Blog
RSS FeedPosted by Michael Wonder on 14 Jan 2019
A revolutionary drug that could treat a rare and devastating disease is prohibitively expensive. But one state has a plan to pay for its potential $5 million price tag.
13 January 2019 - A one-time treatment for a devastating rare disease could be paid for with an instalment plan as ...
Posted by Michael Wonder on 02 Jan 2019
ProQR receives fast track designation from FDA for QR-421a for Usher Syndrome Type 2
2 January 2019 - ProQR Therapeutics today announced that it received fast track designation from the FDA for QR-421a. ...
Posted by Michael Wonder on 23 Dec 2018
Gritstone Oncology announces FDA fast track designation for GRANITE-001 for the treatment of colorectal cancer
20 December 2018 - Gritstone Oncology today announced that the U.S. FDA has granted fast track designation to GRANITE-001 for the ...
Posted by Michael Wonder on 03 Dec 2018
Novartis announces FDA filing acceptance and priority review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1
3 December 2018 - The AVXS-101, now known as Zolgensma (onasemnogene abeparvovec-xxxx), filing is supported by data from the START trial ...
Posted by Michael Wonder on 28 Nov 2018
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy and fast track designations for RP-L102 gene therapy for Fanconi anaemia
27 November 2018 - Global trial of RP-L102 utilising no conditioning and “Process B” on track for early 2019. ...
Posted by Michael Wonder on 19 Nov 2018
Gottlieb pushes for funding to speed gene therapy reviews
16 November 2018 - The FDA is working to increase its investment in reviewing gene therapy products, Commissioner Scott Gottlieb ...
Posted by Michael Wonder on 05 Nov 2018
Novartis says SMA gene therapy is cost-effective at $4-5 million per patient
5 November 2018 - Novartis, which is shifting into rare diseases, said on Monday it believes its new gene therapy ...
Posted by Michael Wonder on 08 Oct 2018
Akcea announces its access and distribution strategy for Tegsedi (inotersen)
5 October 2018 - Express Scripts’ Accredo selected as specialty pharmacy for its expertise in providing timely access and support services ...
Posted by Michael Wonder on 05 Oct 2018
Akcea and Ionis receive FDA approval of Tegsedi (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
5 October 2018 - Tegsedi provides powerful knockdown of TTR protein, delivering significant and sustained benefits in neuropathy and quality of ...
Posted by Michael Wonder on 21 Sep 2018
Adverum Biotechnologies receives fast track designation for ADVM-022 gene therapy for the treatment of wAMD
19 September 2018 - ADVM-022 is a unique single-administration gene therapy delivered intra-vitreally for the treatment of wAMD. ...
Posted by Michael Wonder on 11 Sep 2018
Amicus Therapeutics announces regulatory and clinical updates for AT-GAA in Pompe disease
10 September 2018 - Amicus Therapeutics announced today regulatory and clinical advancements in its development program AT-GAA for Pompe disease. ...
Posted by Michael Wonder on 07 Sep 2018
Fibrocell announces FDA fast track designation of FCX-013 for treatment of moderate to severe localised scleroderma
5 September 2018 - Fibrocell Science today announced that the U.S. FDA has granted fast track designation to FCX-013, the Company’s ...
Posted by Michael Wonder on 21 Aug 2018
MeiraGTx announces AAV-CNGB3 granted fast track designation by U.S. FDA for treatment of achromatopsia
20 August 2018 - MeiraGTx today announced that the U.S. FDA has granted fast track designation for its AAV-CNGB3 gene therapy ...
Posted by Michael Wonder on 12 Aug 2018
Alnylam prices first gene silencing drug at $450,000 per patient, but offers money-back guarantee
10 August 2018 - This morning, after 16 years and $2.5 billion in investment, the Cambridge, Massachusetts-based company Alnylam finally ...
Posted by Michael Wonder on 11 Aug 2018
FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease
10 August 2018 - The U.S. FDA today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused ...