ...