Genprex receives U.S. FDA fast track designation for gene therapy that targets lung cancer

21 January 2020 - Genprex today announced that the U.S FDA has granted fast track Designation for Genprex’s Oncoprex immunogene ...

Read more →

BioMarin explores pricing experimental gene therapy at $2 million to $3 million

16 January 2020 - Valrox would be first gene therapy approved in U.S. for inherited type of haemophilia. ...

Read more →

B.C. funding decision on pricey drug leaves teen with rare disease facing 'scary' future

13 January 2020 - Miles Ambridge just misses cut-off age to access Spinraza, a potentially life-changing treatment. ...

Read more →

Roche aims to 'underwhelm' on SMA drug price to challenge rivals

14 January 2020 - Swiss drug maker Roche plans to price its oral spinal muscular atrophy drug risdiplam aggressively to challenge ...

Read more →

PTC Therapeutics provides corporate update

13 January 2020 - PTC-AADC MAA submitted; BLA now expected to be submitted in 2Q 2020. ...

Read more →

At 16, she’s a pioneer in the fight to cure sickle cell disease

11 January 2020 - Helen Obando is the youngest person ever to get a gene therapy that scientists hope will cure ...

Read more →

New CADTH process for cell and gene therapies

9 January 2020 - CADTH has undertaken an internal review of our processes for drugs and devices, and established a revised ...

Read more →

Aruvant receives FDA rare paediatric disease designation for ARU-1801 for the treatment of sickle cell disease

8 January 2020 - ARU-1801 is being developed as a potential one-time treatment for patients suffering from sickle cell disease and ...

Read more →

ICER posts draft scoping document for the assessment of treatments for beta thalassaemia

6 January 2020 - Document open to public comment until 27 January 2020. ...

Read more →

Canadian parents of babies with rare deadly disease look to Novartis treatment lottery

5 January 2020 - Canadian babies with a rare and often fatal muscle-wasting disease may soon have access to a ...

Read more →

ICER to assess gene therapy for haemophilia A

3 January 2020 - Report will be subject of New England CEPAC meeting in August 2020; open Input now being accepted ...

Read more →

NICE refuses Akcea’s Waylivra

3 January 2020 - NICE has issued an Evaluation Consultation Document, refusing Waylivra (volanesorsen) as a treatment for familial chylomicronaemia ...

Read more →

Toronto parents of baby needing $2.8 million drug turn to Swiss drug maker's dose lottery as a last resort

31 December 2019 - Critics call on Ottawa to improve access to expensive drugs that treat rare diseases. ...

Read more →

BioMarin submits biologics license application to U.S. FDA for valoctocogene roxaparvovec to treat haemophilia A

23 December 2019 - First marketing application submission in U.S. for gene therapy directed at any type of haemophilia. ...

Read more →

European Medicines Agency validates BioMarin's marketing authorisation application for valoctocogene roxaparvovec to treat severe haemophilia A

23 December 2019 - Potential first gene therapy in Europe directed at any type of haemophilia. ...

Read more →