Blog
RSS FeedPosted by Michael Wonder on 20 Jul 2020
Rare disease drove two women on a mission to change FDA drug review process
19 July 2020 - Melissa Goetz and Lindsey Sutton are campaigning to improve the US drug review process for rare diseases. ...
Posted by Michael Wonder on 19 Jun 2020
Dicerna receives rare paediatric disease designation from U.S. FDA for nedosiran for the treatment of primary hyperoxaluria
18 June 2020 - Dicerna Pharmaceuticals today announced that the U.S. FDA has granted rare paediatric disease designation for nedosiran, ...
Posted by Michael Wonder on 11 Jun 2020
Rare disease therapy development and access remain top FDA priorities during COVID-19
11 June 2020 - The FDA’s work on behalf of people with rare diseases is more important than ever as these ...
Posted by Michael Wonder on 03 Jun 2020
Clarity Pharmaceuticals announces that the US FDA grants rare paediatric disease designation to Copper Cu 67 sartate for the treatment of neuroblastoma
3 June 2020 - Clarity Pharmaceuticals is pleased to announce that the U.S. FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 26 Mar 2020
Gilead declines 'rare disease' status for experimental coronavirus drug
25 March 2020 - In a surprising turnabout, drugmaker Gilead Sciences asked the FDA on Wednesday to rescind orphan status ...
Posted by Michael Wonder on 26 Mar 2020
Orphan Technologies receives rare paediatric disease designation from FDA for OT-58 to treat cystathionine B-synthase deficiency homocystinuria
26 March 2020 - Orphan Technologies today announced that OT-58 for the treatment of cystathionine beta synthase deficiency homocystinuria has ...
Posted by Michael Wonder on 23 Feb 2020
Rare Disease Day 2020: FDA continues important work on treatments for rare diseases
21 February 2020 - As leaders of the FDA, every day we see the unwavering commitment of our agency in working ...
Posted by Michael Wonder on 16 Feb 2020
After a new version of a decades-old drug gets orphan status, the price suddenly skyrockets
12 February 2020 - For years, hospitals and clinics have used an injectable medicine called dehydrated alcohol to treat such ...
Posted by Michael Wonder on 30 Jan 2020
ProQR receives rare paediatric disease designation from FDA for QR-421a
30 January 2020 - Designation is for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 ...
Posted by Michael Wonder on 19 Jan 2020
FDA harnesses technology and collaboration to support rare disease product development
17 January 2020 - Announcing an orphan drug technology modernisation effort and the 2020 FDA Rare Disease Day meeting. ...
Posted by Michael Wonder on 02 Jan 2020
Does an orphan drug policy make a difference in access? A comparison of Canada and Australia.
2 January 2020 - Canada has been discussing whether to implement an orphan drug policy for more than 25 years. Recently, ...
Posted by Michael Wonder on 11 Dec 2019
Vertex announces European Commission approval for Kalydeco (ivacaftor) in infants with cystic fibrosis ages 6 months to less than 12 months with certain mutations in the CFTR gene
10 December 2019 - Ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic ...
Posted by Michael Wonder on 27 Nov 2019
Our drug policy often prioritises eliminating rare disease. One researcher asks: Is that the right goal?
26 November 2019 - Dr. Peter Bach wants to turn the entire philosophical underpinnings of America’s health care system upside ...
Posted by Michael Wonder on 11 Nov 2019
FDA admits it goofed when granting orphan status to an opioid addiction treatment
8 November 2019 - In an unusual move, the FDA has acknowledged a mistake and revoked orphan drug status for ...
Posted by Michael Wonder on 01 Nov 2019
Vertex announces EMA marketing authorisation application validation for VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination treatment in cystic fibrosis
31 October 2019 - Application supported by positive results from two global Phase 3 studies in people with cystic fibrosis ages ...